Clinical trial NCT02828111
Conditions: Basal Cell Carcinomas
Keywords: Gorlin Syndrome Basal Cell Nevus Syndrome BCNS nevoid basal cell carcinoma syndrome Basal cell carcinoma Hedgehog inhibitor Surgically Eligible Basal Cell Carcinomas High Frequency Basal Cell Carcinomas
Sponsor: PellePharm, Inc.
Start date: 2016-11-11; Completion date: 2017-11-09
Enrollment: 36 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle in patients with Basal Cell Carcinoma. One investigational center (metasite) in the United States will participate in this study. Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four sequential cohorts. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or vehicle gel.

Detailed description:
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle. Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four sequential cohorts. As soon as one cohort has been completely enrolled, the next cohort will be enrolled. Each subject will treat no more than two previously untreated biopsy confirmed treatment-targeted nodular BCCs. If the subject has additional non-treatment targeted BCCs they can be treated surgically prior to or during the trial. Within each cohort subjects will be randomized in a 2:1 ratio to receive active or vehicle gel. The sequential cohorts will be:

* Cohort 1: patidegib gel 2% or vehicle, once daily
* Cohort 2: patidegib gel 4% or vehicle, once daily
* Cohort 3: patidegib gel 2% or vehicle, twice daily
* Cohort 4: patidegib gel 4% or vehicle, twice daily

The study drug will be applied topically to the treatment-targeted BCCs and a rim of adjacent skin for 12 weeks. Information on reported and observed adverse events (AEs) will be obtained at each visit. An abbreviated physical examination (PE) will be performed at Baseline and Week 12. The treatment-targeted BCCs will be identified by the Investigator at the Baseline visit and will be circled in ink at Baseline, Weeks 6 and 12 and photographed, and measured at all study visits (Baseline, Weeks 2, 6, 8, 10, and 12). Blood samples for complete blood count and serum chemistry and urine for urinalysis will be collected from subjects at Screening, Week 6, and Week 12. Subjects who terminate study participation early will be asked to complete all Week 12 assessments, as appropriate, prior to commencement of any alternative therapy for BCCs (if possible). Subjects who discontinue from the study during the treatment period will not be replaced.

Arms: arm 1: Patidegib gel 2%, applied topically, once daily for 12 weeks (Cohort 1) arm 2: Patidegib gel 4%, applied topically, once daily for 12 weeks (Cohort 2) arm 3: Vehicle gel, applied topically, once daily for 12 weeks (Cohort 1) arm 4: Patidegib gel 2%, applied topically, twice daily for 12 weeks (Cohort 3) arm 5: Patidegib gel 4%, applied topically, twice daily for 12 weeks (Cohort 4) arm 6: Vehicle gel, applied topically, once daily for 12 weeks (Cohort 2) arm 7: Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 3) arm 8: Vehicle gel, applied topically, twice daily for 12 weeks (Cohort 4)
Interventions: intervention 1: Patidegib intervention 2: Vehicle gel
Intervention descriptions: intervention 1: None intervention 2: None

Protocol documents:
Prot_001.pdf: 
PellePharm, Inc. 
Dose Escalating Study in Sporadic Basal Cell Carcinomas 
 
 
Pelle-926-202, V4, 24AUG2017 
PROPRIETARY AND CONFIDENTIAL 
 
Page 1 of 71 
 
Clinical Study Protocol 
 
Protocol Pelle-926-202 
 
Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of Concept Clinical 
Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the 
GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas 
Development Phase of Study: 
2A 
Study design: 
 
IND #: 
Multicenter, Double-Blind, Dose Escalating, 
Vehicle-Controlled Clinical Study 
125461 
Date of Original Protocol: 
Amendment 1— Protocol Version 
2.0 
Amendment 2--- Protocol Version 
3.0 
Amendment 3--- Protocol Version 
4.0 
23 June 2016 
22 September 2016 
 
16 August 2017 
 
24 August 2017 
Sponsor Representative: 
Ervin Epstein, M.D. 
Chief Medical Officer 
Phone: 
 
Email: 
 
Sponsor: 
PellePharm, Inc. 
275 Middlefield Rd., Suite 100 
Menlo Park, CA 94025 
 
CONFIDENTIAL 
Nothing herein is to be disclosed without prior approval of the sponsor. 
 
This protocol will be conducted in compliance with procedures outlined in this document, Good 
Clinical Practice (GCP) guidelines and applicable regulatory requirements.  This study will not be 
initiated without the approval of the Institutional Review Board (IRB) or Independent Ethics 
Committee (IEC).  Any changes to the protocol will be approved in writing by the IRB/IEC before 
implementation except where necessary to eliminate an immediate harm to the patient. 
PPD
PPD


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Protocol Review and Approvals 
 
Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of Concept Clinical 
Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the 
GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas 
 
Reviewed and approved: 
 
Ervin Epstein M.D. 
 
 
 
 
Chief Medical Officer 
Signature 
Date 
PellePharm, Inc. 
 
 
 


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Dose Escalating Study in Sporadic Basal Cell Carcinomas 
 
 
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PROPRIETARY AND CONFIDENTIAL 
 
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Principal Investigator Protocol Agreement Page 
I have carefully read the protocol entitled: “Double-Blind, Dose Escalating, Randomized, Vehicle-
Controlled Proof of Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once 
or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas” 
and, I declare that as a Principal Investigator the clinical protocol was subject to critical review 
and is approved by the Sponsor.  
I agree to conduct this study in compliance with procedures outlined in this document according 
to International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines, the 
Declaration of Helsinki, and applicable regulatory requirements.  This study will not be initiated 
without the approval of the Institutional Review Board (IRB) or Independent Ethics Committee 
(IEC) and the competent authority, if applicable. 
I understand that any substantial changes to the protocol must be approved in writing by the 
IRB/IEC and the competent authority, if applicable, before it can be implemented except where 
necessary to eliminate immediate harm to the subject.  I will provide copies of the protocol and 
access to all information furnished by PellePharm to study personnel under my supervision and 
will discuss this material with them to ensure they are fully informed about the study. I understand 
that the study may be terminated or enrollment suspended at any time by PellePharm with or 
without cause, or by me if it becomes necessary to protect the best interests of the study subjects. 
 
 
Investigator Signature 
 
 
 
 
 
Printed Name 
 
Date 
Institution Name 
 
 
Address 
 
 
City, State Zip Code 
 
Phone Number 


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1. SYNOPSIS 
Name of Sponsor: PellePharm, Inc. 
Name of Investigational Product: patidegib gel 
Name of Active Ingredients: patidegib HCl 
Title of Study: Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of 
Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily 
to Decrease GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas 
Number of Clinical Centers: One investigational center (metasite) in the United States will 
participate in this study. 
Objective: 
The primary objectives of the study are to evaluate the following: 
1. The safety and tolerability of treatment with patidegib gel 2%, 4%, or vehicle applied 
once or twice daily for 12 weeks. 
2. The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) 
signaling pathway (GLI1 biomarker) after treatment with patidegib gel 2%, 4%, or 
vehicle applied once or twice daily for 12 weeks to treatment-targeted BCCs. 
 
The secondary objective of the study is to evaluate the following: 
1. The clinical efficacy of patidegib as defined by the percent decrease in greatest 
diameter of baseline treatment-targeted basal cell carcinomas after 12 weeks of 
treatment. Treatment-targeted Basal Cell Carcinomas (BCCs) will be biopsied prior 
to treatment to confirm the clinical diagnosis of nodular BCC. Eligible tumors will 
have the clinical features of nodular BCC and prior to biopsy will be no less than 5 
mm or greater than 15 mm in greatest diameter on the face (excluding the nose and 
periorbital skin) and no less than 9 mm or greater than 20 mm in greatest diameter at 
sites other than the face. 
 
Exploratory objective: 
1. To evaluate the utility of an investigator static global tumor assessment (ISGTA) in 
assessing the proportion of baseline treatment targeted basal cell carcinomas that are 
evaluated as being clear or almost clear. 
2. To evaluate the utility of blinded assessment of change in BCC size using photographs 


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Methodology: 
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to 
compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in 
comparison with that of vehicle.  
Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four 
sequential cohorts. As soon as one cohort has been completely enrolled, the next cohort will 
be enrolled. Each subject will treat no more than two previously untreated biopsy confirmed 
treatment-targeted nodular BCCs. If the subject has additional non-treatment targeted BCCs 
they can be treated surgically or with topical agents prior to or during the trial.  Within each 
cohort subjects will be randomized in a 2:1 ratio to receive active or vehicle gel. The 
sequential cohorts will be: 
• Cohort 1:  patidegib gel 2% or vehicle, once daily 
• Cohort 2:  patidegib gel 4% or vehicle, once daily  
• Cohort 3:  patidegib gel 2% or vehicle, twice daily 
• Cohort 4:  patidegib gel 4% or vehicle, twice daily  
 
The study drug will be applied topically to the treatment-targeted BCCs and a rim or 
adjacent skin for 12 weeks. 
Information on reported and observed adverse events (AEs) will be obtained at each visit. 
An abbreviated physical examination (PE) will be performed at Baseline and Week 12.  
The treatment-targeted BCCs will be identified by the Investigator at the Baseline visit and 
will be circled in ink at Baseline, Weeks 6 and 12, and photographed, and measured at all 
study visits (Baseline, Weeks 2, 6, 8, 10, and 12). 
Blood samples for complete blood count and serum chemistry and urine for urinalysis will 
be collected from subjects at Screening, Week 6, and Week 12.  
Subjects who terminate study participation early will be asked to complete all Week 12 
assessments, as appropriate, prior to commencement of any alternative therapy for BCCs (if 
possible). Subjects who discontinue from the study during the treatment period will not be 
replaced. 
If signs or symptoms develop in the treatment areas during the treatment period that restrict 
daily activities or make continued application of the study drug difficult due to discomfort, 
the Investigator may instruct the subject to interrupt use of the study drug temporarily and to 
resume application of the study drug once the signs and symptoms have resolved adequately 
to the point that the Investigator believes that treatment can safely be resumed. The 


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Investigator should try to minimize study drug interruptions; and if needed, make best efforts 
to limit a “drug holiday” to 7 days. If the study drug interruption exceeds 4 consecutive days, 
the Investigator should consult with the Medical Monitor to determine a course of action. If 
the study drug is interrupted, discontinued, or a concomitant medication is used to treat a 
sign or symptom, an AE shall be recorded. 
Subjects who discontinue from the study due to clinically significant laboratory 
abnormalities, AEs or any other reason will be asked to complete all Week 12 evaluations. 
Any subject who has an AE during the treatment period will be monitored by the 
Investigator until resolution (return to normal or to the baseline state) or stabilization, as 
determined by the Investigator. 
In addition, application of study drug may be delayed or halted at any time if ongoing safety 
data evaluations raise concern for subject safety. If the subject participation is suspended, all 
of the subject’s safety data will be reviewed by the Medical Monitor in conjunction with the 
Investigator to determine the course of action. 
Number of Subjects Planned: 
Approximately 36 subjects who meet the study entry criteria will be enrolled into one of four 
sequential cohorts.  
Inclusion Criteria: 
1. 
The subject is from 18 to 85 years of age, inclusive. 
2. 
The subject must provide electronic informed consent prior to any study 
procedures.  
3. 
If the subject is a woman of childbearing potential1, she is willing to use two 
effective methods of birth control during the duration of the trial and for one month 
after the last application of the gel. The two forms of birth control authorized are 
defined as the use of a barrier method of contraception (condom with spermicide) 
in association with one of the following methods of birth control: bilateral tubal 
ligation; combined oral contraceptives (estrogens and progesterone) or implanted 
or injectable contraceptives with a stable dose for at least 1 month prior to Baseline; 
hormonal intra-uterine device (IUD) inserted at least 1 month prior to Baseline. 
This proscription is based on the key role of the HH pathway in embryogenesis, 
the known preclinical teratogenic effects of systemic cyclopamine, a naturally 
                                                 
1 For the purpose of this trial, a female not of childbearing potential is defined as a sexually mature woman who: 1) 
has undergone a hysterectomy or bilateral oophorectomy; or 2) has been naturally postmenopausal for at least 12 
consecutive months (i.e., has not had menses at any time in the preceding 12 consecutive months); or 3) is 41 years 
of age or older with physiologic symptoms of menopause and a Follicle Stimulating Hormone (FSH) level of ≥ 30 
IU/L or 4) whose male sexual partner has had a vasectomy. 


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occurring inhibitor of SMO, and the unknown level of systemic exposure following 
topical application of patidegib in humans.  
  
4. 
If the subject is a male with a female sexual partner who is of childbearing 
potential, the couple is willing to use two effective methods of birth control during 
the duration of the trial and for one month after the last application of the gel. 
Authorized birth control methods are outlined in Inclusion Criterion #3. Any 
woman of childbearing potential applying the gel to themselves, or assisting a 
subject, must comply with the same birth control measures.  
5. 
One or two previously untreated BCCs with the clinical features of a nodular BCC 
confirmed by a biopsy done at or prior to screening confirming nodular BCC.  
These tumors must be suitable for surgical excision. The BCCs prior to biopsy 
must be no less than 5 mm or greater than 15 mm in greatest diameter on the face 
and no less than 9 mm or more than 20 mm in greatest diameter at sites other than 
the face. Tumors on the nose, periorbital skin, or on or below the knee are excluded. 
6. 
The subject is willing to abstain from application of non-study topical prescription 
and over the counter medications within 5 cm of a treatment-targeted BCC for the 
duration of the study except as prescribed by the Investigator.  Moisturizers and 
emollients are allowable.  Subjects will be encouraged to use sunscreen with a 
sunscreen protection factor (SPF 15 or higher) at least once daily on all exposed 
skin sites. 
7. 
Female subjects must have negative serum pregnancy test at Screening. 
8. 
The subject is willing to contact the study center after each primary skin care 
physician (PSCP) visit to provide the study center details of the visit and any 
treatment of skin tumors. 
 
9. 
The subject is willing to forego alternative treatment of the treatment-targeted 
baseline BCC for the duration of the trial.  


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Exclusion Criteria: 
1. Subjects with basal cell nevus syndrome (BCNS, Gorlin syndrome, nevoid basal cell 
carcinoma syndrome; OMIM #109400). 
2. The subject has used topical products within 5 cm of a treatment- targeted BCC or 
systemic therapies that might interfere with the evaluation of the study medication 
during the study. Specifically, these include the use of: 
a. Topical glucocorticoids 30 days prior to screening 
b. Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) 
systemically or topically, or > 5% of an alphahydroxy acid (e.g., 
glycolic acid, lactic acid), photodynamic therapy (PDT), or 5-
fluorouracil or imiquimod (except as topical treatment to discrete 
BCCs) systemically or topically to the skin during the six months prior 
to entry. 
c. Systemic chemotherapy within one year prior to screening. (Note: field 
therapy with topically applied treatments can be done as long as they 
are not applied within 5 cm of a treatment-targeted tumor). 
d. Known inhibitors of the HH signaling pathway (e.g., vismodegib, 
patidegib, sonidegib, and itraconazole) topically or systemically within 
6 months of entry into the study. 
3. The subject has a history of hypersensitivity to any of the ingredients in the study 
medication formulation. 
4. The subject is unable or unwilling to make a good faith effort to be present for all 
follow-up visits and tests. 
5. The subject is a woman who is currently nursing. 
6. The subject has any systemic disease that in the Investigator’s opinion would interfere 
with the subject’s ability to participate. 
7. The subject has a clinically significant history of liver disease, including viral hepatitis, 
current alcohol abuse, or cirrhosis that in the investigator’s opinion would interfere 
with the subject’s ability to participate. 
8. The subject has any condition or situation, which in the Investigator’s opinion may put 
the subject at significant risk, could confound the study results, or could interfere 
significantly with the subject’s participation in the study.  This includes history of other 


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skin conditions or diseases, metabolic dysfunction, physical examination findings, or 
clinical laboratory findings giving reasonable suspicion of a disease or condition that 
contraindicates use of this investigational drug or that might affect interpretation of the 
results of the study or render the subject at high risk from treatment complications. 
9. The subject has a history of invasive cancer within the past five years excluding non-
melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of breast, or 
chronic lymphocytic lymphoma (CLL) (Stage 0). 
10. The subject is currently participating in an experimental drug study, (within 4 weeks 
of Baseline visit), or plans to participate in an experimental drug study while enrolled 
in this study. 
11. The subject is on a concomitant medication that is a strong CYP3A4 inhibitor.  These 
include, but are not limited to: larithromycin, telithromycin, nefazodone, itraconazole, 
ketoconazole, atazanavir, darunavir, indinavir, lopinavir, nelfinavir, ritonavir, 
saquinavir, and tipranavir. 
 


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Investigational Product, Reference Therapy, Dosage and Mode of Administration: 
 
• patidegib gel 2%, applied topically, once daily for 12 weeks 
• patidegib gel 4%, applied topically, once daily for 12 weeks 
• patidegib gel 2%, applied topically, twice daily for 12 weeks 
• patidegib gel 4%, applied topically, twice daily for 12 weeks 
• vehicle gel, applied topically, once daily for 12 weeks 
• vehicle gel, applied topically, twice daily for 12 weeks 
Application Instructions: 
 
The Investigator or their designee will instruct the subject on how to apply the study drug to 
the previously biopsied treatment-targeted BCCs identified at the Baseline visit by the 
Investigator. The staff member will instruct the subject on the proper application procedure 
during the Baseline visit.  The quantity of gel to be applied will be defined using laminated 
dosing cards.  Approximately 20 mg of gel will be applied to each tumor at each treatment. 
The subject is to treat no more than two treatment-targeted BCCs. If the subject has 
additional BCCs they may be treated surgically or topically during the trial. In addition to 
the verbal instructions given during the visit, the subjects will be provided with written 
instructions for proper application technique. 
Because sunlight can increase the development of skin cancers subjects will be advised to 
avoid or minimize exposure to direct sunlight while in the study. Subjects will also be 
advised to wash their hands before and after application of the study drug. 
The amount of study drug used by the subjects will be guided by instructing the subjects to 
use dosing cards that will provided to them, and monitored by weighing each newly 
dispensed study drug tube, and re-weighing each returned study drug tube. 
Duration of Treatment: 
All subjects will be treated for 12 weeks. 
Criteria for Evaluation: 
The study will be conducted as outlined in the Schedule of Assessments (Table 1). For all 
efficacy measurements, the Investigators will be provided with study specific training to 
ensure consistent evaluations. The assessments for a particular subject should be performed 
by the same investigator at all study visits whenever possible. 
 


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Safety Measurements: 
Dermal Safety and Tolerability: Safety and tolerability will be evaluated through assessment 
of selected local signs and symptoms (pain / burning, pruritus, erythema, edema, and 
scabbing / crusting). Each of the identified BCCs will be evaluated separately for these signs 
and symptoms of application site reactions. Any local skin reaction that requires use of a 
concomitant therapy or causes study drug interruption or discontinuation should be reported 
as an AE. The scales to be used for assessing local skin reactions at treatment sites follow: 
Score  Grade  
Description 
Pain/Burning: as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baseline or since the last 
visit at subsequent visits. 
0 
None 
No pain/burning 
1 
Mild 
Slight burning/stinging sensation; not really bothersome 
2 
Moderate Definite warm, burning/stinging that is somewhat bothersome 
3 
Severe 
Hot burning/stinging sensation that causes definite discomfort and 
may interrupt daily activities and/or sleep 
Pruritus: as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baseline or since the last 
visit at subsequent visits. 
0 
None 
No pruritus 
1 
Mild 
Slight pruritus, not really bothersome 
2 
Moderate Definite pruritus that is somewhat bothersome 
3 
Severe 
Intense pruritus that may interrupt daily activities and/or sleep 
Erythema: as assessed by the Investigator at each site 
0 
None 
No erythema present 
1 
Mild 
Slight pink coloration 
2 
Moderate Definite redness 
3 
Severe 
Marked erythema, bright red to dusky dark red in color 
Score  Grade  
Description 
Edema: as assessed by the Investigator at each site 
0 
None 
No edema 


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1 
Mild 
Slight, but definite edema 
2 
Moderate Definite edema 
3 
Severe 
Marked edema 
Scabbing/Crusting: as assessed by the Investigator at each site 
 
0 
None 
No scabbing/crusting 
1 
Mild 
Slight, but definite scabbing/crusting 
2 
Moderate Definite scabbing/crusting 
3 
Severe 
Marked scabbing/crusting 
Symptoms of Hedgehog Inhibitor Toxicity: At each visit subjects will be asked if they have 
experienced any symptoms that have been associated with systemic use of this class of 
drugs, in particular hair loss, taste loss, muscle cramps, bowel changes, fatigue.  
Adverse Events: During the study, subjects will be assessed for the occurrence of new and 
ongoing AEs. Descriptions of AEs will include the dates of onset and resolution (if 
resolved), maximum severity, and seriousness, action taken regarding the study drug, 
corrective treatment, outcome, and the Investigator’s assessment of causality. AEs present at 
any visit will be followed to resolution (return to normal or to the baseline state) or until 
clinically stable as determined by the Investigator. 
Safety Laboratory Tests: Routine safety laboratory tests (complete blood count/differential 
urinalysis and serum chemistry) will be performed at Screening, Week 6, and Week 12. Any 
out-of-range laboratory result that is considered clinically significant by the Investigator will 
be recorded as an AE and should be confirmed by repeat testing at the discretion of the 
Investigator. Clinically significant laboratory abnormalities at any visit will be followed to 
resolution (return to normal or to the baseline state) or until clinically stable as determined 
by the Investigator. 
Physical Examinations: An abbreviated physical examination including measurements of 
height, weight, and vital signs (blood pressure, heart rate, respiration rate, and oral 
temperature) will be performed at Baseline and Week 12. 
Pregnancy Tests: All female subjects will have a pregnancy test performed at the Screening 
visit. Females of childbearing potential will take a home pregnancy urine test at Weeks 2, 8, 
and 10 and will have a serum pregnancy test at Weeks 6 and 12. 
Efficacy Measurements: 
Primary: 
Biomarker: At screening the treatment-targeted BCC will be biopsied to confirm the clinical 
diagnosis of nodular BCC.  In addition, at or within 14 days of the Week 12 visit the 


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treatment site will be excised. Both of these tissue samples will be tested for GLI1 mRNA 
levels.  
Secondary: 
The clinical efficacy of patidegib as defined by the percent decrease from Baseline of the 
longest diameter of treatment-targeted BCC after 12 weeks of treatment.   
Exploratory: 
• To evaluate the utility of an investigator static global tumor assessment (ISGTA) in 
assessing the proportion of baseline treatment-targeted BCCs that are evaluated as 
being clear or almost clear. 
• Histologic Cure: The proportion of the excisional samples obtained at or shortly after 
the Week 12 visit that demonstrate no residual BCC. 
Statistical Methods: 
All subjects who are randomized and dispensed study drug will be included in the intent-to-
treat (ITT) analysis set. All subjects who are randomized, receive at least one confirmed 
dose of study drug, and have at least one post-baseline safety assessment will be included in 
the safety population. 
No imputation will be made for the primary endpoint.  For the secondary endpoint, last 
observation carried forward (LOCF) will be used to impute efficacy data that are missing 
post-baseline through Week 12. No imputations will be made for missing safety data. 
Per protocol analysis will be performed – we will exclude samples with insufficient BCC, 
subjects with poor treatment compliance, or tumors where the diameter was incorrectly 
measured based on review of photographs. We will also analyze BCC size change based on 
photographs.  
Efficacy Summaries: 
The efficacy endpoints are intended to compare once and twice daily application of 2% 
patidegib gel, 4% patidegib gel, and vehicle gel. Efficacy assessments will be summarized 
descriptively by treatment group and visit.  
Primary Efficacy Endpoints:  
• 
Change in GLI1 mRNA levels in drug-treated vs. vehicle-treated tumors after 12 
weeks of treatment.   
Secondary Efficacy Endpoint: 


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• 
Decrease in tumor size defined as percent decrease in the greatest diameter of 
baseline treatment-targeted BCCs at the Week 12. 
Exploratory Efficacy Endpoint: 
• 
The proportion of baseline treated tumors are smaller based on photographic review 
• 
The proportion of baseline treatment targeted BCCs that are evaluated as being clear 
or almost clear at Baseline, Weeks 2, 6, 8, 10, and 12 based on the ISGTA.  
Efficacy Analyses: 
The primary endpoint and the secondary endpoints of change in tumor size, and reduction in 
the HH signaling pathway will be evaluated with an analysis of covariance (ANCOVA) with  
treatment group as factor and baseline value as a covariate. Pairwise comparisons will be 
performed using contrasts within the ANCOVA. Additionally, t-tests for pairwise 
comparisons of the primary endpoint will be done, or alternatively a non-parametric approach 
may be used. 
The exploratory analysis of ISGTA will be analyzed with a Cochran-Mantel-Haenszel test  
Safety: 
Safety Endpoints: 
• 
Adverse Events 
• 
Dermal Safety and Tolerability including pain/burning, pruritus, erythema, edema, 
and scabbing/crusting 
• 
Clinical laboratory assessments 
Safety Analyses: 
Subjects will be assessed for the occurrence of new and ongoing AEs. Descriptions of AEs 
will include the dates of onset and resolution (if resolved), maximum severity, seriousness, 
action taken regarding the study drug, corrective treatment, outcome, and Investigator’s 
assessment of causality. All AEs will be recorded and classified using terminology from the 
Medical Dictionary for Regulatory Activities (MedDRA). All reported treatment-emergent 
AEs (TEAEs), defined as any AE with an onset on or after the date of first study drug 
application, will be summarized by treatment group, the number of subjects reporting 
TEAEs, system organ class, preferred term, severity, and relationship to study drug. When 
summarizing TEAEs by severity or relationship to study drug, each subject will be counted 
only once within a system organ class or a preferred term using the event with the greatest 
severity or causality, respectively, within each category. All reported serious adverse events 


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(SAEs) will be summarized by treatment group, the number of subjects reporting SAEs, 
system organ class, preferred term, severity, and relationship to study drug. 
All information pertaining to AEs noted during the study will be listed by subject and will 
include a verbatim description of the event as reported by the Investigator, as well as the 
preferred term, system organ class, start date, stop date (if stopped), seriousness, severity, 
action taken regarding the study drug, corrective treatment, outcome and relationship to the 
study drug. In addition, a listing of subjects who prematurely discontinue from the study due 
to AEs will be provided as well as a listing of subjects who reported an SAE. 
The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, 
erythema, edema, and scabbing/crusting will be summarized by treatment group and visit.  
Changes from baseline in safety laboratory values and vital sign measurements will be 
summarized with descriptive statistics for each treatment group at all applicable study visits. 
Shift tables will be presented for changes in safety laboratory values to summarize 
laboratory test results collected at Screening and Weeks 6 and 12. Normal ranges established 
by the central laboratory will be used to determine the shifts. A listing of all out-of-range 
laboratory test results at any assessment time point will also be provided. Determination of 
clinical significance for all out-of-range laboratory values will be made by each Investigator 
and included in the listing. In addition, a listing of all clinically significant laboratory test 
results will be provided. 
Sample Size Calculations:  
This is a dose ranging study. 
 


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2. TABLE OF CONTENTS 
1. 
SYNOPSIS ...............................................................................................................4 
2. 
TABLE OF CONTENTS .......................................................................................17 
3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ........................20 
4. 
INTRODUCTION .................................................................................................22 
5. 
STUDY OBJECTIVES ..........................................................................................27 
5.1 
Overall Study Design and Plan ..................................................................27 
6. 
SELECTION AND WITHDRAWAL OF SUBJECTS .........................................32 
6.1 
Subject Inclusion Criteria ..........................................................................32 
6.2 
Subject Exclusion Criteria .........................................................................33 
6.3 
Subject Withdrawal Criteria ......................................................................35 
6.4 
Study Drug Discontinuation ......................................................................36 
6.5 
Discontinuation of the Study .....................................................................36 
6.6 
Stopping Criteria ........................................................................................37 
7. 
TREATMENT PLAN ............................................................................................37 
7.1 
Methods of Assigning Subjects to Treatment Groups ...............................37 
7.2 
Randomization and Blinding .....................................................................37 
7.3 
Unblinding .................................................................................................37 
7.4 
Prior and Prohibited Concomitant Medication or Therapy........................38 
7.5 
Treatment Compliance ...............................................................................39 
7.6 
Protocol Deviations and Violations ...........................................................39 
8. 
STUDY DRUG MATERIALS AND MANAGEMENT ......................................41 
8.1 
Identity of the Study Drug .........................................................................42 
8.2 
Packaging and Dispensation of Study Drug ..............................................42 
8.3 
Storage, Handling, and Disposal of Study Drug ........................................42 
8.4 
Application .................................................................................................42 
8.5 
Study Drug Accountability ........................................................................43 
9. 
STUDY PROCEDURES AND EVALUATIONS ................................................44 
9.1 
Schedule of Evaluations and Procedures ...................................................44 
9.1.1 Screening Visit (Day -42 to Day 0) ...............................................44 
9.1.2 Baseline Visit (Day 1) ....................................................................45 
9.1.3 Weeks 2, 6, 8, and 10 .....................................................................46 
9.1.4 Week 12 (Day 85 ± 3 Days) / Early Termination ..........................47 
9.1.5 Week 14 (Day 99 ± 7 Days) / Post Operation Follow-up ..............48 
9.2 
Evaluation of Efficacy ...............................................................................48 
9.2.1 Assessment of BCCs ......................................................................49 


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9.2.2 Messenger RNA (mRNA)..............................................................49 
9.2.3 Investigator Static Global Tumor Assessment ...............................49 
9.3 
Evaluation of Safety ...................................................................................49 
9.3.1 Dermal Safety and Tolerability ......................................................49 
9.3.2 Medical History and Abbreviated Physical Examination ..............51 
9.3.3 Safety Laboratory Tests .................................................................51 
9.3.4 Pregnancy Tests .............................................................................51 
9.3.5 Adverse Events ..............................................................................51 
9.3.5.1 
Definition of Adverse Event .........................................51 
9.3.5.2 
Documenting Adverse Events .......................................52 
9.3.5.3 
Serious Adverse Events.................................................53 
9.3.5.4 
Assessment of Severity .................................................53 
9.3.5.5 
Assessment of Causality ...............................................54 
9.3.5.6 
Reporting of Serious Adverse Events ...........................54 
9.3.5.7 
Emergency Contact .......................................................55 
9.3.5.8 
Expedited Serious Adverse Event Reports ...................56 
9.3.5.9 
Other Required Safety Assessments .............................56 
10. 
STATISTICS .........................................................................................................58 
10.1 
Analysis Populations ..................................................................................58 
10.2 
Subject Disposition ....................................................................................58 
10.3 
Demographics and Baseline Characteristics ..............................................58 
10.4 
Protocol Deviations ....................................................................................59 
10.5 
Compliance ................................................................................................59 
10.6 
Interim Analyses ........................................................................................59 
10.7 
Assessment of Efficacy ..............................................................................59 
10.7.1 Efficacy Summaries .......................................................................59 
10.7.1.1 Primary Efficacy Endpoint: ..........................................59 
10.7.1.2 Secondary Efficacy Endpoints: .....................................59 
10.7.1.3 Exploratory Efficacy Endpoints: ...................................59 
10.7.2 Efficacy Analyses ..........................................................................60 
10.8 
Assessment of Safety .................................................................................60 
10.8.1 Dermal Safety and Tolerability ......................................................60 
10.8.2 Adverse Events ..............................................................................60 
10.8.3 Safety Laboratory Values and Vital Sign Measurements ..............61 
10.8.4 Pregnancy Tests .............................................................................61 
10.8.5 Handling of Missing Data ..............................................................61 
10.8.6 Multicenter Issues ..........................................................................61 
10.8.7 Multiplicity Issues ..........................................................................61 


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10.9 
Sample Size Determination........................................................................61 
11. 
QUALITY CONTROL AND QUALITY ASSURANCE .....................................62 
11.1 
Study Monitoring .......................................................................................62 
11.2 
Audits and Inspections ...............................................................................62 
11.3 
Data Quality Assurance .............................................................................63 
12. 
ETHICS AND ADMINISTRATIVE ISSUES ......................................................64 
12.1 
Ethical Conduct of the Study .....................................................................64 
12.2 
Independent Review Board Review...........................................................64 
12.3 
Informed Consent.......................................................................................64 
12.4 
Subject Data Protection..............................................................................64 
12.5 
Financial Disclosure...................................................................................65 
12.6 
Investigator Obligations .............................................................................65 
12.7 
Changes to the Protocol .............................................................................65 
12.8 
Confidentiality/Publication of the Study ...................................................65 
13. 
DATA HANDLING AND RECORD KEEPING .................................................66 
13.1 
Inspection of Records ................................................................................66 
13.2 
Retention of Records..................................................................................67 
13.3 
Electronic Case Report Form Completion .................................................67 
14. 
REFERENCES ......................................................................................................68 
15. 
APPENDICES .......................................................................................................69 
15.1 
Body Diagram ............................................................................................69 
15.2 
Safety Laboratory Tests .............................................................................71 
 
 
List of Tables 
Table 1:  Schedule of Assessments ....................................................................................30 
Table 2:  Excipients of Patidegib Topical Gel ...................................................................41 
Table 3:  Specific Compositions of the Vehicle, 2% and 4% Patidegib Gels ...................41 
Table 4:  Investigator Static Global Tumor Assessment ...................................................49 
Table 5:  Dermal Safety and Tolerability Scales ...............................................................50 
 


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3. LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
              Abbreviation or    
              Specialist 
Term 
               Definition or Explanation 
AE 
ALT 
API 
AST 
Adverse Event 
Alanine Aminotransferase 
Active Pharmaceutical Ingredient 
Aspartate Aminotransferase 
ANCOVA 
Analysis of Covariance 
BCC 
Basal Cell Carcinoma 
BCNS 
BUN 
Basal Cell Nevus Syndrome 
Blood Urea Nitrogen 
CBC/Diff 
Complete Blood Count with Differential 
CLL 
Chronic Lymphocytic Lymphoma 
eCRF 
Electronic Case Report Form  
ET 
FDA 
FSH 
Early Termination 
Food and Drug Administration 
Follicle-Stimulating Hormone 
GCP 
Good Clinical Practice 
GLI 1 
Glioma-associated oncogene homolog 1 
GMP 
GRAS 
Good Manufacturing Practices 
Generally Recognized as Safe 
HCl 
Hydrogen Chloride 
HH 
Hedgehog 
ICF 
Informed Consent Form 
ICH 
International Conference on Harmonization 
IEC 
IND 
IRB 
Independent Ethics Committee 
Investigational New Drug  
Institutional Review Board  
ISGTA 
Investigator Static Global Tumor Assessment 
ITT 
Intent-to-Treat  
LOCF 
Last Observation Carried Forward 
MedDRA 
MCH 
MCHC 
MCV 
Medical Dictionary for Regulatory Affairs 
Mean Corpuscular Hemoglobin 
Mean Corpuscular Hemoglobin Concentration 
Mean Corpuscular Volume 
mRNA 
Messenger Ribonucleic Acid  
PDT 
PTCH1 
PSCP 
Photodynamic Therapy 
Patched Protein 1 
Primary Skin Care Physician 
RBC 
Red Blood Cell 
SAE 
Serious Adverse Event 


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              Abbreviation or    
              Specialist 
Term 
               Definition or Explanation 
SAP 
Statistical Analysis Plan 
SAS 
SEB 
Statistical Analysis System 
Surgically Eligible Basal Cell Carcinoma 
SMO 
SPF 
Smoothened 
Sunscreen Protection Factor 
SAP 
Statistical Analysis Plan 
TEAE 
Treatment-Emergent Adverse Event  
US 
United States 
UV 
WBC 
Ultraviolet 
White Blood Cell Count 
 


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4. INTRODUCTION 
PellePharm, Inc. is developing patidegib gel for the management of the burden of disease in 
patients with basal cell nevus syndrome (BCNS), a rare genetic disease characterized by the 
development of numerous basal cell carcinomas over a lifetime. This trial will evaluate the ability 
of patidegib gel to shrink BCC tumors in patients with sporadic BCCs as a proof-of-concept for 
the mechanism of patidegib on BCCs.  It is hoped that results from this study will inform the dose 
and dosing regimen prior for future trials, thereby maximizing the therapeutic potential. 
Basal cell carcinomas: These tumors are the most common of human cancers, occurring in an 
estimated 2-3 million Americans each year. The overwhelming majority of these tumors occur 
sporadically with a strong predilection for sun exposed skin sites in persons of Northern Europe 
descent who have had excessive sun exposure.  Sporadic BCCs are most commonly seen starting 
in the 4th decade of life. Current treatment depends in large part on physical destruction of the 
tumors, most commonly with surgery and less commonly with ionizing radiation.  
Basal cell nevus syndrome: BCCs are one of the most prominent phenotypic features of patients 
with the BCNS (Gorlin) syndrome (OMIM #109400]1. This syndrome is inherited as an 
autosomal dominant condition, and patients are susceptible to many abnormalities, including, 
most frequently, palmar and plantar pits and jaw cysts, and medulloblastomas.  Reports of its 
prevalence vary; the highest estimate is 1:31,000.  In one study of patients with heritable cancer 
syndromes studies, those with BCNS had the longest expected lifespan of 73 years2, which is not 
unexpected for a cancer that grows locally and metastasizes only very rarely.  Diagnosis of 
BCNS commonly is made when patients are in their teens3. The burden of BCCs varies among 
BCNS patients. For example, in a Children’s Hospital Oakland and Stanford combined registry 
of 100 BCNS patients living throughout the United States, 1/3 had a total of 5 or fewer BCCs in 
the previous two years, 1/3 had 6-20 BCCs treated, and 1/3 had more than 20 BCC in the past 2 
years3. 
Aberrant Hedgehog signaling in BCCs: In 1996, two groups identified the PTCH1 gene as the 
locus of the mutations that cause BCNS4-6. Patients with BCNS have one defective heritable copy 
of this gene, and sporadic loss of the second allele is crucial to the development of all of their 
BCCs.  Despite this identification, diagnosis of BCNS still relies primarily on clinical findings7 
because a significant percentage of patients with clinically typical BCNS have PTCH1 mutations 
that are not detectable with current sequencing approaches. The function of the PTCH1 gene is 
well known from studies conducted over three decades - it encodes the primary inhibitor of the 
hedgehog (HH) signaling pathway and its function is to inhibit signaling by the next “downstream” 
member of the HH pathway - SMOOTHENED (SMO).  Approximately 90% of sporadic BCCs 
have lost function of PTCH1, and approximately 10% of sporadic BCCs have an activating 
mutation in SMO that renders the protein resistant to inhibition by PTCH1 protein8.  By 
comparison, all BCNS BCCs have loss of PTCH1 without activating mutations of SMO9. Indeed 


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all BCCs, whether arising in BCNS patients or sporadically, have up-regulated output of the HH 
pathway as their fundamental molecular defect.  This signaling pathway is crucial to development 
of many organs during embryogenesis but in adult life functions in only a limited number of sites, 
among which is the hair follicle. This is consistent with the finding that in some mouse models 
BCCs arise from the hair follicles.  The importance of aberrant HH signaling as the driver of BCCs 
was proved indisputably by the strong anti-tumor effect of small molecules that down-regulate this 
pathway [vide infra]. 
Growth of BCCs: As expected, given the common underlying molecular underpinning of all BCCs, 
the biology of sporadic BCCs and BCCs in BCNS patients is very similar and is characterized by 
slow growth and very low frequency of metastasis. Thus Kirkup and De Berker found a median 
increase of 0.5 mm in the longest diameter of sporadic BCCs untreated for a mean of 70 days10.  
Similarly, Tan et al found an increase in longest diameter of 0.75 mm per month in periocular 
BCCs11.  Growth specifically of BCCs in BCNS patients also is slow, with minimal enlargement 
in subjects randomized to the placebo arm of a double-blinded study of the efficacy of 
vismodegib12. 
BCNS is a chronic disease: In one recent report of a cohort of BCNS patients, the average time 
since diagnosis was 28 years13 and in a combined registry of 100 patients at Children’s Hospital 
Oakland and Stanford, the median age of diagnosis was 15 years3, the median survival in BCNS 
patients being reported as 73 years2. Fewer than 10% of BCNS patients report having locally 
advanced BCCs – the majority had tumors that were surgically operable. 
In general, BCNS patients have their BCCs treated as they become problematic, i.e. at risk of 
invasion of vital structures such as eyes, nose, or ears or large enough off the face such that they 
are uncomfortable, bleed, etc.  Thus BCNS patients typically never are free of BCCs - in a trial of 
the effects of vismodegib vs. BCCs in BCNS patients (vide infra), BCNS subjects had an average 
of 27 BCCs present at baseline, and although oral vismodegib can produce complete clinical 
clearing, once the drug is stopped the BCCs recur12. 
Pharmacologic treatment of BCCs:  Several topically-applied drugs are used in the treatment of 
BCCs, such as imiquimod and 5-fluoruracil.  Both of these can cure approximately 80% of the 
superficial subtype of BCCs most of which generally occur off the face, but generally are not 
useful vs. nodular BCCs, which are the more prevalent subtype, especially on the face.  Prevention 
of sporadic BCCs so far has been limited to admonitions to avoid sunlight, advice which is 
followed infrequently. With identification of uncontrolled HH signaling as the driving molecular 
abnormality in all BCCs, several anti-HH drugs have been developed for oral treatment of BCCs, 
and two of these - vismodegib (Genentech/Roche) and sonidegib (Novartis) - have been approved 
for systemic treatment of advanced BCCs. The latter are defined as BCCs whose surgical excision 
likely would produce unsatisfactory results (i.e. “locally advanced”) or those which have become 
metastatic14.  Approximately 50% of such BCCs fail to respond initially, frequently due to 


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mutations in the SMOOTHENED gene, which encodes the protein to which these drugs bind. Of 
those that do respond, a significant proportion develop secondary resistance, often due to mutations 
in the drug binding pocket of the SMO protein15.  Vismodegib also has been studied for efficacy 
vs. BCCs in patients with BCNS12.   Unlike results in advanced BCCs, all Gorlin non-advanced 
tumors respond by shrinking, eventually most disappearing completely both clinically and 
histologically. This is consistent with the finding that all non-advanced BCCs in BCNS patients 
lack SMO mutations. In addition, the BCCs in BCNS patients fail to develop resistance and, so 
long as BCNS patients continue to take the drug, no new surgically-eligible BCCs (SEB) 
developed.  The combined result of shrinkage of existing BCCs and blockage of the development 
of new BCCs is that these patients have no need for surgery so long as they continue to ingest 
vismodegib.  But because of annoying class-specific side effects most patients discontinue 
vismodegib, and all the clinically and histologically cleared BCCs recur to the same size as before 
treatment12.  Of note, the rate of development of new SEBs was lower after vismodegib was 
discontinued (0.7 new SEBs/month) than it had been during initial placebo treatment (2.4 new 
SEBs/month), suggesting that vismodegib may have a more robust, long-lasting effect on smaller 
BCCs than on SEBs and hence may be more effective at inhibiting progression by blocking the 
growth of new SEBs than by shrinking existing SEBs12. Small as well as large but operable 
sporadic BCCs respond to vismodegib unless they are among the approximately 10% of sporadic 
BCCs that are driven by SMO mutations rather than by loss of PTCH1 mutations16. 
Patidegib is a semi-synthetic small molecule, and the topical drug product is manufactured with 
generally accepted, safe excipients. Oral patidegib has a good therapeutic efficacy versus locally 
advanced and metastatic BCCs but produces the same types of adverse effects as do other systemic 
HH inhibitors. Topical patidegib has been shown to be stable in the developed gel formulation and 
can be applied to mini-pig skin without irritation.  Topical application of patidegib significantly 
reduces murine BCC tumor size in vivo and reduces GLI1 biomarker expression in vitro in human 
BCC tumor explants. 
If proven to be safe and effective in future clinical trials patidegib gel may offer BCNS patients a 
safe and effective therapy to manage their burden of disease by decreasing the number of surgeries 
they will require over their lifetime.  This trial will evaluate the safety, the tolerability, and the 
ability of four different dosing regimens applied for 3 months to shrink nodular BCC tumors in 
patients with sporadic BCCs. 
The goal of the present trial is to evaluate topical patidegib’s safety, tolerability, and effects on the 
size of preexisting sporadic previously untreated nodular BCCs and thereby to establish a dose and 
dosing regimen for future trials in the BCNS orphan population. It is anticipated that subsequent 
trials will focus on reducing the number of facial surgeries required by patients with BCNS and 
not on the “treatment” of individual sporadic BCCs. If the efficacy with topical patidegib can 
approach the level of efficacy of oral HH inhibitors seen in BCNS patients while avoiding their 
systemic side effects, it would represent a major advance for BCNS patients.  


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Rationale for using telemedicine-based and photographic assessments of BCC in clinical 
trials: For medical science to progress, technology must advance to bridge the gap between 
research and the real-world patient application of medical discovery. For example, researchers 
are beginning to have a better understanding of the complexity of the genetic and environmental 
factors that contribute to the development of skin cancer.  But to decipher the complexity of this 
relationship across growing and diverse populations, new technology and methodology is needed 
to connect with patients. Keeping pace with the advent of electronic medical records, social 
networking, and smartphones, new applications of technology will allow patients to interact with 
physicians and researchers more regularly and directly from home in a way that will lead to 
better treatment and understanding of the diseases that afflict us. These technologies will also 
unlock access to a more diverse population of patients and also to allow for more real time 
evaluation of adverse events in these patients. 
 
The concept of using mobile smartphone technology in medicine and research is not new. Even 
with regard to skin cancer, research instruments such as telemedicine, mobile electronic devices, 
and the internet have been used. Specifically, this technology has been used to assist with 
compliance, medical education, physician-patient interactions, medical access, and evaluation of 
treatment efficacy in many areas including dermatology.  Many FDA guidelines for registered 
trials in skin disease strongly suggest that a digital photographic record of all patient visits be 
made available as the gold standard for FDA audit of clinical outcomes. 
Access issues continue to limit biomedical research and healthcare. Access limits connections 
between patients and physicians and between researchers and needed subjects. As modern 
medical practice shifts toward patient-centered care, a push toward greater patient engagement 
must also evolve. A better partnership between patients and doctors to improve understanding 
and management of disease will lead to better healthcare. As an example, patients will be able to 
enter their own data into secure electronic health records (EHR). Today, some patients enjoy 
access to their EHR through providers such as Kaiser Permanente and are allowed to procure 
certain samples for lab analysis (e.g. fecal occult blood test) in their home. This very same 
principle of patient empowered medical evaluation can be applied to research and made easy for 
patients to access. Note that physician guidance is still the foundation of medical and 
investigational practice. However, application of patient involvement can be as simple as using a 
mobile device to securely update reliable patient reported outcomes (PROs) within clinical trials. 
Additionally, less than 5% of clinical research participants nationally are minorities.  The 
decentralized clinical trial model will unlock access to minorities and underserved populations 
that are not normally cared for at traditional clinical research sites and thus better reflect the 
national diversity of race and ethnicity in the United States.   
New technology exists to bridge access between clinical research and the need for patient-
centered care. One such application of technology is NORA (Network Oriented Research 
Assistant). NORA is currently being used successfully in other FDA-registered decentralized 


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clinical trials to assess rare skin and mucosal disease in the home.  NORA is a combination 
technology that includes the functionality of a telemedicine platform, an EMR, an EDC, an 
eConsent and a mobile data collection tool.  NORA was recently used to compare digital 
photography as a correlate of face-to-face acne and blister scoring but has been used in other 
therapeutic areas as well. This approach will hopefully improve patient outcomes and advance 
the field of medicine by using more real world data and a better diversity of patients. 
 
 
 


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5. STUDY OBJECTIVES  
The primary objectives of the study are to evaluate the following: 
 
1. The safety and tolerability of treatment with patidegib gel 2% or 4% or vehicle applied 
once or twice daily for 12 weeks. 
2. The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) 
signaling pathway after treatment with patidegib gel 2% or 4% or vehicle applied twice 
daily for 12 weeks to treatment-targeted BCCs. 
The secondary objectives of the study are to evaluate the following: 
 
1. The clinical efficacy of patidegib as defined by the percent decrease in greatest diameter 
of baseline treatment targeted basal cell carcinomas after 12 weeks of treatment. Treatment 
targeted BCCs will be biopsied prior to treatment to confirm the clinical diagnosis of 
nodular BCC. Eligible tumors will have the clinical features of nodular BCC and prior to 
biopsy will be no less than 5 mm or greater than 20 mm in longest diameter on the face 
(excluding the nose and periorbital skin) and no less than 9 mm or greater than 20 mm in 
longest diameter at sites other than the face. 
The exploratory objectives of the study are to evaluate the following 
 
1. To evaluate the utility of an investigator static global tumor assessment (ISGTA) in 
assessing the proportion of baseline treatment-targeted BCCs that are evaluated as being 
clear or almost clear. 
 
2. To evaluate the utility of blinded assessment of change in BCC size using photographs 
5.1 
Overall Study Design and Plan 
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to assess 
the efficacy and safety of patidegib gel 2% and 4% in comparison with vehicle applied once or 
twice daily. The population of subjects will be enrolled from the 
. 
Approximately 36 subjects who meet the study entry criteria will be randomized to one of four 
sequential cohorts. Each cohort will have 6 subjects randomized to active drug and 3 randomized 
to vehicle. The four sequential cohorts will be patidegib gel 2% or vehicle applied once daily, 
patidegib gel 4% or vehicle applied once daily, patidegib gel 2% or vehicle applied twice daily, 
and patidegib gel 4% or vehicle applied twice daily. As soon as one cohort has been enrolled the 
next cohort will be recruited. The study drug will be applied topically to the treatment-targeted 
PPD


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nodular BCCs once or twice daily for 12 weeks of treatment. Subjects will apply their treatments 
at home as explained by the Investigator or their designee. The subject will apply the initial 
application under the supervision of the study staff. If a subject has a treatment targeted BCC on 
an anatomical location that the subject cannot reach such as the back, a friend or family member 
may apply the treatment. In this case the friend or family member applying the gel,  a woman of 
childbearing potential should follow the birth control requirements outlined in the Inclusion 
Criteria, and the person should apply the gel using a latex or vinyl glove. As skin cancer patients 
are routinely advised, all subjects will be instructed to avoid exposure to direct sunlight and to 
continue their use of sunscreens to minimize their exposure to ultraviolet (UV) radiation.  
It should be noted that 3 months of treatment with oral vismodegib caused a 50% reduction in the 
sum of the greatest diameters of BCCs in 50% of BCNS patients.  Therefore, even if topical 
patidegib were as effective as oral vismodegib in shrinking BCCs there likely will be enough 
residual BCC at Week 12 to allow for meaningful evaluation of biomarkers. 
Subjects will return their tubes of study drug, which will be evaluated for drug usage compliance. 
While individual usage rates of topical products will vary, the use of a dosing card should help 
limit the variation in dosing inherent with topical products. Approximately 20 mg of the gel 
represents a single applied dose. Thus a subject treating a single tumor once daily for 12 weeks 
will apply a total dose of 1.6 gm of gel and those treating two tumors twice daily for 12 weeks will 
apply a total dose of 6.4 gm of gel. In terms of actual drug, 20 mg of patidegib gel 2% and 4% 
contains 0.4 mg and 0.8 mg of patidegib, respectively. Thus the total dose of drug in the 2% once 
daily group treating a single tumor will be approximately 34 mg while subjects treating two tumors 
twice daily for 12 weeks with patidegib gel 4% will receive a total of 134 mg of patidegib. To put 
this in perspective in previous trials with an oral formulation of patidegib subjects were treated 
with a daily oral dose of 130 – 160 mg, many for longer than 6 months. 
Upon completion of the 12-week treatment period, all subjects will be asked to return to the 
investigational center for final evaluation. At the Week 12 visit the treatment site(s) will be 
excised. The area excised should at a minimum include the original diameter of the tumor. This 
means that if the tumor has shrunk in size the entire original area of the BCC will be excised, not 
just the visually apparent residual tumor.  During the study, subjects will be allowed to use 
moisturizers and emollients and sunscreen. The Investigator will assess the areas affected at each 
visit.  
The treatment-targeted BCC(s) will be identified by the Investigator at the Baseline visit and will 
be circled in ink at Baseline, Weeks 6 and 12, and photographed, and measured at all study visits 
(Baseline, Weeks 2, 6, 8, 10, and 12).  
Biomarkers:  On each subject one or two baseline previously untreated nodular BCCs designated 
as a treatment-targeted tumor will be biopsied and at the end of 12 weeks of treatment the treatment 


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site(s) will be excised. Both samples will be evaluated histologically and for determination of GLI1 
mRNA levels.  
Blood samples for complete blood count and serum chemistry and urine for urinalysis will be 
collected from subjects at Screening, Week 6, and Week 12.  
Subjects who terminate study participation early will be asked to complete all Week 12 
assessments, as appropriate, prior to commencement of any alternative therapy for BCC (if 
possible). Subjects who discontinue from the study during the treatment period will not be 
replaced. 
If signs or symptoms develop in the treatment areas during the treatment period that restrict daily 
activities or make continued application of the study drug difficult due to discomfort, the 
Investigator may instruct the subject to interrupt use of the study drug temporarily and to resume 
application of the study drug once the signs/symptoms have subsided. The Investigator should try 
to minimize study drug interruptions; and if needed, make best efforts to limit a “drug holiday” to 
no more than 7 days. If the study drug interruption does exceed 4 consecutive days, the Investigator 
should consult with the Medical Monitor to determine a course of action. If the study drug is 
interrupted, discontinued, or a concomitant medication is used to treat a sign or symptom, an AE 
shall be recorded. 
Subjects who discontinue participation in the study due to clinically significant laboratory 
abnormalities or AEs will be asked to complete all Week 12 evaluations. Any subject who has an 
AE during the treatment period will be monitored by the Investigator until resolution (return to 
normal or to the baseline state) or stabilization, as determined by the Investigator. 
In addition, application of study drug may be delayed or halted at any time if ongoing safety data 
evaluations raise concern for subject safety. If the subject participation is suspended, all of the 
subject’s safety data will be reviewed by the Medical Monitor in conjunction with the Investigator 
to determine course of action. 
 


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Table 1:  Schedule of Assessments 
 
 
 
 
 
 


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6. SELECTION AND WITHDRAWAL OF SUBJECTS 
6.1 
Subject Inclusion Criteria 
Subjects meeting all of the following criteria will be eligible for study entry: 
1. 
The subject is from 18 to 85 years of age, inclusive. 
2. 
The subject must provide electronic informed consent prior to any study 
procedures.  
3. 
If the subject is a woman of childbearing potential2, she is willing to use two 
effective methods of birth control during the duration of the trial and for one month 
after the last application of the gel. The two forms of birth control authorized are 
defined as the use of a barrier method of contraception (condom with spermicide) 
in association with one of the following methods of birth control: bilateral tubal 
ligation; combined oral contraceptives (estrogens and progesterone) or implanted 
or injectable contraceptives with a stable dose for at least 1 month prior to Baseline; 
hormonal intra-uterine device (IUD) inserted at least 1 month prior to Baseline. 
This proscription is based on the key role of the HH pathway in embryogenesis, the 
known preclinical teratogenic effects of systemic cyclopamine, a naturally 
occurring inhibitor of SMO, and the unknown level of systemic exposure following 
topical application of patidegib in humans.  
 
4. 
If the subject is a male with a female sexual partner who is of childbearing potential, 
the couple is willing to use two effective methods of birth control during the 
duration of the trial and for one month after the last application of the gel. 
Authorized birth control methods are outlined in Inclusion Criterion #3. Any 
woman of childbearing potential applying the gel to themselves, or assisting a 
subject, must comply with the same birth control measures.  
5. 
One or two previously untreated BCCs with the clinical features of a nodular BCC 
confirmed by a biopsy done at or prior to screening confirming nodular BCC.  
These tumors must be suitable for surgical excision. The BCCs prior to biopsy must 
be no less than 5 mm or greater than 15 mm in greatest diameter on the face and no 
                                                 
2 For the purpose of this trial, a female not of childbearing potential is defined as a sexually mature woman 
who: 1) has undergone a hysterectomy or bilateral oophorectomy; or 2) has been naturally postmenopausal for 
at least 12 consecutive months (i.e., has not had menses at any time in the preceding 12 consecutive months); or 
3) is 41 years of age or older with physiologic symptoms of menopause and a Follicle Stimulating Hormone 
(FSH) level of ≥ 30 IU/L or 4) whose male sexual partner has had a vasectomy. 


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less than 9 mm or more than 20 mm in greatest diameter at sites other than the face. 
Tumors on the nose, periorbital skin, or on or below the knee are excluded. 
6. 
The subject is willing to abstain from application of non-study topical prescription 
and over the counter medications within 5 cm of a treatment-targeted BCC for the 
duration of the study except as prescribed by the Investigator.  Moisturizers and 
emollients are allowable.  Subjects will be encouraged to use sunscreen with a 
sunscreen protection factor (SPF 15 or higher) at least once daily on all exposed 
skin sites. 
7. 
Female subjects must have negative serum pregnancy test at Screening. 
8. 
The subject is willing to contact the study center after each primary skin care 
physician (PSCP) visit to provide the study center details of the visit and any 
treatment of skin tumors. 
9. 
The subject is willing to forego alternative treatment of the treatment-targeted 
baseline BCC for the duration of the trial.  
6.2 
Subject Exclusion Criteria 
Subjects meeting any of the following criteria will be excluded from the study: 
1. Subjects with basal cell nevus syndrome (BCNS, Gorlin syndrome, nevoid basal cell 
carcinoma syndrome; OMIM #109400). 
2. The subject has used topical products within 5 cm of a treatment- targeted BCC or 
systemic therapies that might interfere with the evaluation of the study medication 
during the study. Specifically, these include the use of: 
a. Topical glucocorticoids 30 days prior to screening 
b. Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) 
systemically or topically, or > 5% of an alphahydroxy acid (e.g., 
glycolic acid, lactic acid), photodynamic therapy (PDT), or 5-
fluorouracil or imiquimod (except as topical treatment to discrete 
BCCs) systemically or topically to the skin during the six months prior 
to entry. 
c. Systemic chemotherapy within one year prior to screening. (Note: field 
therapy with topically applied treatments can be done as long as they 
are not applied within 5 cm of a treatment-targeted tumor). 


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d. Known inhibitors of the HH signaling pathway (e.g., vismodegib, 
patidegib, sonidegib, and itraconazole) topically or systemically within 
6 months of entry into the study. 
3. The subject has a history of hypersensitivity to any of the ingredients in the study 
medication formulation. 
4. The subject is unable or unwilling to make a good faith effort to be present for all 
follow-up visits and tests. 
5. The subject is a woman who is currently nursing. 
6. The subject has any systemic disease that in the Investigator’s opinion would interfere 
with the subject’s ability to participate. 
7. The subject has a clinically significant history of liver disease, including viral hepatitis, 
current alcohol abuse, or cirrhosis that in the investigator’s opinion would interfere 
with the subject’s ability to participate. 
8. The subject has any condition or situation, which in the Investigator’s opinion may put 
the subject at significant risk, could confound the study results, or could interfere 
significantly with the subject’s participation in the study.  This includes history of other 
skin conditions or diseases, metabolic dysfunction, physical examination findings, or 
clinical laboratory findings giving reasonable suspicion of a disease or condition that 
contraindicates use of this investigational drug or that might affect interpretation of the 
results of the study or render the subject at high risk from treatment complications. 
9. The subject has a history of invasive cancer within the past five years excluding non-
melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of breast, or 
chronic lymphocytic lymphoma (CLL) (Stage 0). 
10. The subject is currently participating in an experimental drug study, (within 4 weeks of 
Baseline visit), or plans to participate in an experimental drug study while enrolled in 
this study. 
11. The subject is on a concomitant medication that is a strong CYP3A4 inhibitor.  These 
include, but are not limited to: larithromycin, telithromycin, nefazodone, itraconazole, 
ketoconazole, atazanavir, darunavir, indinavir, lopinavir, nelfinavir, ritonavir, 
saquinavir, and tipranavir. 
 


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6.3 
Subject Withdrawal Criteria 
Participation in the study is strictly voluntary. A subject has the right to withdraw from the 
study at any time for any reason and is under no obligation to disclose the reason. If a subject 
withdraws, the Investigator is to be informed immediately. 
The Investigator has the right to terminate participation of a subject at any time for any of the 
following: 
• Intake or use of non-permitted concomitant medication 
• Lack of subject compliance 
• Protocol violation - Contact PellePharm or designee before making decision. 
• Disease progression – The Investigator and/or PSCP believe that delaying the 
treatment of the treatment targeted tumor until the end of the trial would cause harm 
to the subject. 
• Worsening of any condition - Subject requires alternate treatment before the end of 
the study and the Investigator determines it is not due to lack of efficacy. 
• A perceived safety risk 
• Adverse Event – Complete AE form 
• Lost to Follow-Up – Document with at least 2 phone calls and a certified letter 
• Subject Request – Consent withdrawal, subject moved, schedule conflicts 
• Pregnancy – If subject or subject’s sexual partner become pregnant the subject will 
discontinue study drug immediately, but the pregnancy will be followed until 
delivery. Complete pregnancy form 
• Lack of Efficacy – Subject requires alternate treatment after at least 2 weeks of 
study drug treatment and the Investigator determines that the risk of continuing the 
subject in the study outweighs the benefit. However, if a subject has two treatment-
targeted BCCs and one of the BCCs requires treatment because of an increase in 
size, the tumor can be treated and the subject can remain in the trial. 
• Other – Specify in comments section of electronic case report form (eCRF) 
If a study subject experiences disease progression or begins another treatment for his/her 
disease, follow-up per protocol will no longer be required. 
Subjects who terminate treatment early will be asked to complete all Week 12 assessments 
prior to commencement of any alternative therapy (if possible). Subjects who discontinue from 
the study during the treatment period will not be replaced. 


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All subjects are free to withdraw from participating in this study at any time and for whatever 
reason, specified or unspecified, and without prejudice. No constraints will be placed on 
ordinary subject management, and subjects, when appropriate, will be placed on other 
conventional therapy upon request or whenever clinically necessary as determined by their 
physician. 
All premature discontinuations and their reasons must be carefully documented by the 
Investigator on the final eCRF, and, if need be, on the AE form.  
If, for any reason, a subject is discontinued during the treatment period prior to Week 12, all 
end of treatment study (i.e., Week 12) evaluations should be performed at the time of early 
termination and the reason for termination will be recorded in the end of study source 
documentation. All data gathered on the subject prior to termination will be made available to 
PellePharm. Subjects who discontinue during the post-treatment follow-up period should have 
the assessments performed and reported on the eCRFs for that corresponding visit. 
6.4 
Study Drug Discontinuation 
The Investigator may discontinue study drug administration for any subject at any time. The 
study drug administration must be discontinued for any of the following: 
• Occurrence of an exclusion criterion that is clinically relevant and affects the subject’s 
safety, if discontinuation is considered necessary by the Investigator. 
• Occurrence of AEs, if discontinuation of study drug is desired or considered necessary 
by the Investigator or subject. 
• Pregnancy 
• Disease progression -The Investigator and/or PSCP believe that delaying the treatment 
of the treatment targeted tumor until the end of the trial would cause harm to the subject. 
6.5 
Discontinuation of the Study 
Study discontinuation is at the discretion of PellePharm or the Investigator in any of, but not 
limited to, the following events: 
• Occurrence of unusual AEs in terms of their nature, severity, duration, or unexpected 
incidence. 
• Medical or ethical reasons affecting the continued performance of the study. 
• Difficulties in the recruitment of the subjects. 


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6.6 
Stopping Criteria 
Sponsor will monitor frequency of systemic signs and symptoms associated with this class of 
drug as well as any lab abnormalities. If clinically significant signs or symptoms of hedgehog 
inhibitors are detected, in particular hair loss, taste loss, muscle cramps, bowel changes, 
fatigue, as well as abnormal liver function tests, dosing will be stopped. 
7. TREATMENT PLAN 
7.1 
Methods of Assigning Subjects to Treatment Groups 
This is a double-blinded study, in which the identity of the study drug will be unknown to 
Investigator and subjects, as well as to all individuals closely associated with the study. 
Subjects will be enrolled into 1 of the 4 sequential study drug groups of 9 subjects with 6 being 
randomized to active and 3 to vehicle within each group. The sequential groups will be 
patidegib or vehicle gel 2% applied once daily, patidegib or vehicle gel 4% applied once daily, 
patidegib or vehicle gel 2% applied twice daily, patidegib or vehicle gel 4% applied twice 
daily). Each screened subject will be assigned a unique 5-digit study subject number assigned 
by the investigational center, which will consist of the 2 digit investigational center number 
and the 3 digit chronological screening order number, starting with 001 (e.g., 01001, 01002). 
The study drug kit will be assigned to subjects based on a randomization code, and kits will be 
dispensed to the subjects at Baseline in the order that they are enrolled by taking the lowest 
numbered kit available in inventory at the investigational site. A study drug accountability log 
will document the inventory and dispensing of study drug at the investigational center. 
7.2 
Randomization and Blinding 
The study drugs will be packaged and labeled identically, and the study drug kits will be 
numbered sequentially and dispensed randomly to the subjects on the study. Study drug 
supplies will be distributed to the investigational center to maintain the randomization ratio. 
As a double-blinded study, the Investigators, the site staff, PellePharm, and the Clinical 
Monitor(s) will not be aware of the treatment assigned to the individual study subjects. 
Delegated staff members at the investigational center will dispense the study drugs and will 
collect and weigh all used and unused study drug tubes as scheduled. 
7.3 
Unblinding 
The treatment assignments for all enrolled subjects will be unblinded on a cohort by cohort 
bases. The sponsor and CRO, but not site staff, will be unblinded in order to examine limited 
safety and efficacy results. 


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In the case of a medical emergency, the Investigator can break the blind for the subject involved 
only after making an effort to contact the Medical Monitor. After the code is broken the 
Investigator will contact the PellePharm representative for unblinding information. The 
Investigator will record the code break in the subject’s source documents. 
7.4 
Prior and Prohibited Concomitant Medication or Therapy 
Subjects must not have used topical products within 5 cm of a treatment targeted SEB or 
systemic therapies that could interfere with the evaluation of the study medication during the 
study. Specifically, these include use of the following: 
• Topical glucocorticoids 30 days prior to Screening 
• Retinoids (e.g., etretinate, isotretinoin, tazarotene, tretinoin, adapalene) systemically or 
topically, or > 5% of an Alphahydroxy acid (e.g., glycolic acid, lactic acid), 
photodynamic therapy (PDT), or 5-fluorouracil, or imiquimod (except as topical 
treatment to discrete BCCs) systemically or topically to the skin during the six months 
prior to entry.   
• Treatment with systemic chemotherapy within one year prior to screening. 
• Known inhibitors of the HH signaling pathway (e.g., vismodegib, patidegib, 
itraconazole, and sonidegib) topically or systemically within 6 months of entry into the 
study. 
• Strong CYP3A4 inhibitors, including but not limited to: larithromycin, telithromycin, 
nefazodone, itraconazole, ketoconazole, atazanavir, darunavir, indinavir, lopinavir, 
nelfinavir, ritonavir, saquinavir, and tipranavir. 
 
Subjects will not be allowed to use topical or systemic therapies that may affect treatment 
targeted BCCs.  Tumors that the Investigator or PSCP determines require treatment during the 
trial can be removed by the modality selected by their PSCP. 
During the study, subjects will be allowed to use moisturizers and emollients and will be 
encouraged to use sunscreen of SPF at least 15 at least once daily on all exposed skin sites. 
Subjects using concomitant therapies during the course of the study that could interfere with 
the interpretation of the study results (including but not limited to those listed above) should 
not be withdrawn, but the use of the concomitant product should be discontinued. No other 
topical treatment (except as noted above) other than the study drug will be permitted. 
Information on concomitant therapies will be recorded in the source document. Any therapy 
used 
by 
the 
subject 
will 
be 
considered 
concomitant 
therapy 
(e.g., aspirin, 


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paracetamol/acetaminophen, vitamins, moisturizers, sunscreens). Every attempt should be 
made to keep concomitant therapy dosing constant during the study. Any change to 
concomitant therapy should be noted on the source document and eCRF. 
7.5 
Treatment Compliance 
Each subject will be instructed on the importance of returning the study drug. Each tube will 
be weighed by the Investigator or designee prior to dispensation and after collection. The 
subject will be asked to keep a record of missed doses. A subject who deviates significantly 
from the prescribed application amount will be counseled. Any missed applications of study 
drug will be noted in the appropriate source document. Missed applications will be documented 
in the eCRF. 
The Investigator will record the time and dose of all administrations in the eCRF.  Any reasons 
for non-compliance will also be documented including: 
• Missed visits 
• Interruptions in the schedule of administration  
• Non-permitted medications 
7.6 
Protocol Deviations and Violations 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Whenever possible, any planned deviations should be agreed to by prior discussion with the 
Medical Monitor and the Investigator, with appropriate documentation of the Medical 
Monitor’s approval prior to effecting the changes agreed upon. 
A deviation from the protocol is an unintended and/or unanticipated departure from the 
procedures and/or processes approved by PellePharm and the Institutional Review Board (IRB) 
or Independent Ethics Committee (IEC) and agreed to by the Investigator. Deviations usually 
have an impact on individual subjects or a small group of subjects and do not involve eligibility 
or primary endpoint criteria. 
A protocol violation occurs when there is non-adherence to the protocol that results in a 
significant, additional risk to the subject, when the subject or Investigator has failed to adhere 
to significant protocol requirements (eligibility criteria) and the subject was enrolled without 
prior PellePharm approval, or when there is non-adherence to Food and Drug Administration 
(FDA) or local authority regulations and/or ICH GCP guideline. 
The Investigator or designee must document and explain in the subjects’ source documentation 
any deviation from the approved protocol. The Investigator may implement a deviation from, 
or a change of, the protocol to eliminate an immediate hazard to study subjects without prior 


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IRB/IEC approval. As soon as possible after such an occurrence, the implemented deviation 
or change, the reasons for it, and any proposed protocol amendment(s) should be submitted to 
the IRB/IEC for review and approval, to PellePharm for agreement, and to the regulatory 
authorities, if required. 


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8. STUDY DRUG MATERIALS AND MANAGEMENT 
Patidegib is generated from a plant source by the extraction of crude cyclopamine.  
Cyclopamine is carried through a series of synthetic steps to yield the well-characterized 
starting material for Good Manufacturing Practice (GMP) conversion to patidegib.  
Patidegib HCl active ingredient is found as a white free-flowing crystalline solid. Patidegib 
topical gel is a smooth clear viscous hydro-alcoholic gel for topical administration. The gel is 
non-irritating and easy to spread on the lesions as directed.  
The following excipients are used in the formulation of patidegib topical gel: All components 
of patidegib gel meet standard US or international compendial standards and are generally 
recognized as safe (GRAS). 
Table 2:  Excipients of Patidegib Topical Gel 
 
 
 
 
 
 
*Components for pH 7.5 borate buffer 
 
Table 3:  Specific Compositions of the Vehicle, 2% and 4% Patidegib Gels 
Component 
4%* 
2%* 
Placebo 
Patidegib  
4.97 
2.49 
0.00 
Transcutol P 
18.80 
18.80 
18.80 
Ethanol 
23.50 
23.50 
23.50 
Propylene Glycol 
18.80 
18.80 
18.80 
pH 7.5 Borate buffer 
30.93 
33.41 
35.90 
Phenoxyethanol 
1.00 
1.00 
1.00 
HPC-HF**  
1.00 
1.00 
1.00 
 
 
 
 
TOTAL  
100.00 
100.00 
100.00 
*The label strength of patidegib topical gels is on a free base adjusted for HCl, trace solvents, 
moisture etc.; while compounding is on the as-is bases corrected by the potency of the 
particular API lot. 
**Hydroxypropylcellulose –HF 
Component 
Monograph 
Patidegib  
N/A 
Transcutol P 
USP, NF, PH. Eur. 
Ethanol 
USP,NF,JP, Ph. Eur. 
Propylene Glycol 
USP, NF, Ph. Eur. 
Purified Water * 
USP, Ph. Eur. 
Boric Acid * 
Ph. Eur. 
Sodium Hydroxide * 
NF, BP, JP, Ph. Eur. 
Phenoxyethanol 
Ph. Eur. 
Hydroxypropylcellulose HF 
Ph. Eur. 


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8.1 
Identity of the Study Drug 
The study drug is identified as patidegib with appropriate labeling, marked “New Drug – 
Limited by Federal (or United States) law to investigational use only”.  
8.2 
Packaging and Dispensation of Study Drug 
The study drug will be dispensed by a trained qualified member of the study staff assigned by 
the Investigator to this task. The study drug will be packaged in 15 gm tubes and provided in 
kits. Subjects will be dispensed the study medication at Baseline and resupplied as needed, 
through Week 12. The tubes will be weighed prior to dispensing. The subject will return the 
tube(s) to the study center, where they will be collected and weighed; partially used tubes can 
be re-dispensed along with new tubes. If the subject loses a tube (lost or damaged tube), another 
tube will be dispensed. The goal is to ensure that the subject has an adequate supply of gel to 
able to administer all scheduled treatments.  
Refer to the Pharmacy Manual for additional details about administration requirements, study 
drug supply and accountability procedures. 
8.3 
Storage, Handling, and Disposal of Study Drug 
The tubes should be stored under controlled room temperature at 20° – 25° C (68° – 77° F). 
Normal variations in temperatures are expected and acceptable around these target 
temperatures for subject’s storage of dispensed tubes. 
8.4 
Application 
The Investigator or their designee will instruct the subject to apply the study drug to the 
affected treatment areas identified at the Baseline visit by the Investigator. The Investigator or 
their designee will instruct the subject on the proper use of laminated dosing cards during the 
Baseline visit. In addition to the verbal instructions given during the visit, the subjects will be 
provided with written instructions.  
Subjects will be instructed to apply a thin layer of study drug to each treatment-targeted BCC 
as indicated on the body diagram once or twice daily up to the Week 12 visit. Subjects will be 
advised to avoid or minimize exposure to direct sunlight while in the study and to wash their 
hands before and after application of the study drug. 
Subjects will be instructed to store their test article in a secure location away from children. 
Women of childbearing potential should not come in contact with the gel. 
The amount of study drug used by the subjects will be monitored by weighing each newly 
dispensed study drug tube and re-weighing each returned study drug tube at all applicable study 
visits. 


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8.5 
Study Drug Accountability 
Upon receipt of the study drug, the Investigator is responsible for ensuring that the designated 
study drug staff conduct a complete inventory of study materials and assume responsibility for 
their storage and dispensing. The Investigators must agree to keep all study materials in a 
secure location with restricted access. The Investigator will keep a record of the inventory and 
dispensing of all study drugs. This record will be made available to the Clinical Monitor for 
the purpose of accounting for all clinical supplies. Any significant discrepancy and/or 
deficiency must be recorded with an explanation. 
All supplies sent to the investigators will be accounted for and, in no case, used in any 
unauthorized situation. Each tube will be weighed (with the cap on) before dispensing to and 
upon return by the subjects, and weights will be recorded on the pharmacy log and appropriate 
eCRF. All supplies will be returned to the sponsor for destruction at the conclusion of the 
study. 


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9. STUDY PROCEDURES AND EVALUATIONS 
The study will be conducted as outlined in the Schedule of Assessments (Table 1). The 
Screening, Week 6, Week 12 and Week 14  study visits will take place in person. The Weeks 
2, 8, and 10 visits may take place using telemedicine and/or an in-person visit. Telemedicine 
will be conducted using a 21 CFR Part 11 and HITRUST/HIPAA compliant platform on a 
mobile device. Telemedicine video and phone conferences are not recorded.  
All subject information and data obtained during the study visits will be recorded in the 
electronic source documents, applicable study logs, and eCRFs. 
Investigators must have appropriate, documented experience and training, or obtain approval 
from PellePharm based on experience (or through additional training organized by 
PellePharm). 
At each study visit, every attempt should be made to ensure that the same Investigator assesses 
the same subject. The sponsor realizes that the conduct of a clinical trial involves a team of 
study staff who perform a variety of functions under the supervision of the Principal 
Investigator. Tasks and responsibilities are delegated by the Principal Investigator as dictated 
by local laws and institutional regulations. There are specific tasks that PellePharm requires be 
done by a licensed healthcare professional with appropriate experience. As outlined below and 
in the Schedule of Assessments (Table 1), these tasks include biopsy for biomarkers, measuring 
and mapping of treatment-targeted tumor(s), determination of clinical tumor type, recording 
application site reactions, and the determination of the ISGTA. 
PellePharm also realizes that scheduling visits can be challenging and has allowed for study 
windows. It should be noted that the indicated visit day is in reference to the Baseline visit. 
For example the Week 6 (Day 43) visit is intended to be Day 43 after the Baseline visit. 
9.1 
Schedule of Evaluations and Procedures 
9.1.1 
Screening Visit (Day -42 to Day 0) 
Following virtual electronic informed consent from each subject, the Investigator will 
determine whether subjects are eligible to participate in the study by performing screening tests 
and evaluations. 
At the Screening visit, continuous monitoring of current and concomitant medications and 
therapies (including prophylactic treatments and medical interventions) and AEs throughout 
the study period will begin. 
Screen failure information will be maintained at the site to document specified information, 
including but not limited to, reason for failure.   


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The following procedures will be conducted at this visit: 
1. Obtain informed consent from the subject prior to performing any study related 
procedures. The subject will receive a copy of their signed consent form. 
2. Review and explain the nature of the study and what will be expected at each visit to 
ensure subject can meet the requirements and has adequate transportation. 
3. Assign the subject a 5-digit subject number, which will consist of the 2-digit site 
number and the 3-digit chronological screening order number, starting with 001 
(e.g., 01 001, 01 002). 
4. Record the subject’s demographic information. 
5. Record the subject’s medical history. 
6. Record all medications for BCC used during the prior year in the eCRF. Include all 
medications used in the past 30 days and any therapy that requires a washout prior to 
Baseline. 
7. Record any prescription or over-the-counter therapies that are being used 
concomitantly in the eCRF. 
8. The Investigator will identify 1 or 2 previously untreated non central facial BCCs  
9. Verify that the subject meets the applicable inclusion/exclusion criteria as outlined in 
Sections 6.1 and 6.2. 
10. Discuss the use of moisturizers and emollients with the subject. 
11. Obtain a biopsy from the baseline treatment targeted BCC(s). 
12. Collect 
blood 
samples 
for 
routine 
laboratory 
analysis 
[complete 
blood 
count/differential (CBC/Diff), urinalysis, serum chemistry and serum pregnancy test 
for females]. 
13. Record any AEs related to screening procedures on the AE eCRF. 
14. Schedule subject’s Baseline/Day 1 visit. If the subject requires a washout, schedule the 
Baseline/Day 1 visit to occur after the washout is complete. 
9.1.2 
Baseline Visit (Day 1) 
The following procedures will be conducted at this visit: 
1. Record any changes in medical history since Screening. 
2. Record changes in any previous BCC medications since the previous visit in eCRF. 
Check for prohibited concomitant therapies and confirm any therapy that requires a 
washout prior to Baseline as per Section 7.4. 
3. Record changes in any concomitant medications since the previous visit in eCRF. 
Check for prior and concomitant therapies as per Section 7.4. 


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4. Verify that the subject continues to meet the applicable study eligibility criteria as 
outlined in Sections 6.1 and 6.2. 
5. An abbreviated physical examination including measurements of weight. 
6. The Investigator will perform the clinical evaluation to identify the treatment-targeted 
tumors which will be clinically classified as superficial, nodular, infiltrative, morphic, 
pigmented or micronodular/morpheaform to be circled in ink, photographed, measured, 
and recorded on a body diagram.  
7. The Investigator or designee will assess the areas to be treated by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation including 
pain/burning, pruritus, erythema, edema, and scabbing/crusting. 
8. The Investigator will query the subject about the symptoms of any abnormalities in 
taste, frequency and severity of muscle cramps, recent changes in quality or quantity 
of hair in treated and untreated areas and any change in frequency of shaving of treated 
or untreated areas, any change in frequency of obtaining haircuts.  
9. Randomize the subject to a treatment group and record the assigned kit number in the 
source document and in the eCRF. 
10. The designated study drug staff will weigh each tube within the assigned kit and 
dispense them to the subject. 
11. The Study Coordinator or designee will instruct the subject on the proper application 
procedure for the study drug. For the first application, the subject will apply the study 
drug at the investigational center under the direction of the Study Coordinator or 
designee. The study drug should be applied after all clinical assessments. The subjects 
will be asked to avoid exposure to direct sunlight on the initial application day and 
thereafter. The Study Coordinator or designee will instruct the subjects to apply the 
study drug once or twice daily at home and review patient instruction sheets and dosing 
card. 
12. Record any AEs reported spontaneously by the subject. 
13. If the subject is a woman of childbearing potential, the Study Coordinator or designee 
will provide the subject with three home pregnancy urine tests. 
14. Schedule the next study visit at Week 2 (Day 15 ± 2 days). 
9.1.3 
Weeks 2, 6, 8, and 10  
The following procedures will be conducted at this visit: 
1. Record changes in any concomitant medications since the previous visit in the eCRF. 
Check for prior and concomitant therapies as per Section 7.4. 
2. Record any new AEs reported spontaneously by the subject or changes in any ongoing 
AEs. 
3. The treatment-targeted BCCs identified by the Investigator at the Baseline visit will be 
photographed and measured using the provided hand held device.  For the Week 6 visit 


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only, the treatment-targeted tumors will be, circled in ink, photographed, measured, and 
recorded on a body diagram. 
4. The Investigator or designee will assess the treated areas by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation including 
pain/burning, pruritus, erythema, edema, and scabbing/crusting.  
5. The Investigator will question the subject about any concurrent illness or systemic sign 
or symptom including but not limited to fatigue, loss or change in taste, nausea, 
vomiting, diarrhea, muscle spasms or cramps, changes in quality or quantity of hair in 
treated areas and any change in frequency of shaving or haircuts.  
6. Collect blood samples for routine laboratory analysis (CBC/Diff and serum chemistry) 
(Weeks 6 only).   
7. If the subject is a woman of childbearing potential, they will take a home pregnancy 
urine test and disclose the results to the Investigator or designee (Weeks 2, 8, and 10 
only). 
8. The Study Coordinator or designee will weigh the previously dispensed study drug 
tube. The study coordinator or designee will weigh and dispense a new study drug tube 
from the subject assigned kit if necessary. (Baseline and Week 6 only) 
9. Any missed doses or deviations should be reported. 
10. The Study Coordinator or designee will remind the subject of the proper technique for 
application of the study drug. If needed, the subject can apply the study drug at the 
investigational center during the day under the direction of the Study Coordinator or 
designee to confirm proper technique. 
11. 
Schedule the next study visit.  
9.1.4 
Week 12 (Day 85 ± 3 Days) / Early Termination 
The following procedures will be conducted at this visit: 
1. Record changes in any concomitant medications since the previous visit in the eCRF. 
Check for prior and concomitant therapies as per Section 7.4. 
2. Record any new AEs reported spontaneously by the subject or changes in any ongoing 
AEs. Record subject weight. 
3. Perform a brief physical exam including vital signs and weight.  
4. Collect blood samples for routine laboratory analysis (CBC/Diff and serum chemistry). 
5. The Investigator or designee will assess the treated areas by observations and 
questioning the subjects as necessary for the signs and symptoms of irritation including 
pain/burning, pruritus, erythema, edema, and scabbing/crusting.  
6. The treatment site(s) will be excised.  
7. The Investigator will question the subject about any concurrent illness or systemic sign 
or symptom including but not limited to fatigue, loss or change in taste, nausea, 


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vomiting, diarrhea, muscle spasms or cramps, changes in quality or quantity of hair in 
treated areas and any change in frequency of shaving.  
8. The Study Coordinator or designee will collect and weigh the previously dispensed 
study drug tube.  
9. Any missed doses or deviations should be reported. 
10. Exit the subject from the study and complete the end of study eCRFs. 
9.1.5 
Week 14 (Day 99 ± 7 Days) / Post Operation Follow-up 
The following procedures will be conducted at this visit: 
1.  The Principal Investigator will do a clinical evaluation of the surgical site(s) to 
confirm adequate healing. 
9.2 
Evaluation of Efficacy 
Clinical Descriptions of Basal Cell Carcinoma 
Nodular basal cell carcinoma 
A pearly, waxy, semi-translucent nodule sometimes forming a central depression that may or 
not be ulcerated or crusted.  There can be overlying telangiectasias and characteristically there 
is a “rolled border”. 
Superficial basal cell carcinoma  
An erythematous scaly thin plaque or patch often with areas of hypopigmentation and 
superficial scarring, occasionally with a thin crust.  Careful examination will often show a 
subtle raised border. 
Infiltrative basal cell carcinoma 
An erythematous, frequently ulcerated or crusted ill-defined plaque.  Occasionally it can be 
quite indurated or elevated. 
Morpheic or sclerosing basal cell carcinoma 
An ill-defined hypopigmented sclerotic plaque, sometimes with subtle telangiectasias.  Rarely 
ulcerated or crusted.  They can present with an atrophic or depressed appearance centrally often 
mimicking an old scar. 
Micronodular basal cell carcinoma 
A dome-shaped sclerotic hypopigmented or flesh colored nodule or plaque with undermining 
borders where a mass-like effect is palpable below normal appearing skin. 
Pigmented basal cell carcinoma 


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Each of the classic variants described above may be pigmented, particularly in Types III-V 
skin.  These will have blue, black, brown pigmentation usually involving part or most of the 
tumor with more classic or characteristic features often noted elsewhere. 
9.2.1 
Assessment of BCCs 
One or two treatment-targeted BCCs on each subject will be identified by the Investigator at 
the following visits: Screening, Baseline, Weeks 2, 6, 8, 10, and 12. The BCCs being followed 
will be circled in ink, photographed, measured the greatest diameter, and recorded on a body 
diagram.   Circling with ink will only be performed at Baseline, Week 6 and Week 12. 
9.2.2 
Messenger RNA (mRNA) 
Biopsies of treatment targeted tumors done at Baseline as well as excisional samples from the 
end of study excisions will be evaluated for GLI1 mRNA levels.  
9.2.3 
Investigator Static Global Tumor Assessment  
The Investigator will assess and record the Investigator Static Global Tumor Assessment 
(ISGTA) of baseline treatment targeted BCC(s) at Baseline, Weeks 2, 6, 8, 10 and Week 12. 
Table 4:  Investigator Static Global Tumor Assessment 
0 
Clear - no evidence of residual tumor is seen or palpated, faint ill-defined macular 
erythema may be present; normal skin markings are seen 
1 
Almost clear - residual macular erythema is clearly seen but there is no palpable* 
obvious tumor, scale, erosions, or ulcerations; normal skin markings are seen 
2 
Minimal residual tumor - macular erythema is clearly seen and visible slight palpable 
tumor with or without scale, erosions, ulcerations may be present; normal skin markings 
are not clearly visible 
3 
Clearly visible tumor - is seen and felt on palpation with or without rolled borders 
erosion, ulceration, or scale; normal skin markings not seen 
*Only evaluated at visits conducted at the study center. 
9.3 
Evaluation of Safety 
9.3.1 
Dermal Safety and Tolerability 
Safety and tolerability will be evaluated through assessments of selected local signs and 
symptoms (pain / burning, pruritus, erythema, edema, and scabbing / crusting). Each of the 


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identified BCCs will be evaluated separately for these signs and symptoms of application site 
reactions. Any local skin reaction that requires use of a concomitant therapy or is a cause for 
study drug interruption or discontinuation should be reported as an AE. The scales to be used 
for assessing local skin reactions follow: 
Table 5:  Dermal Safety and Tolerability Scales 
Score  Grade  
Description 
Pain/Burning: as reported by the subject as being the greatest intensity they have 
experienced at the application site within the last 24 hours at Baseline or since the last 
visit at subsequent visits. 
0 
None 
No pain/burning 
1 
Mild 
Slight burning/stinging sensation; not really bothersome 
2 
Moderate Definite warm, burning/stinging that is somewhat bothersome 
3 
Severe 
Hot burning/stinging sensation that causes definite discomfort and 
may interrupt daily activities and/or sleep 
Pruritus: as reported by the subject as being the greatest intensity they have experienced 
at the application site within the last 24 hours at Baseline or since the last visit at 
subsequent visits. 
0 
None 
No pruritus 
1 
Mild 
Slight pruritus, not really bothersome 
2 
Moderate Definite pruritus that is somewhat bothersome 
3 
Severe 
Intense pruritus that may interrupt daily activities and/or sleep 
Erythema: as assessed by the Investigator at each site 
0 
None 
No erythema present 
1 
Mild 
Slight pink coloration 
2 
Moderate Definite redness 
3 
Severe 
Marked erythema, bright red to dusky dark red in color 
Edema: as assessed by the Investigator at each site 
0 
None 
No edema 
1 
Mild 
Slight, but definite edema 
2 
Moderate Definite edema 
3 
Severe 
Marked edema 
Scabbing/Crusting: as assessed by the Investigator at each site 
 


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0 
None 
No scabbing/crusting 
1 
Mild 
Slight, but definite scabbing/crusting 
2 
Moderate Definite scabbing/crusting 
3 
Severe 
Marked scabbing/crusting 
 
9.3.2 
Medical History and Abbreviated Physical Examination 
A medical history will be taken at Screening, and confirmed and revised if needed, at Baseline. 
Medical conditions that resolved 2 or more years before baseline need not be collected unless 
considered relevant by the Investigator.  
An abbreviated physical examination including measurements of height, weight and vital signs 
(blood pressure, heart rate, respiration rate, and oral temperature) will be performed at Baseline 
and Week 12. Height will be collected at Baseline only. 
9.3.3 
Safety Laboratory Tests 
Routine safety laboratory tests as per Appendix 16.4 will be performed at Screening, Week 6, 
and Week 12. Any out-of-range laboratory result that is considered clinically significant by the 
Investigator will be recorded as an AE and should be accessed for reproducibility by repeat 
testing at the discretion of the Investigator. Clinically significant laboratory abnormalities at 
any visit will be followed to resolution (return to normal or to the Baseline state) or until 
clinically stable as determined by the Investigator. 
9.3.4 
Pregnancy Tests 
All female subjects will have a serum pregnancy test at Screening.  Females of childbearing 
potential will take a home pregnancy urine test at Weeks 2, 8, and 10 and will have a serum 
pregnancy test at Weeks 6 and 12. 
9.3.5 
Adverse Events 
9.3.5.1 
Definition of Adverse Event 
An AE is any untoward medical occurrence in a subject or clinical investigation subject 
administered a medicinal product and which does not necessarily have a causal relationship 
with the study drug. AEs include any unfavorable and unintended illness, sign (e.g., including 
an abnormal laboratory finding), symptom, clinically significant laboratory test abnormality, 
or disease temporally associated with the use of a medicinal product that has appeared or 


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worsened during the course of the clinical trial, regardless of causal relationship to the study 
drug(s) under study.  
9.3.5.2 
Documenting Adverse Events 
It is the responsibility of the Investigator to document all AEs that occur during the course of 
the study. The AEs should be documented as a single medical diagnosis. When this is not 
possible, the AE should be documented in terms of signs and/or symptoms observed by the 
Investigator or reported by the subject at each study visit. 
All AEs occurring after the subject signs the informed consent through the last study visit must 
be reported, regardless of whether or not the AEs are considered drug-related. All AEs, whether 
in response to a query, observed by the study site personnel, or reported spontaneously by the 
subject, will be recorded. 
At each visit during the study, the subject will be assessed for the occurrence of new and 
ongoing AEs. Dermal safety and tolerability that result in the subject’s requiring a concomitant 
therapy or discontinuation from the study will be reported as an AE. The following data will 
be collected on all AEs and recorded on the appropriate CRF: 
• Event name (diagnosis preferred, if unknown, record the signs/symptoms) 
• Onset date and end date 
• Maximum intensity (severity) 
• Seriousness 
• Action taken regarding study drug 
• Corrective treatment, if given 
• Outcome 
In addition, the Investigator’s assessment of causality will be recorded. 
Vital sign abnormalities are to be recorded as AEs only if they are clinically significant (for 
example: are symptomatic, requiring corrective treatment, leading to discontinuation or 
fulfilling a seriousness criterion). 
Subjects will be questioned about any concurrent illness or systemic sign or symptom including 
but not limited to fatigue, loss or change in taste, nausea, vomiting, alopecia, diarrhea, and 
muscle spasms. Any incidence of these systemic signs or symptoms will be reported on the AE 
eCRF. 


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9.3.5.3 
Serious Adverse Events 
All AEs will be assessed as either serious or non-serious. 
An SAE or serious adverse reaction is defined as any untoward medical occurrence that at any 
dose: 
• Results in death 
• Is immediately life threatening, (the term "life threatening" in the definition of 
"serious" refers to an event in which the subject is at risk of death at the time of the 
event; it does not refer to an event which hypothetically might have caused death if 
it were more severe) 
• Requires in subject hospitalization or prolongation of existing hospitalization 
(hospitalization for elective surgery for a baseline condition is not considered an 
AE) 
• Results in persistent or significant disability/incapacity (permanent or substantial 
disruption of a person’s ability to conduct normal life functions) 
• Is a congenital anomaly/birth defect 
• Is a medically important event that may not be immediately life threatening or result 
in death or hospitalization, but may jeopardize the subject and may require medical 
or surgical intervention to prevent any of the above listed outcomes 
Note: A spontaneous abortion will be considered an SAE, and must be reported per Reporting 
of SAEs under Section 9.3.5.6. 
9.3.5.4 
Assessment of Severity 
The severity assigned to an AE should be determined by the maximum severity of the AE. The 
categories described below should be used to estimate the severity of AEs: 
• Mild: Transient or mild discomfort; no limitation in activity; no medical 
intervention/therapy required 
• Moderate: Mild to moderate limitation in activity; some assistance may be needed; 
no or minimal medical intervention/therapy required 
• Severe: Marked limitation in activity; some assistance usually required; medical 
intervention/therapy required; hospitalization or prolongation of current 
hospitalization possible; may be incapacitating or life threatening 


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9.3.5.5 
Assessment of Causality 
The Investigator should assess the relationship of the AE, if any, to the study drug. The 
following should be taken into account when assessing SAE causality: 
• Positive temporal relationship to study drug, such as if the study drug was 
withdrawn and the SAE resolved or the event recurred after re-introduction. 
• If there is a reasonable possibility that the AE is associated with an underlying or 
concomitant illness. 
• Possible association with previous or concomitant therapy. 
• No temporal relationship to the study drug and/or a more likely alternative etiology 
exists. 
• If the AE is directly related to study procedures or a lack of efficacy. 
The following classifications should be used when evaluating the relationship of AEs and 
SAEs to the investigational drug. 
1. None: No relationship between the experience and the administration of study drug; related 
to other etiologies such as concomitant medications or subject’s clinical state. 
2. Unlikely: The current state of knowledge indicates that a relationship is unlikely.   
3. Possibly: A reaction that follows a plausible temporal sequence from administration of the 
study drug and follows a known response pattern to the suspected study drug.  The reaction 
might have been produced by the subject’s clinical state or other modes of therapy 
administered to the subject. 
4. Probably: A reaction that follows a plausible temporal sequence from administration of 
the study drug and follows a known response pattern to the suspected study drug.  The 
reaction cannot be reasonably explained by the known characteristics of the subject’s 
clinical state or other modes of therapy administered to the subject. 
5. Definitely: A reaction that follows a plausible temporal sequence from administration of 
the study drug and follows a known response pattern to the suspected study drug and can 
be confirmed with a positive re-challenge test or supporting laboratory data 
9.3.5.6 
Reporting of Serious Adverse Events 
When new significant information is obtained as well as when the outcome of an event is 
known, the Investigator should record the information on a new SAE form.  If the subject was 
hospitalized, a copy of the discharge summary must be included as part of the subject’s medical 
file.  In all instances, the Investigator should follow up with subjects until the outcome of the 
SAE is known. 


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All AEs spontaneously reported by the subject and/or in response to an open question from 
study personnel or revealed by observation, physical examination or other diagnostic 
procedures will be recorded on the AE eCRF.  Any clinically relevant change in laboratory 
assessments or other clinical findings is considered an AE and must be recorded on the AE 
eCRF.  All AEs are to be followed until the event resolves or the clinical course is stabilized. 
For SAEs, a SAE form must be completed with as much information as possible and submitted 
in the time frame described below. 
PellePharm must be notified of all SAEs (regardless of casual relationship to study drug) within 
24 hours of first knowledge of the event by the Investigator or other study personnel by faxing 
a completed SAE form to the contact information below.   
Safety Reporting Contact 
Name: 
 
Telephone: 
 
 
Mobile: 
 
Facsimile: 
 
Email: 
 
 
If there are serious, unexpected AEs associated with the use of the study drug, PellePharm will 
notify the appropriate regulatory agency(ies) and all appropriate parties on an expedited basis.  
It is the responsibility of the Investigator to promptly notify the IRB/IEC of all unexpected 
SAEs involving risk to human subjects. 
The Investigator should take all appropriate measures to ensure the safety of the subjects, 
notably and should follow a subject with an SAE until the event has resolved or the condition 
has stabilized. This may imply that follow-up will continue after the subject has left the study, 
and that additional investigations may be requested by PellePharm. 
9.3.5.7 
 Emergency Contact 
In the event of a medical emergency (i.e., an event that requires immediate attention regarding 
the treatment of a subject, operation of the clinical study, and/or the use of investigational 
drug), investigational site personnel must immediately contact the Medical Monitor. 
 
 
PPD
PPD
PPD
PPD
PPD
PPD


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Medical Monitor Contact 
Name: 
 
Telephone: 
 
Email: 
 
 
9.3.5.8 
Expedited Serious Adverse Event Reports 
An AE, whether serious or non-serious, is designated unexpected (unlabeled) if it is not 
reported in the clinical safety section of the Investigator Brochure or if the event is of greater 
frequency, specificity or severity. 
Expedited SAE reports are those that document AEs that are both unexpected based on the 
reference document (Investigator Brochure) and are related (i.e., the relationship cannot be 
ruled out) to the study drug. These expedited reports are subject to reporting timelines of 7 
(SAEs) and/or 15 (non SAE) calendar days to the regulatory reporting agency(ies). PellePharm 
will notify regulatory authorities of these AEs and all participating investigational centers in 
writing for submission by the Investigator to the IRB/IEC. This notification will be in the form 
of a Safety Update to the Investigator Brochure (i.e., “15-day letter”). 
Upon receiving such notices, the Investigator must review and retain the notice with the 
Investigator Brochure and immediately submit a copy of this information to the responsible 
IRB/IEC according to local regulations. The Investigator and IRB/IEC will determine if the 
informed consent requires revision. The Investigator should also comply with the IRB/IEC 
procedures for reporting any other safety information. 
9.3.5.9 
Other Required Safety Assessments 
A clinically significant worsening from Baseline of any abnormal study assessment, such as 
laboratory test, physical examination, or vital signs, should be considered an AE and recorded 
accordingly.  If possible, a diagnosis for the clinically significant study assessment should be 
provided by the Investigator (e.g., urinary tract infection or anemia).  In the absence of a 
diagnosis, the abnormal study assessment itself should be listed as the AE (e.g., bacteria in 
urine or decreased hemoglobin).  
An abnormal study assessment is considered clinically significant if the subject has one or 
more of the following related to the abnormal study assessment: 
1. 
Concomitant clinical signs or symptoms  
2. 
Further diagnostic testing or medical/surgical intervention 
3. 
A change in the dose of study drug or is discontinued from the study 
PPD
PPD
PPD


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Repeat testing to determine whether the result is abnormal, in the absence of any of the above 
criteria, does not necessarily meet clinically significant criteria.   
 


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10. STATISTICS 
All statistical processing will be performed using SAS® unless otherwise stated. If 
determined appropriate by the Sponsor, limited safety and efficacy interim analysis on tumor 
shrinkage and biomarkers may be performed.  
No inferential testing will be performed. Descriptive statistics will be used to provide an 
overview of the efficacy and safety results. For categorical parameters, the number and 
percentage of subjects in each category will be presented. For continuous parameters, 
descriptive statistics will include n (number of subjects), mean, and standard deviation, 
median, minimum, and maximum.  
No imputation will be made for the primary endpoint.  For the secondary endpoint, the last 
observation carried forward method (LOCF) will be used to impute missing efficacy data (i.e., 
the last available on-therapy observation for a subject will be used to estimate subsequent 
missing data points). No imputations will be made for missing safety data. 
A statistical analysis plan (SAP), describing all statistical analyses will be provided as a 
separate document. The SAP will be finalized prior to unblinding of the study treatments. 
10.1 
Analysis Populations 
Per protocol analysis will be performed –samples with insufficient BCC, subjects with poor 
treatment compliance, or tumors where the diameter was incorrectly measured based on review 
of photographs will be excluded. BCC size change based on photographs will also be analyzed. 
Safety analyses will be performed using the safety population. All subjects who are 
randomized, receive at least 1 confirmed dose of study drug and have at least one post-baseline 
safety assessment will be included in the safety analysis set. 
10.2 
Subject Disposition 
A tabulation of subject disposition will be provided. The tabulation will include the numbers 
of subjects who enter the study, complete the study, and discontinue the study. The reasons for 
discontinuation will be included. 
10.3 
Demographics and Baseline Characteristics 
Subject demographic data and baseline characteristics will be summarized by treatment group 
using descriptive statistics for the ITT and Safety populations.  


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10.4 
Protocol Deviations 
All protocol deviations will be reported to the PellePharm and recorded throughout the study. 
A tabulation of protocol deviations will be included in the final study report. 
10.5 
Compliance 
No formal evaluations of compliance are planned.  
10.6 
Interim Analyses 
If determined appropriate by the Sponsor, limited safety and efficacy interim analysis on 
tumor shrinkage and biomarkers may be performed.  
 
10.7 
Assessment of Efficacy 
10.7.1 
Efficacy Summaries 
The efficacy endpoints are intended to compare once and twice daily application of 2% 
patidegib gel, 4% patidegib gel, and vehicle gel. Efficacy assessments will be summarized 
descriptively by treatment group and visit.   
10.7.1.1 
Primary Efficacy Endpoint:  
Change in GLI1 mRNA levels in drug-treated versus vehicle-treated tumors after 12 weeks 
of treatment.  
10.7.1.2 
Secondary Efficacy Endpoints: 
The secondary efficacy endpoint is the decrease in tumor size defined as percent decrease in 
the greatest diameter of baseline treatment targeted BCCs at the Week 12. 
10.7.1.3 
Exploratory Efficacy Endpoints: 
To evaluate the utility of an investigator static global tumor assessment (ISGTA) in assessing 
the proportion of baseline treatment-targeted BCCs that are evaluated as being clear or 
almost clear. 
To evaluate if the proportion of baseline treated tumors are smaller based on photographic 
review 
 


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10.7.2 
Efficacy Analyses.  
The primary endpoint and the secondary endpoints of change in tumor size, and reduction in 
the HH signaling pathway will be evaluated with an analysis of covariance (ANCOVA) with  
treatment group as a factor and baseline value as a covariate. Pairwise comparisons will be 
performed using contrasts within the ANCOVA. Additionally, t-tests for pairwise comparisons 
of the primary endpoint will be done, or alternatively a non-parametric approach may be used. 
The exploratory analysis of ISGTA will be analyzed with a Cochran-Mantel-Haenszel test 
10.8 
Assessment of Safety 
10.8.1 
Dermal Safety and Tolerability 
The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, 
erythema, edema, and scabbing/crusting will be summarized descriptively by treatment group 
and visit. 
10.8.2 
Adverse Events 
Subjects will be assessed for the occurrence of new and ongoing AEs. Descriptions of AEs 
will include the dates of onset and resolution (if resolved), maximum severity, seriousness, 
action taken regarding the study drug, corrective treatment, outcome, and Investigator’s 
assessment of causality. All AEs will be recorded and classified using terminology from the 
Medical Dictionary for Regulatory Activities (MedDRA). All reported treatment-emergent 
AEs (TEAEs), defined as any AE with an onset on or after the date of first study drug 
application, will be summarized by treatment group, the number of subjects reporting TEAEs, 
system organ class, preferred term, severity, and relationship to study drug. When summarizing 
TEAEs by severity or relationship to study drug, each subject will be counted only once within 
a system organ class or a preferred term using the event with the greatest severity or causality, 
respectively, within each category. All reported SAEs will be summarized by treatment group, 
the number of subjects reporting SAEs, system organ class, preferred term, severity, and 
relationship to study drug. 
All information pertaining to AEs noted during the study will be listed by subject and will 
include a verbatim description of the event as reported by the Investigator, as well as the 
preferred term, system organ class, start date, stop date (if stopped), seriousness, severity, 
action taken regarding the study drug, corrective treatment, outcome and relationship to the 
study drug. In addition, a listing of subjects who prematurely discontinue from the study due 
to AEs will be provided as well as a listing of subjects who reported an SAE. 


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10.8.3 
Safety Laboratory Values and Vital Sign Measurements 
Changes from Baseline in safety laboratory values and vital sign measurements will be 
summarized with descriptive statistics for each treatment group at all applicable study visits. 
Shift tables will be presented for changes in safety laboratory values to summarize laboratory 
test results collected at Screening and Weeks 6, and 12. Normal ranges established by the 
local laboratory will be used to determine the shifts. A listing of all out-of-range laboratory 
test results at any assessment time point will also be provided. Determination of clinical 
significance for all out-of-range laboratory values will be made by each Investigator and 
included in the listing. In addition, a listing of all clinically significant laboratory test results 
will be provided. 
10.8.4 
Pregnancy Tests 
Pregnancy test results will be presented in a data listing. 
10.8.5 
Handling of Missing Data 
The last observation carried forward (LOCF) method will be used to impute missing efficacy 
data (i.e., the last available on-therapy observation for a subject will be used to estimate 
subsequent missing data points). No imputations will be made for missing safety data. 
10.8.6 
Multicenter Issues 
The study will be conducted at one metasite in the United States with the intention of pooling 
the results for analysis. 
10.8.7 
Multiplicity Issues 
Not applicable. Inferential tests will not be performed. 
10.9 
Sample Size Determination 
This is a dose ranging study. 


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11. QUALITY CONTROL AND QUALITY ASSURANCE 
11.1 
Study Monitoring 
An Investigator Meeting and/or an initiation visit will be conducted with the Principal 
Investigator, Sub-Investigator, and Study Coordinator by PellePharm and/or its designee. 
During this meeting, an extensive review and discussion of the protocol, the role of the study 
technician, all study procedures, source documents, and eCRFs will be conducted. Evaluation 
procedures will be reviewed extensively and documentation of training will be recorded for 
training of sponsor-approved evaluators. 
The clinical monitors will be trained prior to study initiation. Following this training, an 
overview of the study disease and study material background will be understood. Specific 
monitoring guidelines and procedures to be followed during monitoring visits will also be 
utilized. During the course of the study, all data will be 100% source document verified by the 
monitors. All subject source records must be made available to the monitors. 
The conduct of the study will be closely monitored by the sponsor following GCP guidelines. 
The reports of these verifications will also be archived with the study report. In addition, 
inspections or on site audits may be carried out by local authorities or by the sponsor's Quality 
Assurance Department. The Investigators will allow the sponsor's representatives and any 
regulatory agency to examine all study records, corresponding subject medical records, clinical 
dispensing records and storage area, and any other documents considered source 
documentation. The Investigators agree to assist the representative, if required. 
11.2 
Audits and Inspections 
The study will be conducted under the sponsorship of PellePharm in conformation with all 
appropriate legal regulations, as well as ICH guidelines. Interim and end of study audits of raw 
data, study files, and final report may be conducted by PellePharm’s Quality Assurance 
Department or designee. 
The sponsor is responsible for implementing and maintaining quality assurance and quality 
control systems to ensure that studies are conducted and data are generated, documented, and 
reported in compliance with the protocol, GCP, and applicable regulatory requirements. In 
addition, the sponsor will be responsible for securing agreement from all involved parties to 
ensure direct access to all study related investigational centers, source data/documents, eCRFs, 
and reports for the purpose of monitoring and auditing by the sponsor, and inspection by 
domestic and foreign regulatory authorities. 


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11.3 
Data Quality Assurance 
All assessments performed will be accurately documented in the subject’s source documents 
and eCRFs. The Investigator or designee will enter the information required by the protocol 
into the source documents and eCRFs provided by the sponsor or designee. Subjects will be 
identified in the eCRFs by their assigned subject number and initials only. 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Any deviations should be agreed to by prior discussion between the sponsor and the 
Investigator, with appropriate written protocol amendments made prior to implementing the 
agreed changes. Any amendment containing major modifications (particularly if it may involve 
an increased risk to the subjects) will be approved by the IRB before it may be implemented. 
No change in the conduct of the study can be instituted without written approval from the 
sponsor. 


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12. ETHICS AND ADMINISTRATIVE ISSUES 
12.1 
Ethical Conduct of the Study 
This study will be conducted in accordance with the ethical principles originating from the 
Declaration of Helsinki, ICH guidelines, GCP, and in compliance with local regulatory 
requirements. The Investigator agrees, when signing the protocol, to adhere to the 
instructions and procedures described in it and thereby to adhere to the principles of GCP. 
12.2 
Independent Review Board Review 
This protocol, proposed informed consent form and other information to subjects, and all 
appropriate amendments will be properly reviewed and approved by the IRB/IEC. A signed 
and dated notification of the IRB/IEC approval will be provided to the sponsor and Investigator 
prior to study initiation. The name and occupation of the chairman and members of the 
IRB/IEC will be supplied to the sponsor. The Investigator will provide required progress 
reports and report all SAEs to the IRB/IEC as required by the IRB/IEC. 
12.3 
Informed Consent 
Electronic informed consent is required from each subject prior to any testing under this 
protocol, including screening tests and evaluations. The informed consent form (ICF), as 
specified by the investigational site’s IRB/IEC, must follow the Protection of Human Subjects 
regulations listed in 21 CFR Part 50.   
The background of the proposed study and the benefits and risks of the procedures and study 
must be explained to the subjects.  It is the responsibility of the Investigator to obtain consent 
and to provide the subject with a copy of the signed and dated ICF.  Confirmation of a subject’s 
informed consent must also be documented in the source documentation prior to any testing 
under this protocol, including screening tests and evaluations. 
All ICFs used in this study must be approved by the appropriate IRB/IEC and by PellePharm 
or designee.  The ICF must not be altered without the prior agreement of the relevant IRB/IEC 
and PellePharm. 
12.4 
Subject Data Protection 
Subject data will be protected by ensuring that no captured data contain subject names, 
addresses, telephone numbers, email addresses, or other direct personally identifying 
information. It is acknowledged that subject initials, demographics (including birthdate), 
medical histories, and prior concomitant medication uses, along with the name and address of 
the enrolling investigator may allow for personal identification of study participants. Other 
than where necessary to meet regulatory requirements, all data collected in this study will be 


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presented in tabulated (i.e., aggregate) form and listings containing information that could be 
used to identify an individual subject will not be included in any public disclosures of the study 
data or the study results. 
12.5 
Financial Disclosure 
Financial disclosures will be obtained from all investigators in order to document any potential 
conflicts of interest. 
12.6 
Investigator Obligations 
The Investigator agrees, when signing the protocol, to adhere to the instructions and procedures 
described in it and thereby to adhere to the principles of Good Clinical Practice (GCP). 
12.7 
Changes to the Protocol 
The Investigators must read the protocol thoroughly and must follow the instructions exactly. 
Whenever possible, any planned deviations should be agreed to by prior discussion between 
the sponsor and the Investigator, with appropriate documentation of sponsor approval prior to 
effecting the changes agreed upon. Any amendment to the protocol containing major 
modifications (particularly if it may involve an increased risk to the subjects) will be approved 
by the IRB before it may be implemented. No change in the conduct of the study can be 
instituted without written approval from the sponsor. 
12.8 
Confidentiality/Publication of the Study 
All the data furnished to the Investigator and his/her staff and all data obtained through this 
protocol will be regarded as confidential and proprietary in nature and will not be disclosed to 
any third party, except for the FDA or other regulatory body, without written consent from the 
sponsor. 


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13. DATA HANDLING AND RECORD KEEPING 
13.1 
Inspection of Records 
Investigators must maintain detailed records on all study subjects who are enrolled in the study 
or undergo screening. Data will be recorded in the subject’s source documents and in 
applicable study logs provided by the sponsor. Source documents include subject medical 
records, hospital charts, clinic charts, investigator subject study files, as well as the results of 
diagnostic tests (e.g., laboratory tests). All required data should be recorded in the study 
documentation completely for prompt data review. Upon study completion or at any other time 
specified by the sponsor or designee, the appropriate study documents must be submitted. 
The Investigator must keep accurate separate records (source documentation) of all subject 
visits, being sure to include all pertinent study related information. At a minimum, this includes 
the following information: 
• A statement indicating that the subject has been enrolled in the study and the subject 
number 
• Date that virtual electronic informed consent was obtained 
• Evidence that the subject meets study eligibility requirements (e.g., medical history, 
screening evaluations) 
• Dates of all study related visits and results of any evaluations/procedures 
performed, including who performed each assessment at each visit 
• Use of any concurrent medications during the study 
• Documentation of study drug accountability 
• Any and all side effects and AEs must be thoroughly documented to conclusion 
• Results of any diagnostic tests conducted during the study 
• The date the subject exited the study and a statement indicating that the subject 
completed the study or was discontinued early, including the reason for 
discontinuation 
Notes describing telephone conversations and all electronic mail with the subject or the sponsor 
(sponsor’s designee) concerning the study must be recorded or kept on file. All source 
documents must be made available to the sponsor and the sponsor’s designated monitor upon 
request. 


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13.2 
Retention of Records 
The Investigator should properly store and maintain all study records in accordance with 
sponsor directives. All records relating to the conduct of this study are to be retained by the 
Investigator until notified by the sponsor in writing that the records may be destroyed. 
The Investigator will allow representatives of the sponsor’s monitoring team, the governing 
IRB/IEC, the FDA or other applicable local authorities to inspect all study records, CRFs, and 
corresponding portions of the subject’s clinic and/or hospital medical records at regular 
intervals throughout the study. These inspections are for the purpose of verifying adherence to 
the protocol, completeness and accuracy of the data being entered onto the CRF, and 
compliance with FDA or other local authority regulations. 
13.3 
Electronic Case Report Form Completion 
eCRFs will be completed for each enrolled subject.  It is the Investigator’s responsibility to 
ensure the accuracy, completeness, and timeliness of the data reported in the subject’s eCRF.  
Source documentation supporting the eCRF data should indicate the subject’s participation in 
the study and should document the dates and details of study procedures, AEs, and subject 
status. 
Investigators will maintain copies of the eCRFs at the investigational site.  For subjects who 
discontinue or terminate from the study, the eCRFs will be completed as much as possible, and 
the reason for the discontinuance or termination clearly and concisely specified on the 
appropriate eCRF. 
 


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14. REFERENCES 
1. 
Gorlin RJ, Goltz RW. Multiple nevoid basal-cell epithelioma, jaw cysts and bifid rib: 
a syndrome. New England Journal of Medicine 1960;262:908-12. 
2. 
Wilding A, Ingham SL, Lalloo F, et al. Life expectancy in hereditary cancer 
predisposing diseases: an observational study. Journal of medical genetics 2012;49:264-9. 
3. 
Peters M, Ransohoff K, Lindgren J, Tang J, Epstein E. Prospective United States 
based Registry of Basal Cell Nevus Syndrome (Gorlin) patients.  EADV 2015 Annual 
Meeting2015. 
4. 
Hahn H, Wicking C, Zaphiropoulos PG, et al. Mutations of the human homolog of 
Drosophila patched in the nevoid basal cell carcinoma syndrome. Cell 1996;85:841-51. 
5. 
Johnson RL, Rothman AL, Xie J, et al. Human homolog of patched, a candidate gene 
for the basal cell nevus syndrome. Science 1996;272:1668-71. 
6. 
Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nature Reviews Cancer 
2008;8:743-54. 
7. 
Kimonis VE, Goldstein AM, Pastakia B, et al. Clinical manifestations in 105 persons 
with nevoid basal cell carcinoma syndrome. American journal of medical genetics 
1997;69:299-308. 
8. 
Xie J, Murone M, Luoh S-M, et al. Activating Smoothened mutations in sporadic 
basal-cell carcinoma. Nature 1998;391:90-2. 
9. 
Sarin A, Migden M, al e. Lack of resistant SMO mutations and decreased mutational 
load of Gorlin-associated basal cell cancers explain marked response to smoothened 
inhibitors. SID 2015, Atlanta Poster #135. 
10. 
Kirkup M, De Berker D. Clinical measurement of dimensions of basal cell carcinoma: 
effect of waiting for elective surgery. British Journal of Dermatology 1999;141:876-9. 
11. 
Tan E, Lin F, Sheck L, Salmon P, Ng S. Growth of periocular basal cell carcinomas. 
British Journal of Dermatology 2015;172:1002-7. 
12. 
Tang JY, Mackay-Wiggan JM, Aszterbaum M, et al. Inhibiting the hedgehog 
pathway in patients with the basal-cell nevus syndrome. New England Journal of Medicine 
2012;366:2180-8. 
13. 
Mathias S, Chren MM, Crosby R, et al. Reliability and Validity of the Advanced 
Basal Cell Carcinoma Index (aBCCdex). British Journal of Dermatology 2015. 
14. 
Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced 
basal-cell carcinoma. New England Journal of Medicine 2012;366:2171-9. 
15. 
Sharpe HJ, Pau G, Dijkgraaf GJ, et al. Genomic Analysis of Smoothened Inhibitor 
Resistance in Basal Cell Carcinoma. Cancer cell 2015;27:327-41. 
16. 
Ally MS, Aasi S, Wysong A, et al. An investigator-initiated open-label clinical trial 
of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. Journal of the 
American Academy of Dermatology 2014;71:904-11. e1. 
 
 
 


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15. APPENDICES 
15.1 
Body Diagram 
 
Appendix: Photographs of tumors and sequence of regional photos 
 
1. At Baseline: Identify the 2 treatment targeted BCCs with labels 
2. Measure greatest diameter of each tumor, perform ISGTA and determine clinical 
type. 
3. Identify all other tumors measure and record their greatest diameter and the clinical 
type of each tumor. 
4. Circle all tumors both treatment targeted tumors and other tumors:  
 
6 quadrants on face/neck: 
1. R forehead 
2. R cheek 
3. R neck 
4. L forehead 
5. L cheek 
6. L neck 
 


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20 quadrants on body: 
1. R chest 
2. L chest 
3. R abdomen 
4. L abdomen 
5. R upper arm 
6. R lower arm 
7. L upper arm 
8. L lower arm 
9. R anterior thigh 
10. L anterior thigh 
11. R anterior leg 
12. L anterior leg 
13. R upper back 
14. L upper back 
15. R lower back 
 
16. L lower back 
 
17. R posterior thigh 
 
18. L posterior thigh 
 
19. R posterior leg 
 
20. L posterior leg 
 
 
 


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15.2 
Safety Laboratory Tests 
The following blood samples for laboratory tests will be collected:  
• Alanine aminotransferase (ALT) 
• Albumin, alkaline phosphatase 
• Aspartate aminotransferase (AST) 
• Total bilirubin 
• Blood urea nitrogen (BUN) 
• Calcium, carbon dioxide, chloride 
• Creatinine 
• Glucose 
• Potassium 
• Protein 
• Sodium 
• White blood cell (WBC) 
• Red blood cell (RBC) 
• Hemoglobin 
• Hematocrit 
• Mean Corpuscular Volume (MCV) 
• Mean corpuscular hemoglobin (MCH) 
• Mean corpuscular hemoglobin concentration (MCHC)  
• Red Cell Distribution Width 
• Platelets, Mean Platelet Volumes 
• Absolute/Percent Neutrophil Count 
• Absolute/Percent Lymphocyte count 
• Absolute/Percent Monocyte count 
• Absolute/Percent Eosinophil Count 
• Absolute/Percent Basophil Count 
 
Urinalysis with reflex microscopic examination.   
 
 
 
 
  
 

SAP_000.pdf: 
 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 1 
Protocol Number: 926-202 
28Sep2017 
 
 
 
STATISTICAL ANALYSIS PLAN 
 
 
 
 
 
 
Protocol Number: Pelle-926-202 
 
 
 
Study Title:  Double-Blind, Dose Escalating, Randomized, Vehicle-Controlled Proof of 
Concept Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily 
to Decrease the GLI Biomarker in Sporadic Nodular Basal Cell Carcinomas 
 
 
 
Development Phase of Study: 2A 
 
 
 
Sponsor: PellePharm, Inc. 
 
Sponsor Contact:  
 
 
 
 
Statistical Analysis Plan based on Protocol Version: 3 24Aug2017 
 
 
 
Statistical Analysis Plan Date: 28Sep2017 
 
Statistical Analysis Plan Version: 1 
 
 
 
PPD


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 2 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Authored by: 
SIGNATURE: _________________________________ 
 
DATE: ________________ 
 
 
 
 
 
 
Reviewed by: 
SIGNATURE: _________________________________ 
 
DATE: ________________ 
 
 
 
 
 
 
 
Approved by: 
SIGNATURE: _________________________________ 
 
DATE: ________________ 
 
PellePharm, Inc. 
 
 
 
 
Revisions to the Statistical Analysis Plan described herein must be approved through a formal written amendment 
with the exception of minor editorial changes to tables, figures, or listing shells, and any necessary textual 
clarifications for programmers that do not affect the stated analysis variables, study endpoints, or statistical methods. 
 
 
 
 
PPD
PPD
PPD
PPD
PPD
PPD
PPD


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 3 
Protocol Number: 926-202 
28Sep2017 
 
 
Change History: 
 
 
Version Date 
Summary of Changes  
Author 
1.0 
28Sep2017 Version 1.0 
 
 
  
 
PPD


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 4 
Protocol Number: 926-202 
28Sep2017 
 
 
TABLE OF CONTENTS 
1. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ....................................6 
2. 
INTRODUCTION .................................................................................................................7 
3. 
STUDY OBJECTIVES .........................................................................................................8 
4. 
STUDY DESIGN ...................................................................................................................9 
4.1 Overall Study Design ......................................................................................................9 
4.1.1 
Schedule of Visits and Assessments ...............................................................10 
4.1.2 
Method of Assigning Subjects to Treatment Groups ......................................10 
4.1.3 
Blinding ...........................................................................................................11 
5. 
EFFICACY AND SAFETY ENDPOINTS .......................................................................11 
5.1 Efficacy Endpoints ........................................................................................................11 
5.1.1 
Primary Efficacy Endpoint ..............................................................................11 
5.1.2 
Secondary Efficacy Endpoint ..........................................................................11 
5.1.3 
Exploratory Efficacy Endpoints ......................................................................12 
5.2 Safety Endpoints ...........................................................................................................12 
6. 
STATISTICAL AND ANALYTICAL PLANS ................................................................12 
6.1 General Methodology ...................................................................................................12 
6.1.1 
Statistical Analysis ..........................................................................................13 
6.1.2 
Baseline Definition ..........................................................................................13 
6.1.3 
Visit Windowing .............................................................................................13 
6.1.4 
Adjustments for Covariates .............................................................................14 
6.1.5 
Handling of Dropouts or Missing Data ...........................................................14 
6.1.6 
Interim Analyses and Data Monitoring ...........................................................14 
6.1.7 
Multicenter Studies ..........................................................................................15 
6.1.8 
Multiple Comparisons/Multiplicity .................................................................15 
6.1.9 
Use of an Efficacy Subset of Subjects .............................................................15 
6.1.10 Active-Control Studies Intended to Show Equivalence ..................................15 
6.1.11 Examination of Subgroups ..............................................................................15 
6.2 Disposition of Subjects .................................................................................................15 
6.3 Protocol Deviations .......................................................................................................15 
6.4 Data Sets Analyzed .......................................................................................................16 
6.4.1 
Intent-to-Treat (ITT) Population .....................................................................16 
6.4.2 
Safety Population ............................................................................................16 


 
 
 
 
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6.4.3 
Per Protocol (PP) Population ...........................................................................16 
6.5 Demographic and Other Baseline Characteristics ........................................................17 
6.6 Prior and Concomitant Medications .............................................................................17 
6.7 Analysis of Efficacy ......................................................................................................17 
6.7.1 
Primary Efficacy Analysis ...............................................................................17 
6.7.2 
Secondary Efficacy Analysis ...........................................................................18 
6.7.3 
Exploratory Efficacy Analysis ........................................................................18 
6.8 Safety Evaluation ..........................................................................................................18 
6.8.1 
Extent of Exposure and Compliance ...............................................................18 
6.8.2 
Adverse Events ................................................................................................19 
6.8.3 
Clinical Laboratory Evaluation .......................................................................19 
6.8.4 
Other Observations Related to Safety .............................................................19 
6.8.4.1 
Dermal Safety and Tolerability ....................................................19 
6.8.4.2 
Systemic Symptoms .....................................................................20 
6.8.4.3 
Vital Signs ....................................................................................20 
6.8.4.4 
Physical Examination...................................................................20 
7. 
DETERMINATION OF SAMPLE SIZE .........................................................................20 
8. 
CHANGES IN THE PLANNED ANALYSES..................................................................20 
9. 
REFERENCES ....................................................................................................................20 
10. INDEX OF PLANNED END-OF-TEXT TABLES AND FIGURES .............................21 
11. INDEX OF PLANNED LISTINGS ...................................................................................55 
 
 


 
 
 
 
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1. LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
AE 
adverse event 
ALT 
Alanine aminotransferase 
ANCOVA 
analysis of covariance 
AST 
Aspartate aminotransferase 
ATC 
anatomical therapeutic chemical 
BCC 
Basal cell carcinoma 
BCNS 
basal cell nevus syndrome 
BUN 
blood urea nitrogen 
cm 
centimeters 
CMH 
Cochran-Mantel-Haenszel 
CRF 
case report form 
eCRF 
electronic case report form 
ET 
early termination 
F 
Fahrenheit 
FDA 
Food and Drug Administration 
GLI 1 
glioma-associated oncogene homolog 1 
HH 
hedgehog 
HR 
heart rate 
hr(s) 
hour(s) 
ISGTA 
Investigator Static Global Tumor Assessment 
ITT 
intent-to-treat 
kg 
kilograms 
LOCF 
last observation carried forward 
max 
maximum 
MCH 
Mean corpuscular hemoglobin 
MCHC 
Mean corpuscular hemoglobin concentration 
MCV 
Mean corpuscular volume 
MedDRA 
Medical Dictionary for Regulatory Activities 
mg 
milligram 
min 
minimum 
mRNA 
messenger ribonucleic acid 
n 
number of observations 
N 
number of subjects (sample size) 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
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PSCP 
primary skin care physician 
QST 
QST Consultations, Ltd. 
RBC 
Red Blood Cell 
SAE 
serious adverse event 
SAS® 
Statistical Analysis System (SAS® Institute Inc., Cary, NC) 
SD 
standard deviation 
SEB 
surgically eligible basal cell carcinoma 
SMO 
smoothened 
SPF 
skin protection factor 
SOC 
system organ class 
TEAE 
treatment-emergent adverse event 
US 
United States 
UV 
ultraviolet 
WBC 
White blood cell count 
WHO 
World Health Organization 
WHO-DDE 
World Health Organization Drug Dictionary 
 
 
2. INTRODUCTION 
PellePharm, Inc. is developing patidegib gel for the management of the burden of disease in 
patients with basal cell nevus syndrome (BCNS), a rare genetic disease characterized by the 
development of numerous basal cell carcinomas over a lifetime. This trial will evaluate the 
ability of patidegib gel to shrink BCC tumors in patients with sporadic BCCs as a proof-of-
concept for the mechanism of patidegib on BCCs.  It is hoped that results from this study will 
inform the dose and dosing regimen prior for future trials, thereby maximizing the therapeutic 
potential. 
Patidegib is a semi-synthetic small molecule, and the topical drug product is manufactured with 
generally accepted, safe excipients. Oral patidegib has a good therapeutic efficacy versus locally 
advanced and metastatic BCCs but produces the same types of adverse effects as do other 
systemic HH inhibitors. Topical patidegib has been shown to be stable in the developed gel 
formulation and can be applied to mini-pig skin without irritation.  Topical application of 
patidegib significantly reduces murine BCC tumor size in vivo and reduces GLI1 biomarker 
expression in vitro in human BCC tumor explants. 
If proven to be safe and effective in future clinical trials patidegib gel may offer BCNS patients a 
safe and effective therapy to manage their burden of disease by decreasing the number of 


 
 
 
 
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surgeries they will require over their lifetime.  This trial will evaluate the safety, the tolerability, 
and the ability of four different dosing regimens applied for 3 months to reduce the Hedgehog 
signaling pathway in nodular BCC tumors in patients with sporadic BCCs. 
The goal of the present trial is to evaluate topical patidegib’s safety, tolerability, and effects on 
the size of preexisting sporadic previously untreated nodular BCCs and thereby to establish a 
dose and dosing regimen for future trials in the BCNS orphan population. It is anticipated that 
subsequent trials will focus on reducing the number of facial surgeries required by patients with 
BCNS and not on the “treatment” of individual sporadic BCCs. If the efficacy with topical 
patidegib can approach the level of efficacy of oral HH inhibitors seen in BCNS patients while 
avoiding their systemic side effects, it would represent a major advance for BCNS patients.  
3. STUDY OBJECTIVES 
The primary objectives of the study are to evaluate the following: 
1. 
The safety and tolerability of treatment with patidegib gel 2% or 4% or vehicle applied 
once or twice daily for 12 weeks. 
2. 
The molecular efficacy of treatment as defined by reduction in the hedgehog (HH) 
signaling pathway after treatment with patidegib gel 2% or 4% or vehicle applied twice 
daily for 12 weeks to treatment-targeted BCCs. 
The secondary objective of the study is to evaluate the following: 
1. 
The clinical efficacy of patidegib as defined by the percent decrease in greatest diameter 
of baseline treatment targeted basal cell carcinomas after 12 weeks of treatment. 
Treatment targeted BCCs will be biopsied prior to treatment to confirm the clinical 
diagnosis of nodular BCC. Eligible tumors will have the clinical features of nodular BCC 
and prior to biopsy will be no less than 5 mm or greater than 20 mm in longest diameter 
on the face (excluding the nose and periorbital skin) and no less than 9 mm or greater 
than 20 mm in longest diameter at sites other than the face. 
Exploratory objectives: 
1. 
To evaluate the utility of an investigator static global tumor assessment (ISGTA) in 
assessing the proportion of baseline treatment-targeted BCCs that are evaluated as being 
clear or almost clear. 
2. 
To evaluate the utility of blinded assessment of change in BCC size using photographs. 


 
 
 
 
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4. STUDY DESIGN 
4.1 
Overall Study Design 
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to assess 
the efficacy and safety of patidegib gel 2% and 4% in comparison with vehicle applied once or 
twice daily. The population of subjects will be enrolled from the 
. 
Approximately 36 subjects who meet the study entry criteria will be randomized to one of four 
sequential cohorts. Each cohort will have 6 subjects randomized to active drug and 3 randomized 
to vehicle. The four sequential cohorts will be: 
• Cohort 1: patidegib gel 2% or vehicle applied once daily,  
• Cohort 2: patidegib gel 4% or vehicle applied once daily,  
• Cohort 3: patidegib gel 2% or vehicle applied twice daily, and  
• Cohort 4: patidegib gel 4% or vehicle applied twice daily.  
As soon as one cohort has been enrolled the next cohort will be recruited. The study drug will be 
applied topically to the treatment-targeted nodular BCCs once or twice daily for 12 weeks of 
treatment. 
Upon completion of the 12-week treatment period, all subjects will be asked to return to the 
investigational center for final evaluation. At the Week 12 visit the treatment site(s) will be 
excised. The area excised should at a minimum include the original diameter of the tumor. This 
means that if the tumor has shrunk in size the entire original area of the BCC will be excised, not 
just the visually apparent residual tumor.  During the study, subjects will be allowed to use 
moisturizers and emollients and sunscreen. The Investigator will assess the areas affected at each 
visit.  
The treatment-targeted BCC(s) will be identified by the Investigator at the Baseline visit and will 
be circled in ink at Baseline, Weeks 6 and 12, and photographed, and measured at all study visits 
(Baseline, Weeks 2, 6, 8, 10, and 12).  
Biomarkers:  On each subject one or two baseline previously untreated nodular BCCs designated 
as a treatment-targeted tumor will be biopsied and at the end of 12 weeks of treatment the 
treatment site(s) will be excised. Both samples will be evaluated histologically and for 
determination of GLI1 mRNA levels.  
Blood samples for complete blood count and serum chemistry and urine for urinalysis will be 
collected from subjects at Screening, Week 6, and Week 12.  
PPD


 
 
 
 
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Subjects who terminate study participation early will be asked to complete all Week 12 
assessments, as appropriate, prior to commencement of any alternative therapy for BCC (if 
possible). Subjects who discontinue from the study during the treatment period will not be 
replaced. 
If signs or symptoms develop in the treatment areas during the treatment period that restrict daily 
activities or make continued application of the study drug difficult due to discomfort, the 
Investigator may instruct the subject to interrupt use of the study drug temporarily and to resume 
application of the study drug once the signs/symptoms have subsided. The Investigator should 
try to minimize study drug interruptions; and if needed, make best efforts to limit a “drug 
holiday” to no more than 7 days. If the study drug interruption does exceed 4 consecutive days, 
the Investigator should consult with the Medical Monitor to determine a course of action. If the 
study drug is interrupted, discontinued, or a concomitant medication is used to treat a sign or 
symptom, an AE shall be recorded. 
Subjects who discontinue participation in the study due to clinically significant laboratory 
abnormalities or AEs will be asked to complete all Week 12 evaluations. Any subject who has an 
AE during the treatment period will be monitored by the Investigator until resolution (return to 
normal or to the baseline state) or stabilization, as determined by the Investigator. 
In addition, application of study drug may be delayed or halted at any time if ongoing safety data 
evaluations raise concern for subject safety. If the subject participation is suspended, all of the 
subject’s safety data will be reviewed by the Medical Monitor in conjunction with the 
Investigator to determine course of action. 
4.1.1 
Schedule of Visits and Assessments 
The schedule of assessments can be found in Section 5.1 of the protocol. 
4.1.2 
Method of Assigning Subjects to Treatment Groups 
This is a double-blinded study, in which the identity of the study drug will be unknown to 
Investigator and subjects, as well as to all individuals closely associated with the study. 
Subjects will be enrolled into 1 of the 4 sequential study drug groups of 9 subjects with 6 being 
randomized to active and 3 to vehicle within each group. The sequential groups will be patidegib 
or vehicle gel 2% applied once daily, patidegib or vehicle gel 4% applied once daily, patidegib or 
vehicle gel 2% applied twice daily, patidegib or vehicle gel 4% applied twice daily. Each 
screened subject will be assigned a unique 5-digit study subject number assigned by the 
investigational center, which will consist of the 2 digit investigational center number and the 3 
digit chronological screening order number, starting with 001 (e.g., 01001, 01002). The study 


 
 
 
 
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drug kit will be assigned to subjects based on a randomization code, and kits will be dispensed to 
the subjects at Baseline in the order that they are enrolled by taking the lowest numbered kit 
available in inventory at the investigational site. A study drug accountability log will document 
the inventory and dispensing of study drug at the investigational center. 
4.1.3 
Blinding 
The study drugs will be packaged and labeled identically, and the study drug kits will be 
numbered sequentially and dispensed randomly to the subjects on the study. Study drug supplies 
will be distributed to the investigational center to maintain the randomization ratio. 
As a double-blinded study, the Investigators, the site staff, PellePharm, and the Clinical 
Monitor(s) will not be aware of the treatment assigned to the individual study subjects. 
Delegated staff members at the investigational center will dispense the study drugs and will 
collect and weigh all used and unused study drug tubes as scheduled. 
The treatment assignments for all enrolled subjects will be completely unblinded only after the 
conclusion of the treatment phase of the study. However, after Cohorts 1 and 2 have complete 
biopsy results, and again after Cohort 3 has complete biopsy results, the sponsor and the CRO 
will be unblinded in order to assess limited safety and efficacy results. 
5. EFFICACY AND SAFETY ENDPOINTS 
5.1 
Efficacy Endpoints 
The efficacy endpoints are intended to compare once and twice daily application of 2% patidegib 
gel, 4% patidegib gel, and vehicle gel. Efficacy assessments will be summarized descriptively by 
treatment group and visit. P-values will be provided for descriptive purposes only. 
5.1.1 
Primary Efficacy Endpoint 
The primary efficacy endpoint is change in GLI1 mRNA levels in drug-treated versus vehicle-
treated tumors at Week 12. 
5.1.2 
Secondary Efficacy Endpoint 
The secondary efficacy endpoint is the decrease in tumor size defined in two ways: 
1. Percent of baseline treatment targeted BCCs with Complete or Partial Response at Week 
12 as assessed by blinded photographic review1 where Complete Response is determined 
when there is no longer any visible evidence of a lesion consistent with BCC at the site, 
Partial Response is determined when although a BCC still remains at this site, it has 
demonstrated a visible decrease in size compared with baseline, and No Response is 


 
 
 
 
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determined when the BCC has not demonstrated any visible decrease in size compared 
with baseline, and; 
2. Percent decrease in the greatest diameter of baseline treatment targeted BCCs at Week 
12. 
5.1.3 
Exploratory Efficacy Endpoints 
The first exploratory endpoint is the proportion of baseline treatment-targeted BCCs that are 
evaluated as being clear or almost clear in the investigator static global tumor assessment 
(ISGTA) assessment. 
The second exploratory endpoint is the proportion of baseline treated tumors that are smaller 
based on blinded photographic review. Tumor area will also be assessed at each study visit. 
From tumor area, a ‘Yes/No’ for whether the tumor is smaller will be derived such that if area at 
visit is less than area at baseline this is ‘Yes’. 
5.2 
Safety Endpoints 
Safety will be assessed through adverse events, dermal safety and tolerability, safety laboratory 
values and vital signs measurements, and pregnancy tests. 
6. STATISTICAL AND ANALYTICAL PLANS 
6.1 
General Methodology 
All statistical processing will be performed using Statistical Analysis System (SAS®) unless 
otherwise stated. No interim analyses are planned. If determined appropriate by the Sponsor, 
limited safety and efficacy interim analysis on tumor shrinkage and biomarkers may be 
performed. 
Formal inferential testing will not be performed, however p-values will be provided for 
descriptive purposes. Descriptive statistics will be used to provide an overview of the efficacy 
and safety results. P-values which are supplied are for descriptive purposes only. For categorical 
parameters, the number and percentage of subjects in each category will be presented. For 
continuous parameters, descriptive statistics will include n (number of subjects), mean, standard 
deviation (SD), median, minimum (min), and maximum (max).  For by treatment group 
summaries the vehicle gel group from each cohort will be combined into a single group. 
The last observation carried forward method (LOCF) will be used to impute missing secondary 
and exploratory efficacy data (i.e., the last available on-therapy observation for a subject will be 
used to estimate subsequent missing data points). Analyses will also be carried out on an ‘as 


 
 
 
 
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observed’ basis without imputation. No imputations will be made for missing primary endpoint 
data or safety data. 
6.1.1 
Statistical Analysis 
All analyses will be performed by QST using SAS® Version 9.3 or later.  All summary tables 
and data listings will be prepared utilizing SAS® software. 
The standard operating procedures (SOPs) of QST will be followed in the creation and quality 
control of all data displays and analyses. 
6.1.2 
Baseline Definition 
Baseline is defined as the last non-missing assessment prior to first dose of study drug.   
6.1.3 
Visit Windowing 
Data will be summarized based on nominal visit indications with the exception of data captured 
at early termination and unscheduled visits. Data from early termination and unscheduled visits 
will be summarized based on mapped visit values.  The analysis windows for early termination 
and unscheduled visits are presented in the following table.  
Analysis Windows for Efficacy and Safety Assessments 
Scheduled Visit 
Target Study Day 
Window (Days) 
Week 2 
15 
13 to 17 
Week 6 
43 
40 to 46 
Week 8 
57 
54 to 60 
Week 10 
71 
68 to 74 
Week 12 
85 
82 to 88 
Week 14 
99 
92 to 106 
 
Data collected at early termination and unscheduled visits prior to study day 13 will not be 
analyzed, with the exception of those identified as Baseline values. Data collected at early 
termination and unscheduled visits after study day 106 will not be included in analyses. 
The definition for the study day included in each study window is defined as below: 
Study Day Prior to Day 1 = Visit Date – Baseline Date 
 
Study Day On or After Day 1 = Visit Date – Baseline Date + 1. 


 
 
 
 
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If an assessment’s mapped visit is a visit at which the subject has data from a scheduled visit 
present, or if no analyses are planned for the assessment at the mapped visit, or if the data maps 
to a time period not covered by windowing, the data collected at the early termination or 
unscheduled visit will not be included in analyses.   
In the event of multiple values from unscheduled or early termination assessments within an 
analysis window, the value closest to the scheduled visit target study day will be used for 
analyses (assuming there were no results from the scheduled visit present).  If two values tie as 
closest to the time point (for example, one value is before and the other value is after the time 
point), then the later value will be selected.  
Data collected at all visits will be included in the data listings with visit presented as reported by 
the site. 
6.1.4 
Adjustments for Covariates 
Treatment group as factor and baseline values as covariate will be included in the primary and 
secondary endpoints analyses.   
6.1.5 
Handling of Dropouts or Missing Data 
If a partial date is recorded for an adverse event start or end date, the following procedure will be 
followed. Other missing safety data dates will not be imputed.  
If a partial date is reported where the day is missing, then the day will be imputed as the first day 
of the month unless the month is the same month as the first dose in which case the day will be 
that of first dose with the month and year remaining the same. If a partial date is reported where 
the month is missing, then the month will be imputed to January unless the year is the same year 
as the first dose in which case the month will be that of first dose with the year remaining the 
same. If a partial date where both the day and month is missing, follow details as stated 
previously.  
Missing data post-Baseline through Week 12 will be imputed using last observation carried 
forward (LOCF) for the secondary endpoint of tumor size as assessed by the investigator.  
Specifically, if a single diameter is missing then that SEB’s previous measurement will be 
carried forward. The analysis will also be performed without imputation of missing data. For the 
secondary endpoint of GLI1, no missing data will be imputed.  
6.1.6 
Interim Analyses and Data Monitoring 
As stated in the protocol, if determined appropriate by the Sponsor, limited safety and efficacy 
interim analysis on tumor shrinkage and biomarkers may be performed. 


 
 
 
 
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Once biomarker results are available for subjects in Cohorts 1 and 2, and again after Cohort 3 
subjects have biomarker results, the database will be used to report on limited safety and efficacy 
results. The sponsor and the CRO (QST) will be unblinded at this time. Site staff will remain 
blinded. 
6.1.7 
Multicenter Studies 
The study will be conducted at one metasite in the United States with the intention of pooling the 
results for analysis. 
6.1.8 
Multiple Comparisons/Multiplicity 
Not applicable. Inferential tests will not be performed.  
6.1.9 
Use of an Efficacy Subset of Subjects 
A Per Protocol population is defined below. 
6.1.10 
Active-Control Studies Intended to Show Equivalence 
Not applicable to this study. 
6.1.11 
Examination of Subgroups 
Not applicable to this study. 
6.2 
Disposition of Subjects 
The number of subjects included in each analysis population (ITT, Safety, and Per Protocol) will 
be summarized by treatment group.  The number of subjects randomized, completed, and 
discontinued (including the reasons for discontinuation) will be summarized for each treatment 
group. 
Reasons for exclusion from study populations will be included in a by-subject listing. 
6.3 
Protocol Deviations 
All protocol deviations will be reported to PellePharm and recorded throughout the study.  A 
listing of protocol deviations by treatment by subject will be included in the final study report.   


 
 
 
 
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6.4 
Data Sets Analyzed 
6.4.1 
Intent-to-Treat (ITT) Population 
All subjects who are randomized and dispensed study drug will be included in the ITT analysis 
set. Subjects will be analyzed according to the treatment group they were randomized.  All 
efficacy analyses will be presented using the ITT population and the Per Protocol population. 
6.4.2 
Safety Population 
All subjects who are randomized, receive at least 1 confirmed dose of study drug and have at 
least one post-baseline safety assessment will be included in the safety analysis set. All safety 
analyses will be according to the treatment actually received. 
6.4.3 
Per Protocol (PP) Population 
All subjects in the ITT population who complete the Week 12 evaluation without any significant 
protocol violations will be included in the PP population and analyzed according to the treatment 
group they received.  The PP population will include subjects in the safety population who do 
not meet any of the following criteria: 
• Violated the inclusion/exclusion criteria; 
• Have taken any interfering concomitant medication; 
• Did not attend the Week 12 visit; 
• Have not been compliant with the dosing regimen, between 80% to 120% 
• Had samples with insufficient BCC  
• Had tumors where the diameter was incorrectly measured based on review of 
photographs 
Subjects that discontinue from the study due to an adverse event related to study treatment or 
documented lack of treatment effect will be included in the PP population.  Prior to breaking the 
blind, other additional criteria may be added to the list to accommodate for unforeseen events 
that occurred during the conduct of the trial that result in noteworthy study protocol violations.  
Efficacy analyses performed on a by-tumor basis will include tumors from subjects who are not 
excluded for any of the above reasons. 
All efficacy analyses will be performed on the ITT population as well as on the PP population. 


 
 
 
 
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6.5 
Demographic and Other Baseline Characteristics 
All baseline summaries will be done on the ITT population. 
Sex, race, and ethnicity will be summarized by counts and percentages.  Age, height (cm), and 
weight (kg) will be summarized with descriptive statistics.   
Medical histories will be coded using the MedDRA dictionary and presented in a by-subject 
listing. 
6.6 
Prior and Concomitant Medications 
Concomitant therapies will be coded to preferred name and Anatomical Therapeutic Chemical 
(ATC) classification of ingredients using the World Health Organization (WHO) Drug dictionary 
(WHO-DDE). 
A by-subject listing of all prior and concomitant medications will be presented. Medications that 
start prior to first dose but continue after the first dose will be considered a concomitant 
medication. Medications that are unable to be classified as either prior or concomitant due to 
missing data will not be considered a concomitant medication.  
6.7 
Analysis of Efficacy 
All efficacy analysis will be done on both the ITT and PP populations. 
6.7.1 
Primary Efficacy Analysis 
The primary endpoint of change in GLI1 mRNA levels will be evaluated with an analysis of 
covariance (ANCOVA) with treatment group as factor and baseline value as covariate.  Analysis 
is on a by tumor level. Pairwise comparisons as well as any treated (combining all patidegib 
groups) versus vehicle will be performed using contrasts within the ANCOVA. 
Additionally, if number of results is sufficient, a t-test will be done to perform pairwise 
comparisons of change in GLI1 mRNA levels of each treatment group with vehicle (and 
combined patidegib versus vehicle).  If assumptions required for a t-test are not met, then a Mann 
Whitney U test will be performed. 
Data will be summarized by visit. Both change from baseline and percent change from baseline 
will be presented. 
This analysis will be done for 2 regions (1) BCC and (2) BCC + tumor-associated stroma. 
No imputation on GLI1 data will be performed. 


 
 
 
 
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6.7.2 
Secondary Efficacy Analysis 
The first secondary endpoint, the proportion of treatment targeted BCCs with Complete or Partial 
Response as determined by blinded photographic review will be evaluated using a 2-sided 
Fisher’s Exact test. This analysis will be performed on all available data with no imputation of 
missing data.  Both pairwise comparisons between treatment groups and all treated (combining 
all patidegib groups) versus vehicle will be done. 
The second secondary endpoint, the greatest diameter of all treatment targeted BCCs, will be 
evaluated similarly to the primary endpoint.This analysis will be performed using LOCF 
imputation as well as ‘as observed’ (i.e. no imputation of missing data).  
6.7.3 
Exploratory Efficacy Analysis 
The exploratory endpoint of investigator static global tumor assessment (ISGTA) will be 
summarized by visit on a by tumor level. Proportion with success as defined by Clear/Almost 
Clear will be provided.  Results will be analyzed with a Cochran-Mantel-Haenszel (CMH) test.  
Tumor area will be evaluated with an analysis of covariance (ANCOVA) with treatment group as 
factor and baseline value as covariate.  Analysis is on a by tumor level. Pairwise comparisons as 
well as any treated (combining all patidegib groups) versus vehicle will be performed using 
contrasts within the ANCOVA. 
The proportion of baseline treated tumors are smaller at each study visit based on photographic 
review will be summarized by treatment group. Results will be analyzed between groups and all 
treated (combining all patidegib groups) versus vehicle with a CMH test. Tumor area will be 
summarized by visit.  
6.8 
Safety Evaluation 
6.8.1 
Extent of Exposure and Compliance 
The extent of exposure to study drug in each treatment group will be summarized by days with 
applications, total number of applications, and number of missed applications.  This data will 
also be provided in a listing.  
Percent compliance will be calculated for each subject as follows: 
(a) Expected Number of Applications = (Date of Last Application – Date of First 
Application + 1) * Number of Expected Applications per day, accounting for possible 
single applications on First and/or Last Day 


 
 
 
 
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(b) Total Number of Applications = Expected Number of Applications – Total Number of 
Missed Applications 
Compliance = (b) / (a) * 100 
6.8.2 
Adverse Events 
All AEs that occur during the study will be recorded and classified on the basis of Medical 
Dictionary for Regulatory Activities (MedDRA) terminology. Treatment-emergent Aes (TEAEs) 
are defined as Aes with an onset on or after the date of the first study drug application. Adverse 
events noted prior to the first study drug administration that worsen after Baseline will also be 
reported as Aes and included in the summaries.  
Treatment-emergent Aes will be summarized by treatment group, the number of subjects 
reporting a TEAE, SOC, preferred term, severity, relationship to study drug (causality), and 
seriousness. When summarizing Aes by severity and relationship, each subject will be counted 
once within a system organ class or a preferred term by using the event with the highest severity 
and greatest relationship within each classification. 
If relationship to study drug is reported as unknown, possible, probable or definite, then this is 
defined as related.  If relationship to study drug is reported as unlikely or not related, then this is 
defined as unrelated. 
Serious Aes will be summarized by treatment group, severity and relationship to study drug. 
Listings will be presented for all adverse events as well as for serious adverse events and adverse 
events leading to study drug withdrawal. 
6.8.3 
Clinical Laboratory Evaluation 
Laboratory test results will be summarized with descriptive statistics at Baseline and Week 12. 
Additionally, shifts from Baseline to Week 12 in laboratory test results based on normal ranges 
will be summarized with frequency counts and percentages. Individual laboratory test results will 
be presented in a by-subject listing.  
6.8.4 
Other Observations Related to Safety 
6.8.4.1 
Dermal Safety and Tolerability 
The frequency of dermal safety and tolerability assessments including pain/burning, pruritus, 
erythema, edema, and scabbing/crusting will be summarized by treatment group and visit.  All 
dermal safety and tolerability results will be listed. 


 
 
 
 
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6.8.4.2 
Systemic Symptoms 
Systemic symptoms will be summarized by treatment group and visit.  Additionally, results will 
be listed. 
6.8.4.3 
Vital Signs 
Vital signs will be summarized with descriptive statistics at Baseline and each applicable study 
visit. Changes from Baseline in vital signs will also be summarized. 
Vital sign measurements will be listed. 
6.8.4.4 
Physical Examination 
Physical examination data will be presented in a by-subject listing.  
7. DETERMINATION OF SAMPLE SIZE 
This is a dose ranging study. 
8. CHANGES IN THE PLANNED ANALYSES 
The protocol defines the secondary objective as the clinical efficacy of patidegib as defined by 
the percent decrease in greatest diameter of baseline treatment targeted basal cell carcinomas 
after 12 weeks of treatment. The secondary objective will now also include the clinical efficacy 
of patidegib as defined by the proportion of Complete or Partial Response in treatment targeted 
BCCs after 12 weeks of treatment as defined by blinded photographic review.References 
9. REFERENCES 
1. Skvara H, Kalthoff F, Meingassner JG, Wolff-Winiski B, Aschauer H, Kelleher JF, Wu 
X, Pan S, Mickel L, Schuster C, Stary G, Jalili A, David OJ, Emotte C, Antunes AMC, 
Rose K, Decker J, Carlson I, Gardner H, Stuetz A, Bertolino AP, Stingl G, De Rie MA. 
Topical Treatment of Basal Cell Carcinomas in Nevoid Basal Cell Carcinoma Syndrome 
with a Smoothened Inhibitor., J Invest Dermatology 2011. 
 
 


 
 
 
 
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10. INDEX OF PLANNED END-OF-TEXT TABLES AND FIGURES 
Table 14.1.1: Summary of Subject Disposition (All Subjects) ......................................................23 
Table 14.1.1.1: Summary of Subject Demographics and Baseline Characteristics (Intent-to-Treat Population) 
25 
Table 14.2.1.1: Analysis of the Primary Efficacy Endpoint: Percent Change in GLI1 mRNA Levels at Week 12 (Intent-to-Treat Population) 
27 
Table 14.2.1.2: Analysis of the Primary Efficacy Endpoint: Percent Change in GLI1 mRNA Levels at Week 12 (Per Protocol Population) 
29 
Table 14.2.2.1.1: Analysis of the Secondary Efficacy Endpoint: Proportion of CR/PR Response at Week 12  (Intent-to-Treat Population) 
30 
Table 14.2.2.1.2: Analysis of the Secondary Efficacy Endpoint: Proportion of CR/PR Response at Week 12 (Per Protocol Population) 30 
Table 14.2.2.2.1: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (LOCF) (Intent-
to-Treat Population) ..............................................................................31 
Table 14.2.2.2.2: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (LOCF) (Per 
Protocol Population) .............................................................................32 
Table 14.2.2.2.3: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (As Observed) 
(Intent-to-Treat Population) ..................................................................32 
Table 14.2.2.2.4: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (As Observed) 
(Per Protocol Population) ......................................................................32 
Table 14.2.2.2.5: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (LOCF) (Intent-to-Treat Population) 33 
Table 14.2.2.2.6: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (LOCF) (Per Protocol Population) 
34 
Table 14.2.2.2.7: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (as Observed) (Intent-to-Treat Population) 
34 
Table 14.2.2.2.8: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (as Observed) (Per Protocol Population) 
34 
Table 14.2.3.1: Analysis of the Exploratory Efficacy Endpoint: ISGTA at Weeks 2, 6, 8, 10 and 12  (Intent-to-Treat Population) 
35 
Table 14.2.3.2: Analysis of the Exploratory Efficacy Endpoint: ISGTA at Weeks 2, 6, 8, 10 and 12  (Per Protocol Population) 36 
Table 14.2.4.1: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review (Intent-to-Treat Population)
 ...............................................................................................................37 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 22 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.4.2: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review (Per Protocol Population)
 ...............................................................................................................38 
Table 14.3.1.1: Summary of Extent of Exposure and Compliance (Safety Population) ...............39 
Table 14.2.4.4: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review (Per Protocol Population)
 ...............................................................................................................40 
Table 14.3.1.2.1: Summary of Dermal Safety and Tolerability (Safety Population) ....................41 
Table 14.3.1.2.2: Summary of Systemic Symptoms (Safety Population) .....................................42 
Table 14.3.2.1.1: Summary of Treatment-Emergent Adverse Event Characteristics (Safety Population) 43 
Table 14.3.2.1.2: Summary of Treatment-Emergent Adverse Events by MedDRA System Organ Class and Preferred Term (Safety Population) 
45 
Table 14.3.2.1.3: Summary of Treatment-Emergent Adverse Events by Severity (Safety Population) 
46 
Table 14.3.2.1.4: Summary of Treatment-Emergent Adverse Events by Relationship (Safety Population) 47 
Table 14.3.2.2.1: Summary of Treatment-Emergent Serious Adverse Event Characteristics (Safety Population) 48 
Table 14.3.2.2.2: Summary of Treatment-Emergent Serious Adverse Events by MedDRA System Organ Class and Preferred Term (Safety Population)
 ...............................................................................................................48 
Table 14.3.2.2.3: Summary of Treatment-Emergent Serious Adverse Events by Severity (Safety Population) 
48 
Table 14.3.2.2.4: Summary of Treatment-Emergent Serious Adverse Events by Relationship (Safety Population) 
48 
Table 14.3.3.1: Summary of Chemistry Laboratory Results (Safety Population) .........................49 
Table 14.3.3.2: Summary of Hematology Laboratory Results (Safety Population) ......................50 
Table 14.3.3.3: Summary of Quantitative Urinalysis Laboratory Results (Safety Population) ....50 
Table 14.3.3.4: Summary of Categorical Urinalysis Laboratory Results (Safety Population) ......51 
Table 14.3.3.5: Summary of Chemistry Laboratory Results - Shift Table (Safety Population) ....52 
Table 14.3.3.6: Summary of Hematology Laboratory Results - Shift Table (Safety Population) .53 
Table 14.3.3.7: Summary of Quantitative Urinalysis Laboratory Results - Shift Table (Safety Population) 
53 
Table 14.3.4.1: Summary of Vital Signs (Safety Population) .......................................................54 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 23 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Table 14.1.1: Summary of Subject Disposition 
(All Subjects) 
 
  
 
Patidegib Gel 2% 
QD 
Patidegib Gel 2% 
BID 
Patidegib Gel 4% 
QD 
Patidegib Gel 4% 
BID 
Vehicle Gel 
Number of Subjects Randomized 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
 
 
 
 
 
Number of Subjects Included in the ITT 
Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Number of Subjects Excluded from the 
ITT Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Number of Subjects Included in the 
Safety Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Number of Subjects Excluded from the 
Safety Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Number of Subjects Included in the Per 
Protocol Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Number of Subjects Excluded from the 
Per Protocol Population 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Completed Study 
 
 
 
 
 
 Yes 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 No 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
   Reason for Discontinuation 
 
 
 
 
 
   Adverse Event 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Lost to Follow-Up 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Pregnancy 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Lack of Efficacy 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Protocol Violation 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Disease Progression 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Withdrawal by Subject 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Other 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 24 
Protocol Number: 926-202 
28Sep2017 
 
 
  
 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 25 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Table 14.1.1.1: Summary of Subject Demographics and Baseline Characteristics 
(Intent-to-Treat Population) 
(Page 1 of 2) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Age (years) 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Sex 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
Male 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Female 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Ethnicity 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
Hispanic or Latino 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Not Hispanic or Latino 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Race 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
American Indian or Alaska Native 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Asian 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Black or African American 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Native Hawaiian or Other Pacific 
Islander 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
White 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
Multiple/Othera 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
  
 
 a See Listing xx for a complete list of all other races. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 26 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.1.1.1: Summary of Subject Demographics and Baseline Characteristics 
(Intent-to-Treat Population) 
(Page 2 of 2) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Height (cm) 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Weight (kg) 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
  
 
 a See Listing xx for a complete list of all other races. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 27 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.1.1: Analysis of the Primary Efficacy Endpoint: Percent Change in GLI1 mRNA Levels at Week 12 
(Intent-to-Treat Population) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
1:  BCC region H-Score 
 
 
 
 
 
Baseline  
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Week 12 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Change from Baselinea 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Percent Change from Baselinea 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
    At least 40% Decrease from Baseline 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 28 
Protocol Number: 926-202 
28Sep2017 
 
 
P-value versus Vehicle Gelb 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDb 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDb 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDb 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gel 
 
 
 
 
x.xxx 
 
 
 
 
 
 
P-value versus Vehicle Gelc 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDc 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDc 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDc 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gel 
 
 
 
 
x.xxx 
 
Repeat Above for 1:BCC + tumor-associated stroma region H-Score 
  
 
a Change from Baseline calculated as (Week 12 – Baseline). Percent Change from Baseline calculated as (Week 12 - Baseline) / Baseline * 100. 
b P-value of pairwise comparison  using ANCOVA with a factor of treatment group and using Baseline value as a covariate. 
c  P-value of pairwise comparison using t-test (or Mann Whitney U if the case). 
Note: Missing values are not imputed; all available data is summarized. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 29 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
Table 14.2.1.2: Analysis of the Primary Efficacy Endpoint: Percent Change in GLI1 mRNA Levels at Week 12 (Per Protocol Population) 
As above Table 14.2.1.1 but for the Per Protocol Population 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 30 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.2.1.1: Analysis of the Secondary Efficacy Endpoint: Proportion of CR/PR Response at Week 12  
(Intent-to-Treat Population) 
 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
Proportion of CR/PR Response at 
Week 12a 
 
 
 
 
 
 
Number of tumors 
     xx 
     xx 
     xx 
     xx 
     xx 
 
CR / PR 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
 
Non Response 
   xx.xx 
   xx.xx 
   xx.xx 
   xx.xx 
   xx.xx 
 
 
 
 
 
 
P-value versus Vehicle Gelc 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDc 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDc 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDc 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gel 
 
 
 
 
x.xxx 
  
 
a As assessed by blinded photographic review. 
b P-value of pairwise comparison  using 2-sided Fisher’s Exact test. 
Note: Missing values are not imputed. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
Table 14.2.2.1.2: Analysis of the Secondary Efficacy Endpoint: Proportion of CR/PR Response at Week 12 (Per Protocol Population) 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 31 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Table 14.2.2.2.1: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (LOCF) 
(Intent-to-Treat Population) 
 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
Percent Decrease in Greatest Diameter of 
Treatment Targeted BCCs at 
Week 12a 
 
 
 
 
 
 
Number of tumors 
     xx 
     xx 
     xx 
     xx 
     xx 
 
Mean 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
 
SD 
   xx.xx 
   xx.xx 
   xx.xx 
   xx.xx 
   xx.xx 
 
Median 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
    xx.x 
 
Min. to Max. 
xx to xx 
xx to xx 
xx to xx 
xx to xx 
xx to xx 
 
 
 
 
 
 
P-value versus Vehicle Gelb 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDb 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDb 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDb 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gel 
 
 
 
 
x.xxx 
 
 
 
 
 
 
P-value versus Vehicle Gelc 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDc 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDc 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDc 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gel 
 
 
 
 
x.xxx 
  
 
a Percent decrease calculated as (Baseline - Week 12) / Baseline * 100. 
b P-value of pairwise comparison  using ANCOVA with treatment group as a factor and Baseline value as a covariate. 
c  P-value of pairwise comparison using t-test (or Mann Whitney U if the case). 
Note: Missing values are imputed using LOCF. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 32 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.2.2.2: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (LOCF) 
(Per Protocol Population) 
As above Table 14.2.2.1 but for Per Protocol population. 
 
 
 
 
Table 14.2.2.2.3: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (As Observed) 
(Intent-to-Treat Population) 
As above Table 14.2.2.1 but with no imputation and the following Note: Missing values are not imputed. 
 
 
Table 14.2.2.2.4: Analysis of the Secondary Efficacy Endpoint: Percent Decrease in Baseline Treatment Targeted Tumor Size at Week 12 (As Observed) 
(Per Protocol Population) 
As above Table 14.2.2.2 but for Per Protocol population. 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 33 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.2.2.5: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (LOCF) 
(Intent-to-Treat Population) 
(Page 1 of x) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
Baseline 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Week 2 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Percent Change from Baselinea 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
 
 
 
 
 
 
Continue for Weeks 6, 8 and 10 
 
 
 
 
 
  
 
a Percent change calculated as (Follow-up - Baseline) / Baseline * 100. 
Note: Missing values imputed using LOCF. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 34 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.2.2.6: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (LOCF) 
(Per Protocol Population) 
As Table 14.2.2.5 but Per Protocol. 
 
Table 14.2.2.2.7: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (as Observed) 
(Intent-to-Treat Population) 
As above Table 14.2.2.5 but with no imputation.  Replace Note with Note: Missing values are not imputed. 
 
 
 
Table 14.2.2.2.8: Summary of Baseline Treatment Targeted Tumor Size at Weeks 2, 6, 8 and 10 (as Observed) 
(Per Protocol Population) 
As Table 14.2.2.7 but Per Protocol. 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 35 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
Table 14.2.3.1: Analysis of the Exploratory Efficacy Endpoint: ISGTA at Weeks 2, 6, 8, 10 and 12  
(Intent-to-Treat Population) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Baseline 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
0 – Clear 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
1 – Almost clear 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
2 – Minimal residual tumor 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
3 – Clearly visible tumor 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Week 2 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
0 – Clear 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
1 – Almost clear 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
2 – Minimal residual tumor 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
3 – Clearly visible tumor 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
  Treatment Successa 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
  Success 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
  Failure 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
P-value versus Vehicle Gelb 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDb 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDb 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDb 
 
 
 
x.xxx 
 
 
 
 
 
 
 
Repeat for Weeks 6, 8 ,10 ,12 
 
 
 
 
 
  
 
 a Success is defined as Clear or Almost clear. 
b P-value from Cochran-Mantel-Haenszel (CMH) test. 
Note: Missing values imputed using LOCF. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 36 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.3.2: Analysis of the Exploratory Efficacy Endpoint: ISGTA at Weeks 2, 6, 8, 10 and 12  
(Per Protocol Population) 
As Table 14.2.3.1 for Per Protocol 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 37 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
 
 
 
 
Table 14.2.4.1: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review 
(Intent-to-Treat Population) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Baseline 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Mid-Study 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Percent Change from Baselinea 
 
 
 
 
 
 
Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
     Smaller 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
     Not smaller 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
P-value versus Vehicle Gelb 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDb 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDb 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDb 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gelb 
 
 
 
 
x.xxx 
 
 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 38 
Protocol Number: 926-202 
28Sep2017 
 
 
P-value versus Vehicle Gelc 
x.xxx 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% QDc 
 
x.xxx 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 2% BIDc 
 
 
x.xxx 
x.xxx 
 
P-value versus Patidegib Gel 4% QDc 
 
 
 
x.xxx 
 
P-value All Patidegib vs Vehicle Gelc 
 
 
 
 
x.xxx 
 
 
 
 
 
 
Repeat for Week 12. 
 
 
 
 
 
  
 
 a Defined as demonstrating no residual BCC. 
b P-value of pairwise comparison  using ANCOVA with treatment group as a factor and Baseline value as a covariate. 
c P-value from Cochran-Mantel-Haenszel (CMH) test. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 
 
Table 14.2.4.2: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review (Per Protocol Population) 
As Table 14.2.4.1 but using Per Protocol population.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 39 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
 
 
 
Table 14.3.1.1: Summary of Extent of Exposure and Compliance 
(Safety Population) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Number of Days oaf Exposure 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Number of Applicationsa 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Number of Missed Applications 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Compliance (%) 
 
 
 
 
 
 N 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
  
 
 a The total number of applications was calculated form the first date of treatment and the last date of treatment minus the missed applications. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 40 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.2.4.4: Analysis of the Exploratory Efficacy Endpoint: Area of Baseline Treated Tumors based on Photographic Review (Per Protocol Population) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 41 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Table 14.3.1.2.1: Summary of Dermal Safety and Tolerability 
(Safety Population) 
(Page 1 of x) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Pain / Burning 
 
 
 
 
 
Baseline 
 
 
 
 
 
   Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
   None 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Week 2 
 
 
 
 
 
   Number of tumors 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
   None 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Continue for Weeks 6, 8, 10, 12 
 
 
 
 
 
 
 
 
 
 
 
  Repeat for Pruritus, Erythema, Edema, Scabbing / Crusting 
  
 
Note: Local safety and tolerability is reported by the subject as the greatest intensity since the last visit or within 24 hours at Baseline. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 42 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.1.2.2: Summary of Systemic Symptoms 
(Safety Population) 
(Page 1 of x) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Fatigue 
 
 
 
 
 
Baseline 
 
 
 
 
 
   n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
   Yes 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   No 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Week 2 
 
 
 
 
 
   n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
   Yes 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   No 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Week 6 
 
 
 
 
 
   n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
   Yes 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   No 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Continue for Weeks 8, 10, 12 
 
 
 
 
 
 
 
 
 
 
 
  Repeat for Loss or Change in Taste, Nausea, Vomiting, Diarrhea, Muscle Spasms or Cramps, Changes of Quality or Quantity of Hair in Treated Areas, 
Change in Frequency of Shaving, Other Systemic Sign or Symptoms, Other 
  
 
 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 43 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
Table 14.3.2.1.1: Summary of Treatment-Emergent Adverse Event Characteristics 
(Safety Population) 
(Page 1 of 2) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
Number (%) of Subjects Reporting At 
Least One Adverse Event 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Number (%) of Subjects Reporting At 
Least One Serious Adverse Event 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Number (%) of Subjects who Died 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Number (%) of Subjects who 
Discontinued Study Medication due to 
Adverse Event 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Maximum Severity 
 
 
 
 
 
 Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Strongest Relationship to Study 
Medication 
 
 
 
 
 
 Not Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
  
 
Note: Treatment-emergent adverse events are those with an onset after use of study medication. Not Related includes Not Related and Unlikely. Related 
includes Possible, Probable or Definite. Any unknown relationship is defined as Related. A subject is counted once according to maximum severity and 
relationship. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 44 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.2.1.1: Summary of Treatment-Emergent Adverse Event Characteristics 
(Safety Population) 
(Page 2 of 2) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
Maximum Severity within Relationship to 
Study Medication 
 
 
 
 
 
 Not Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
   Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
  
 
Note: Treatment-emergent adverse events are those with an onset after use of study medication. Not Related includes Not Related and Unlikely. Related 
includes Possible, Probable or Definite. Any unknown relationship is defined as Related. A subject is counted once according to maximum severity and 
relationship. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 45 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
Table 14.3.2.1.2: Summary of Treatment-Emergent Adverse Events by MedDRA System Organ Class and Preferred Term 
(Safety Population) 
(Page 1 of xx) 
 
  
System Organ Classa 
   Preferred Term 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
System Organ Class 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 Preferred Term 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 Preferred Term 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
a Counts reflect numbers of subjects reporting one or more adverse events that map to MedDRA dictionary (Version 19.0). At each level of summarization 
 (System Organ Class or Preferred Term) subjects are counted once. 
  
Note: Treatment-emergent adverse events are those with an onset after use of study medication. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 46 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.2.1.3: Summary of Treatment-Emergent Adverse Events by Severity 
(Safety Population) 
(Page 1 of xx) 
 
  
System Organ Classa 
 Preferred Term 
Severity 
Patidegib Gel 2% 
QD 
 N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle  
Gel 
 (N=xx) 
 
 
 
 
 
 
 
System Organ Class 
Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 
 Preferred Term 
Mild 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Moderate 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Severe 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
a Counts reflect numbers of subjects reporting one or more adverse events that map to MedDRA dictionary (Version 19.0). At each level of summarization 
 (System Organ Class or Preferred Term) subjects are counted once under the greatest reported severity. 
  
Note: Treatment-emergent adverse events are those with an onset after use of study medication. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 47 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.2.1.4: Summary of Treatment-Emergent Adverse Events by Relationship 
(Safety Population) 
(Page 1 of xx) 
 
  
System Organ Classa 
 Preferred Term 
Relationship 
Patidegib Gel 2% 
QD 
  (N=xx) 
Patidegib Gel 2% 
BID 
(N=xx) 
Patidegib Gel 4% 
QD 
(N=xx) 
Patidegib Gel 4% 
BID 
(N=xx) 
Vehicle  
Gel 
(N=xx) 
 
 
 
 
 
 
 
System Organ Class 
Unrelated 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 
 Preferred Term 
Unrelated 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Related 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
a Counts reflect numbers of subjects reporting one or more adverse events that map to MedDRA dictionary (Version 19.0). At each level of summarization 
 (System Organ Class or Preferred Term) subjects are counted once under the strongest reported relationship. 
  
Note: Treatment-emergent adverse events are those with an onset after use of study medication. Not Related includes Not Related and Unlikely, Related 
includes Possible, Probable, or Definite. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 48 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Table 14.3.2.2.1: Summary of Treatment-Emergent Serious Adverse Event Characteristics (Safety Population) 
Similar to Table 14.3.2.1.1. 
 
Table 14.3.2.2.2: Summary of Treatment-Emergent Serious Adverse Events by MedDRA System Organ Class and Preferred Term (Safety Population) 
Similar to Table 14.3.2.1.2. 
 
Table 14.3.2.2.3: Summary of Treatment-Emergent Serious Adverse Events by Severity (Safety Population) 
Similar to Table 14.3.2.1.3. 
 
Table 14.3.2.2.4: Summary of Treatment-Emergent Serious Adverse Events by Relationship (Safety Population) 
Similar to Table 14.3.2.1.4. 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 49 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
 
 
 
Table 14.3.3.1: Summary of Chemistry Laboratory Results 
(Safety Population) 
(Page 1 of xx) 
 
  
 
Patidegib Gel 2% 
QD 
(N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
(N=xx) 
Patidegib Gel 4% 
BID 
(N=xx) 
Vehicle  
Gel 
(N=xx) 
Alanine Aminotransferase (U/L) 
 
 
 
 
 
Baseline 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Week 6 
 
 
 
 
 
 n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Change from Baselinea 
      n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
      Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
      SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
      Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
      Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Continue for Week 12 
 
 
 
 
 
Repeat for all Chemistry 
 
 
 
 
 
  
 
a Baseline is the last non-missing assessment prior to the first dose of study drug. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 50 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.3.2: Summary of Hematology Laboratory Results (Safety Population) 
Similar to Table 14.3.3.1. 
 
Table 14.3.3.3: Summary of Quantitative Urinalysis Laboratory Results (Safety Population) 
Similar to Table 14.3.3.1. 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 51 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.3.4: Summary of Categorical Urinalysis Laboratory Results 
(Safety Population) 
(Page 1 of x) 
 
  
 
Patidegib Gel 2% 
QD 
(N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
(N=xx) 
Patidegib Gel 4% 
BID 
(N=xx) 
Vehicle  
Gel 
(N=xx) 
Bacteria (/HPF) 
 
 
 
 
 
Baselinea 
 
 
 
 
 
N 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
NONE SEEN 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
1+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
2+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
3+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Week 6 
 
 
 
 
 
N 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
NONE SEEN 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
1+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
2+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
3+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
Week 12 
N 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
NONE SEEN 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
1+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
2+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
3+ 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
xx ( xx.x%) 
 
 
 
 
 
 
Repeat for other qualitative urinalysis. 
 
 
 
 
 
  
 
a Baseline is the last non-missing assessment prior to the first dose of study drug. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 52 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.3.5: Summary of Chemistry Laboratory Results - Shift Table 
(Safety Population) 
(Page 1 of x) 
 
  
 
 
Week 6 
 
 
Patidegib Gel 2% QD (N=xx) 
Patidegib Gel 2% BID (N=xx) 
Patidegib Gel 4% QD (N=xx) 
 
Baseline 
  
BNL 
  
WNL 
  
ANL 
  
BNL 
  
WNL 
  
ANL 
  
BNL 
  
WNL 
  
ANL 
Test name (units) 
BNL 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) 
 
WNL 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) 
 
ANL 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patidegib Gel 4% BID (N=xx) 
Vehicle Gel  (N=xx) 
 
 
 
 
 
  BNL 
   WNL 
   ANL 
   BNL 
   WNL 
   ANL 
 
 
BNL 
 
 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%)  
 
WNL 
 
 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%)  
 
ANL 
 
 
xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%) xx ( xx.x%)  
[continue for all tests] 
 
 
 
 
 
 
 
 
 
 
 
Continue for Week 12 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
Note: BNL=Below Normal Limit, WNL=Within Normal Limit, ANL=Above Normal Limit. 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 53 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
 
Table 14.3.3.6: Summary of Hematology Laboratory Results - Shift Table (Safety Population) 
Similar to Table 14.3.3.5. 
 
Table 14.3.3.7: Summary of Quantitative Urinalysis Laboratory Results - Shift Table (Safety Population) 
Similar to Table 14.3.3.5. 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 54 
Protocol Number: 926-202 
28Sep2017 
 
 
Table 14.3.4.1: Summary of Vital Signs 
(Safety Population) 
(Page 1 of xx) 
 
  
 
Patidegib Gel 2% 
QD 
 (N=xx) 
Patidegib Gel 2% 
BID 
 (N=xx) 
Patidegib Gel 4% 
QD 
 (N=xx) 
Patidegib Gel 4% 
BID 
 (N=xx) 
Vehicle Gel 
 (N=xx) 
Diastolic Blood Pressure (mmHg) 
 
 
 
 
 
Baseline 
 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Week 12 
 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Change from Baseline 
 
 
 
 
 
 
n 
 
xx 
 
xx 
 
xx 
 
xx 
 
xx 
 
Mean 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
SD 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
xx.xx 
 
Median 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
xx.x 
 
Min. to Max. 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
xx to xx 
 
 
 
 
 
 
Continue for all vital signs in order as in CRF: Systolic BP, Diastolic BP, Heart Rate, Resp Rate, Oral Temp, (height and weight)  . 
  
 
  
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE,TIME) 
 
Note 
 
 
No 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 55 
Protocol Number: 926-202 
28Sep2017 
 
 
11. INDEX OF PLANNED LISTINGS 
 
Listing 16.1.7: Randomization Scheme .........................................................................................57 
Listing 16.2.1.1: End of Study Information ...................................................................................58 
Listing 16.2.1.2: Discontinued Subjects ........................................................................................59 
Listing 16.2.1.3: Screen Failures ...................................................................................................60 
Listing 16.2.2: Inclusion/Exclusion Criteria Violations ................................................................61 
Listing 16.2.3: Analysis Populations .............................................................................................62 
Listing 16.2.4.1: Subject Demographic Information .....................................................................63 
Listing 16.2.4.2.1: Unique Medical History Coded to MedDRA System Organ Classes and Preferred Terms 
64 
Listing 16.2.4.2.2: Medical History ...............................................................................................65 
Listing 16.2.4.3: Abbreviated Physical Examination ....................................................................66 
Listing 16.2.4.4.1: Unique Medication Names Coded to WHO-DDE ATC Level 2 Terms and Preferred Names 67 
Listing 16.2.4.4.2: Prior and Concomitant Medications ................................................................68 
Listing 16.2.4.4.3: Prior and Concomitant Therapies and Procedures ..........................................69 
Listing 16.2.4.5: Cleansers, Sunscreens and Moisturizers.............................................................70 
Listing 16.2.5.1: Study Medication Accountability Log ...............................................................71 
Listing 16.2.5.2: Drug Exposure Information ................................................................................72 
Listing 16.2.5.3: Drug Compliance................................................................................................68 
Listing 16.2.6.1: Assessment of Treatment Targeted Basal Cell Carcinomas ...............................74 
Listing 16.2.6.2: GLI1 mRNA Levels ...........................................................................................76 
Listing 16.2.6.3: Residual Tumor ..................................................................................................77 
Listing 16.2.6.4: Photographic Tumor Review ..............................................................................77 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 56 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.1.1: Assessment of Treatment Targeted Basal Cell Carcinomas: Local Signs and Symptoms 
79 
Listing 16.2.7.1.2: Systemic Symptoms ........................................................................................80 
Listing 16.2.7.2.1: Unique Adverse Events Coded to MedDRA System Organ Classes and Preferred Terms 
81 
Listing 16.2.7.2.2: Adverse Events ................................................................................................82 
Listing 16.2.7.2.3: Treatment-Emergent Serious Adverse Events .................................................83 
Listing 16.2.7.2.4: Subjects Who Prematurely Discontinued Study and/or Discontinued Study Drug Due to Adverse Events 
84 
Listing 16.2.8.1: Serum Pregnancy Test ........................................................................................85 
Listing 16.2.8.2.1: Chemistry Laboratory Results .........................................................................86 
Listing 16.2.8.2.2: Hematology Laboratory Results ......................................................................87 
Listing 16.2.8.2.3: Urinalysis Laboratory Results .........................................................................88 
Listing 16.2.8.3.1: Abnormal Chemistry Results ..........................................................................89 
Listing 16.2.8.3.2: Abnormal Hematology Results .......................................................................90 
Listing 16.2.8.3.3: Abnormal Urinalysis Results ...........................................................................91 
Listing 16.2.8.4: Vital Signs ..........................................................................................................92 
Listing 16.2.8.5: Clinical Evaluation .............................................................................................93 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 57 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.1.7: Randomization Scheme 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
 
 
 
 
 
 
 
 
 
Date of First 
Subject         Age/Sex  
Eval      
Randomization  
Application of  
Assigned Arm 
 
Actual Arm     
 
 
 
 
 
 
Date 
 
 
Study Drug 
 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx          xxxx          xxxxxxxx        xxxxxxxxxx            xxxxxxx  
 
xxxxxx       
 
xxxxxx       
 
xxxxxx          xxxx          xxxxxxxx        xxxxxxxxxx            xxxxxxx  
 
xxxxxx       
 
xxxxxx       
                                                                                                     
xxxxxx          xxxx          xxxxxxxx        xxxxxxxxxx            xxxxxxx  
 
xxxxxx       
 
xxxxxx       
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
                                               
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 58 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.1.1: End of Study Information 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                             Date of First     Date of Last                                       Did Subject  Primary Reason 
                             Application       Application           Date of Study Completion/    Complete     for Study 
Subject  Age/Sex  Eval       of Study Drug     of Study Drug         Discontinuation (Day)¹       the Study    Discontinuation 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xx           xxxxx x xxxxxxx 
                                                                                                               xxxxxx xxx xx xxxx 
                                                                                                               xxxxxxxxx xxxxx 
                                                                                                               xxxx xxxxx xxx 
                                                                                                               xxxxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xx           xxxxx x xxxxxxx 
                                                                                                               xxxxxx xxx xx xxxx 
                                                                                                               xxxxxxxxx xxxxx 
                                                                                                               xxxx xxxxx xxx 
                                                                                                               xxxxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx            xxxxxxxxxx xxxx              xxx 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date - Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date - Date of first dose + 1 for dates on or after Baseline. 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Note to Programmer: The ‘If Adverse Event, Protocol Violation, or Other, Please Specify’ free text box should all be concatenated with Primary Reason for Study 
Discontinuation 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 59 
Protocol Number: 926-202 
28Sep2017 
 
 
 
                                              Listing 16.2.1.2: Discontinued Subjects 
                                                             Treatment Arm   
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                             Date of First     Date of Last                                      Primary Reason 
                             Application       Application          Date of 
 
 
for Study 
Subject  Age/Sex  Eval       of Study Drug     of Study Drug        Discontinuation (Day)¹       Discontinuation 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxxxx x xxxxxxx 
                                                                                                 xxxxxx xxx xx xxxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxxxx x xxxxxxx 
                                                                                                 xxxxxx xxx xx xxxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xx           xxxxx x xxxxxxx 
                                                                                                              xxxxxx xxx xx xxxx 
                                                                                                              xxxxxxxxx xxxxx 
                                                                                                              xxxx xxxxx xxx 
                                                                                                              xxxxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
xxxxxx    xxxx    xxxxxxxx   xxxxxxxxxx        xxxxxxxxxx           xxxxxxxxxx xxxx              xxx 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date - Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date - Date of first dose + 1 for dates on or after Baseline. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Note to Programmer: The ‘If Adverse Event, Protocol Violation, or Other, Please Specify’ free text box should all be concatenated with Primary Reason for Study 
Discontinuation 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 60 
Protocol Number: 926-202 
28Sep2017 
 
 
 
                                              Listing 16.2.1.3: Screen Failures 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject  Age/Sex  Reason for Exclusion 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx    xxxx    xxxxxxxx    
 
 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 
Note to Programmer: If Inclusion Criteria Not Met or Exclusion Criteria Met, then include which, eg. Inclusion Criteria Not Met: Inclusion Criteria 5 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 61 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.2: Inclusion/Exclusion Criteria Violations 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
                                                 
Subject       Age/Sex     Eval         Criteria Failed    Description 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
xxxxxx        xxxx        xxxxxxxx     xxxxx              xxxxx xxx xx xxxxxxxx xxxx xxxxxxxxxxxxxxxxxxx xxxxxxxx xxxxxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 62 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.3: Analysis Populations 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject   Age/Sex     Population             Included     Reason(s) Excluded                                  
______________________________________________________________________________________________________________________________________ 
 
xxxxxx     xxxx       Intent-to-Treat        xxx 
                      Safety                 xxx 
                      Per Protocol           xxx 
 
xxxxxx     xxxx       Intent-to-Treat        xxx 
                      Safety                 xxx  
                      Per Protocol           xxx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 63 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.4.1: Subject Demographic Information 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
 _____________________________________________________________________________________________________________________________________ 
 
                                     A: Age      R: Race                                                   
 Subject   Eval      Date of Birth   S: Sex      E: Ethnicity                                             Informed Consent Date 
 _____________________________________________________________________________________________________________________________________ 
 
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                              
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                             
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxxx xxxxxxxx xx xxxxx xxxxxxx xxxxxxxx xxxxxxxxxx  xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
 
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxxxx   E: xxx xxxxxxxx xx xxxxxx                                 
                              
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                                                         
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
 
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                            
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                              
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                                        
                              
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                              
 xxxxxx    xxxxxxxx  xxxxxxxxxx      A: xx       R: xxxxx                                                 xxxxxxxxxx 
                                     S: xxxx     E: xxx xxxxxxxx xx xxxxxx                                 
                              
 
 
 _____________________________________________________________________________________________________________________________________ 
 SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 64 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.4.2.1: Unique Medical History Coded to MedDRA System Organ Classes and Preferred Terms 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
MedDRA System Organ Class                     MedDRA Preferred Term                       Medical History Verbatim Term 
______________________________________________________________________________________________________________________________________ 
 
xxxx xxx xxxxx                                xxxx xxx xxxxx                              xxxx 
 
                                                                                          xxxxxx xxxxxxxxxx xx xxxxxx 
 
                                                                                          xxxxxx xxxxxxxxxx xx xxxxx 
 
 
xxxx xxx xxxxx                                xxxx xxx xxxxx                              xxxx 
 
                                                                                          xxxxxx xxxxxxxxxx xx xxxxxx 
 
                                                                                          xxxxxx xxxxxxxxxx xx xxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
                                                                                           
______________________________________________________________________________________________________________________________________ 
Note: System Organ Class and Preferred Term map to the MedDRA dictionary (Version 19.0). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by System Organ Class, Preferred Term, and Verbatim Term. 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 65 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.4.2.2: Medical History 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                              NBCC  
Any unresolved or 
Condition/Surgery 
P: MedDRA Preferred Term     S: Onset/Surgery Date 
Subject   Age/Sex   Eval      Dx 
clinically relevant 
Verbatim Term   
S: MedDRA System Organ Class O: Ongoing 
 
 
 
 
Date 
conditions? 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx    xxxx     xxxxxxxx   xxxxxx  xx                
xxxxxxxxxxxxxxxxxxxxx P: xxxx xxx xxxxx         
S: xxxxxxx 
                                      
 
 
 
 
 
S: xxxx xxx xxxxx             O: xxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
Note: System Organ Class and Preferred Term map to the MedDRA dictionary (Version 19.0). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Verbatim Term, Start Date, and End Date.  Medical History term of Nodular Basal Cell Carcinoma should also be included. 
                                           
                                            
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 66 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.4.3: Abbreviated Physical Examination 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject         Age/Sex    Eval        Visit                   Date                Was Physical Examination Performed? 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xx 
 
                                       xxxx xx                 xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xx 
 
                                       xxxx xxxxxxxx           xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xx                 xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xxxxxxxx           xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xx                 xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xxxxxxxx           xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xx                 xxxxxxxxxx          xxx 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx               xxxxxxxxxx          xxx 
 
                                       xxxx xxxxxxxx           xxxxxxxxxx          xxx 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 67 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.4.4.1: Unique Medication Names Coded to WHO-DDE ATC Level 2 Terms and Preferred Names 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                                                                                             I: Indication 
ATC Level 2 Term                 Standardized Medication Name           Medication Name                      R: Route 
______________________________________________________________________________________________________________________________________ 
 
xxxxxxxxxxxxxx                   xxxxxxxxxxxxxx                         xxxxxxxx                             I: xxxxxxxxxxx 
                                                                                                             R: xxxxxxxxxxx 
 
                                                                        xxxxxxxxx                            I: xxxxxxxxxxx 
                                                                                                             R: xxxxxxxxxxx 
 
 
xxxxxxxxxxxxxx                   xxxxxxxxxxxxxx                         xxxxxxxx                             I: xxxxxxxxxxx 
                                                                                                             R: xxxxxxxxxxx 
 
                                                                        xxxxxxxxx                            I: xxxxxxxxxxx 
                                                                                                             R: xxxxxxxxxxx 
                                                                                                               
 
             
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
                                                             
______________________________________________________________________________________________________________________________________ 
Note: Standardized Medication Name and ATC Level 2 Term map to the WHO-DDE (Version March 1, 2016). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by ATC Level 2 Term, Standardized Medication Name, Medication Name, Indication, and Route. 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 68 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.4.4.2: Prior and Concomitant Medications 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                     M: Medication Name                                               
                                     P: Standardized Medication Name 
   F: Date of First Application    D: Dose 
                                     A: ATC Level 2 Term                      S: Medication Start Date (Day)¹ U: Unit 
                                     I: Indication                            E: Medication End Date (Day)¹   F: Frequency 
Subject       Age/Sex     Eval       T: Taken for Adverse Event               O: Ongoing                      R: Route 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx         xxxx       xxxxxxxx   M: xxxxxxxxxxxxx                         F: xxxxxxxxxxxxx                D: xx 
                                     P: xxxxxxxxxxxxx                         S: xxxxxxxxxx                   U: xx 
                                     A: xxxxxxxxxxxxx                         E: xxxxxxxxxx                   F: xxxx 
                                     I: xxxxxx xxxxxxxx                       O: xxxxxxx                      R:xxxx  
                                     T: xxxxxxxx                                                     
      
 
                                     M: xxxxxxxxxxxxx                         S: xxxxxxxxxx                   U: xx 
                                     P: xxxxxxxxxxxxx                         E: xxxxxxxxxx                   F: xxxx 
                                     A: xxxxxx xxxxxxxx                       O: xxxxxxx                      R:xxxx  
                                     I: xxxxxxxx                                                     
      
                                     T: xxx 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date – Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date – Date of first dose + 1 for dates on or after Baseline. 
Note: Standardized Medication Name and ATC Level 2 Term map to the WHO-DDE (Version March 1, 2016). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Start Date, End Date, Medication Name, Indication, and Route. 
 
  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 69 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.4.4.3: Prior and Concomitant Therapies and Procedures 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                   
C: System Organ Class            
F: Date of First Application 
                   
P: Preferred Term                     S: Start Date (Day)¹           
T: BCC Treatment Y/N 
         Age/Sex   
M: Procedure/Therapy Name            E: End Date (Day)¹              
B: BCC Category 
Subject  Eval      
I: Indication                         O: Ongoing                            I: BCC ID 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx   xxxx   
C: xxxxxxxxxxxxx               
F: xxxxxxxxxx                       
T: xxx 
         xx 
 
P: xxxxxx xxxxxxx              
S: xxxxxxxxxx xxxx                  
B: xxxxxxxxx xxxxxxx xxxxx 
                   
M: xxxxxxxxxx  
 
 
E: xxxxxxxxxx xxxx                  
I: xxx 
                   
I: xxxxxxxxxx  
 
 
O: xxxxxxxxxx xxxx                  
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date – Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date – Date of first dose + 1 for dates on or after Baseline. 
Note: System Organ Class and Preferred Term map to the MedDRA dictionary (Version 19.0). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Start Date, End Date, Procedure/Therapy Name, Indication, and Route. 
                                                
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 70 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.4.5: Cleansers, Sunscreens and Moisturizers 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
  
              Age/Sex P: Product Name 
 
S: Start Date (Day)¹      
 
Subject       Eval 
T: Product Type 
 
E: End Date (Day)¹     
 
 
 
F: Date of First Application O: Ongoing 
 
 
 
F: Frequency 
____________________________________________________________________________________________________________________________________ 
 
xxxxxx         xxxx    P: xxxxxxxxxxxxx              S: xxxxxxxxxx                       
F: xxx 
               xx 
T: xxxxxx xxxxxxx             E: xxxxxxxxxx xxxx                  
 
                   
F: xxxxxxxxxx  
 
O: xxxxxxxxxx xxxx                  
 
 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date – Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date – Date of first dose + 1 for dates on or after Baseline. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Start Date, End Date, Product Name, Type. 
                                                
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 71 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.5.1: Study Medication Accountability Log 
                                                           Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                               Kit         Tube  
Date              Dispense 
 
Date 
 
Return 
Subject      Age/Sex    Eval   Number      Number 
Dispensed         Weight (gm)      
Returned       Weight (gm)  
______________________________________________________________________________________________________________________________________ 
 
xxxxx         xx/x      xxxxx  xxx        xxx 
2016-07-26        xxx          
2016-08-08 
xx.x 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
If Not Done is checked for Dispensation / Weighed at Dispense / Returned then put NOT DONE for Date Dispense / Return, 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 72 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.5.2: Drug Exposure Information 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
 _____________________________________________________________________________________________________________________________________ 
                                             
              F: Date of             F: Number of Doses on                     
 S: Subject      First Application      Date of First Application      D: Date of Missed Application(s) 
 A: Age/Sex   L: Date of             L: Number of Doses on             N: Number of Missed Application(s)                 
 E: Eval         Last Application       Date of Last Application       R: Reason for Missed Application(s)   
 _____________________________________________________________________________________________________________________________________ 
 
 S: xxxxxx    F: xxxxxxxxxx          F: x                              D: xxxxxxxxxx             
 A: xxxx      L: xxxxxxxxxx          L: xxxxxxx                        N: xxxxxxx  
 E: xxxxxxxx                                                           R: x xxxxxxxx xxxx x xxxxxx xxx xxx xxxxxxxxxxx xx xxxx 
                                                                          xxxxxxxx xx xxxxxxxxxxx xxx xxx xxxxx xx 
                                                                                                                                                                  
                                                                       D: xxxxxxxxxx             
                                                                       N: x  
                                                                       R: xxxx xxxxxxx 
 
 S: xxxxxx    F: xxxxxxxxxx          F: x                                     
 A: xxxx      L: xxxxxxxxxx          L: x                          
 E: xxxx 
 
 S: xxxxxx    F: xxxxxxxxxx          F: x                                     
 A: xxxx      L: xxxxxxxxxx          L: x                          
 E: xxxxxxxx 
 
 S: xxxxxx    F: xxxxxxxxxx          F: x                              D: xxxxxxxxxx                        
 A: xxxx      L: xxxxxxxxxx          L: xxxxxxx                        N: x       
 E: xxxxxxxx                                                           R: xxxxxxx xxxxxx 
 
 S: xxxxxx    F: xxxxxxxxxx          F: x                                     
 A: xxxx      L: xxxxxxxxxx          L: x                          
 E: xxxx 
 
 
 
 _____________________________________________________________________________________________________________________________________ 
  
 SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing Format May Be Altered on Confirmation of Dosing Data Capture. 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 73 
Protocol Number: 926-202 
28Sep2017 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 74 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.5.3: Drug Compliance 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                               Total Number  
Expected Number 
 
Subject      Age/Sex    Eval   of Applications 
of Applications 
Compliance (%) 
______________________________________________________________________________________________________________________________________ 
 
xxxxx         xx/x      xxxxx   
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 75 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.6.1: Assessment of Treatment Targeted Basal Cell Carcinomas 
                                                            Treatment Arm 
                                                           (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                        
 
 
S: Subject   Eval  
Visit 
Visit 
BCC 
Body 
   Greatest 
Classifi-    Photo- 
Biopsied   Excised   ISGTA 
  
A: Age/Sex     
 
Date 
ID 
Location   Diameter 
cation       graphed 
 
______________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxx  xxxxxx xxxxxx xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
     
 
xxxxx xxx 
A: xxxx       
 
 
 
 
 
xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
     
 
xxxxx xxx 
 
 
 
 
xxxxxx xxxxxx xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
    X 
 
xxxxx xxx 
 
 
 
 
 
xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
    X 
 
xxxxx xxx 
 
 
S: xxxxxx    xxxxxxxx  xxxxxx xxxxxx xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
     
 
xxxxx xxx 
A: xxxx       
 
 
 
 
 
xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
     
 
xxxxx xxx 
 
 
 
 
xxxxxx xxxxxx xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
    X 
 
xxxxx xxx 
 
 
 
 
 
xx 
xxxxxxxx   xx mm 
xxxxxxxxxxx 
X 
X 
    X 
 
xxxxx xxx 
   
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 
 
Note to Programmer:  Some fields apply only at certain visits (eg. Excised at Week 12 / ET).  Leave blank at other visits.


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 76 
Protocol Number: 926-202 
28Sep2017 
 
 
  
 
Listing 16.2.6.2: GLI1 mRNA Levels 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject                    
 
T: Total # Cells 
C0: % cells in Bin 0 
C3: % cells in Bin 3  H: Hs-GLI1 H-Score 
A: Age/Sex   V: Visit     
  Tumor  
A: Avg Dots/Cell  
C1: % cells in Bin 1 
C4: % cells in Bin 4 
P: Percent Change from  
E: Eval      D: Date            Site  
QC: QC Pass/Fail 
C2: % cells in Bin 2 
  
 
 
   baseline 
______________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    V: xxxxxxxx        xxxx  
T: xx 
 
 
C0: xx  
 
C3: xx  
 
H: xx.xx  
A: xxxx      D: xxxxxxxx           
 
A: xx 
 
 
C1: xx  
 
C4: xx  
 
P: -- 
E: xxxxxxxx    
 
 
 
QC: xxxx 
 
C2: xx  
 
 
 
                                     
        
 
  xxxxx  
T: xx 
 
 
C0: xx  
 
C3: xx  
 
H: xx.xx  
     
 
A: xx 
 
 
C1: xx  
 
C4: xx  
 
P: xx.xx 
 
 
 
 
 
 
QC: xxxx 
 
C2: xx  
 
 
 
 
S: xxxxxx    V: xxxxxxxx        xxxx  
T: xx 
 
 
C0: xx  
 
C3: xx  
 
H: xx.xx  
A: xxxx      D: xxxxxxxx           
 
A: xx 
 
 
C1: xx  
 
C4: xx  
 
P: -- 
E: xxxxxxxx    
 
 
 
QC: xxxx 
 
C2: xx  
 
 
 
                                     
        
 
  xxxxx  
T: xx 
 
 
C0: xx  
 
C3: xx  
 
H: xx.xx  
     
 
A: xx 
 
 
C1: xx  
 
C4: xx  
 
P: xx.xx 
 
 
 
 
 
 
QC: xxxx 
 
C2: xx  
 
 
 
 
______________________________________________________________________________________________________________________________________ 
 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Visit, Date, Category.  
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 77 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.6.3: Residual Tumor 
                                                           Treatment Arm 
                                                            (Page 1 of xx) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject    Age/Sex  Eval    Visit     Visit 
BCC ID  
Surgical Report 
Evidence / Location   
 
 
 
 
 
Date 
Number  
Available? 
 
Of Residual Tumor? 
 
______________________________________________________________________________________________________________________________________ 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
If Evidence is ‘Y’ then add ‘/ Location’ (Tumor Marker Sample, Hohs Layer, Other).  If ‘Other’ then add text in Brackets. 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 78 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.6.4: Photographic Tumor Review 
                                                           Treatment Arm 
                                                            (Page 1 of xx) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject    Age/Sex  Eval     BCC ID  
  
 
 
 
 
Number  
 
______________________________________________________________________________________________________________________________________ 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Columns will be added as per file received by sponsor. 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 79 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.1.1: Assessment of Treatment Targeted Basal Cell Carcinomas: Local Signs and Symptoms 
                                                            Treatment Arm 
                                                           (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                        
 
 
S: Subject   Eval  
Visit 
Visit 
BCC 
Pain /  
Pruritus 
Erythema 
Edema 
Scabbing / 
A: Age/Sex     
 
Date 
ID 
Burning    
 
 
 
 
 
Crusting 
 
______________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxx  xxxxxx xxxxxx xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
A: xxxx       
 
 
 
 
 
xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
 
 
 
 
xxxxxx xxxxxx xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
 
 
 
 
 
xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
 
 
S: xxxxxx    xxxxxxxx  xxxxxx xxxxxx xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
A: xxxx       
 
 
 
 
 
xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
 
 
 
 
xxxxxx xxxxxx xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
 
 
 
 
 
xx 
xx 
 
xx 
 
xx 
 
xx 
xx 
   
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 80 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.1.2: Systemic Symptoms 
                                                           Treatment Arm 
                                                            (Page 1 of xx) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject    Age/Sex  Eval    Visit                Visit Date      Systemic Symptom                                              Result 
______________________________________________________________________________________________________________________________________ 
 
xxxxx       xx/X    ITT/S   Baseline             xxxx-xx-xx      Fatigue                                                       Xxx 
                                                                 Loss or Change in Taste                                       Xxx 
                                                                 Nausea                                                        Xxx 
                                                                 Vomiting                                                      Xxx 
                                                                 Diarrhea                                                      Xxx 
                                                                 Muscle Spasms or Cramps                                       Xxx 
                                                                 Changes of Quality or Quantity of Hair in Treated Areas       Xxx 
                                                                 Change in Frequency of Shaving                                Xxx 
                                                                 Other: xxxxxxx                                                Xxx 
 
                            Week 2               xxxx-xx-xx      Fatigue                                                       Xxx 
                                                                 Loss or Change in Taste                                       Xxx 
                                                                 Nausea                                                        Xxx 
                                                                 Vomiting                                                      Xxx 
                                                                 Diarrhea                                                      Xxx 
                                                                 Muscle Spasms or Cramps                                       Xxx 
                                                                 Changes of Quality or Quantity of Hair in Treated Areas       Xxx 
                                                                 Change in Frequency of Shaving                                Xxx 
                                                                 Other: xxxxxxx                                                Xxx 
 
                            Week x               xxxx-xx-xx      Fatigue                                                       Xxx 
                                                                 Loss or Change in Taste                                       Xxx 
                                                                 Nausea                                                        Xxx 
                                                                 Vomiting                                                      Xxx 
                                                                 Diarrhea                                                      Xxx 
                                                                 Muscle Spasms or Cramps                                       Xxx 
                                                                 Changes of Quality or Quantity of Hair in Treated Areas       Xxx 
                                                                 Change in Frequency of Shaving                                Xxx 
                                                                 Other                                                         Xxx 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
If Other is marked No show only Other if other is marked Yes, show Other with specification.  
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 81 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.2.1: Unique Adverse Events Coded to MedDRA System Organ Classes and Preferred Terms 
                                                             (Page xx of yy) 
 
 _____________________________________________________________________________________________________________________________________ 
 
 MedDRA System Organ Class                    MedDRA Preferred Term                        Adverse Event 
 _____________________________________________________________________________________________________________________________________ 
 
 xxxxx xxxxx xxxxx                            xxxxx xxxxx xxxxx                            xxxxxxxxxx 
 
                                                                                           xxxxxxxx xxxxxxxx xxxxxxxxxxx 
 
                                                                                           xxxxxxxx xxxxxxxxx xxxxxxxxxxxxxxxx 
 
 
                                              xxxxx xxxxx xxxxx                            xxxxxxxxxx 
 
                                                                                           xxxxxxxx xxxxxxxx xxxxxxxxxxx 
 
                                                                                           xxxxxxxx xxxxxxxxx xxxxxxxxxxxxxxxx 
 
                                                                                            
                                              xxxxxxxxxx                                   xxxxx xxxxx xxxxx                             
 
                                                                                           xxxxxxxx xxxxxxxx xxxxxxxxxxx 
 
                                                                                           xxxxxxxx xxxxxxxxx xxxxxxxxxxxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 _____________________________________________________________________________________________________________________________________ 
 Note: System Organ Class and Preferred Term map to MedDRA dictionary (Version 19.0). 
 SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by MedDRA System Organ Class, Preferred Term, and Adverse Event. 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 82 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.2.2: Adverse Events 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                                                                   S: Severity 
                             C: System Organ Class   
F: Date of First       R: Relationship to Study 
                             P: Preferred Term           
   Application            Drug    
                             V: Verbatim              
S: Start Date (Day)1   E: Serious                  
Subject   Age/Sex  Eval      T: In Treatment Area          E: End Date (Day)1     O: Outcome                 A: Action Taken 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx      xxxx   xxxxxxxx  S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xxxxxxxxxxxxxxx            E: xxxxxxxxxx xxxx     E: xx                               
                             T: xx                                                 O: xxxxxxxxx 
 
                             S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xx                         E: xxxxxxxxxx xxxx     E: xxx                               
                             T: xx                                                 O: xxxxxxxxx 
 
 
xxxxxx      xxxx   xxxxxxxx  S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xxxxxxxxxxxxxxx            E: xxxxxxxxxx xxxx     E: xx               
                             T: xx                                                 O: xxxxxxxxx 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date - Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date - Date of first dose + 1 for dates on or after Baseline. 
Note: System Organ Class and Preferred Term map to the MedDRA dictionary (Version 19.0). 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Start Date, End Date, and Adverse Event. 
Note Action Taken will consist of a concatenation of Action Taken Regarding Study Drug, Action Taken Regarding Hospitalization, Action Taken Concomitant Medication, Action 
Taken Procedure/Therapy, Action Taken Other, and Specify 
 
                                            
  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 83 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.7.2.3: Treatment-Emergent Serious Adverse Events 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                                                                   S: Severity 
                             C: System Organ Class   
F: Date of First       R: Relationship to Study 
                             P: Preferred Term           
   Application            Drug    
                             V: Verbatim              
S: Start Date (Day)1   E: Serious                  
Subject   Age/Sex  Eval      T: In Treatment Area          E: End Date (Day)1     O: Outcome                 A: Action Taken 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx      xxxx   xxxxxxxx  S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xxxxxxxxxxxxxxx            E: xxxxxxxxxx xxxx     E: xx                               
                             T: xx                                                 O: xxxxxxxxx 
 
                             S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xx                         E: xxxxxxxxxx xxxx     E: xxx                               
                             T: xx                                                 O: xxxxxxxxx 
 
 
xxxxxx      xxxx   xxxxxxxx  S: xxxxxxxxxxxxxxx            F: xxxxxxxxxx          S: xxxx                    A: xxxxxxxxxxxxx 
                             P: xxxxxxxxxxxxxxx            S: xxxxxxxxxx xxxx     R: xxxxxxxxxx               
                             V: xxxxxxxxxxxxxxx            E: xxxxxxxxxx xxxx     E: xx               
                             T: xx                                                 O: xxxxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
Note: System Organ Class and Preferred Term map to the MedDRA dictionary (Version xx.x). 
¹ Day is calculated as date - Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date - Date of first dose + 1 for dates on or after Baseline. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
Listing sorted by Subject Number, Start Date, End Date, and Adverse Event. 
Note Action Taken will consist of a concatenation of Action Taken Regarding Study Drug, Action Taken Regarding Hospitalization, Action Taken Concomitant Medication, Action 
Taken Procedure/Therapy, Action Taken Other, and Specify 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 84 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.7.2.4: Subjects Who Prematurely Discontinued Study and/or Discontinued Study Drug Due to Adverse Events 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
                                                                                  ________________Adverse Events___________________  
                                                                                  A: Verbatim 
S: Subject              _________________ Discontinuation______________           S: Severity                    
A: Age/Sex              F: Date of First Application  D: Date (Day)¹              R: Relationship to           S: Start Date (Day)¹  
E: Eval                 L: Date of Last Application   R: Primary Reason              Study Drug                E: End Date (Day)¹ 
______________________________________________________________________________________________________________________________________ 
 
S: xxxxxxxxxxxxxxx      F: xxxxxxxxxxxxxxx            D: xxxxxxxxxxxxxxx        A: xxxxxxxxxxxxxxx            S: xxxxxxxxxxxxxxx     
A: xxxx                 L: xxxxxxxxxxxxxxx            R: xxxxxxxxxxxxxxx        S: xxxxxxxxxx                 E: xxxxxxxxxxxxxxx     
E: xxxxxxxx                                                                     R: xxxxxxxxxx 
 
S: xxxxxxxxxxxxxxx      F: xxxxxxxxxxxxxxx            D: xxxxxxxxxxxxxxx        A: xxxxxxxxxxxxxxx            S: xxxxxxxxxxxxxxx     
A: xxxx                 L: xxxxxxxxxxxxxxx            R: xxxxxxxxxxxxxxx        S: xxxxxxxxxx                 E: xxxxxxxxxxxxxxx     
E: xxxxxxxx                                                                     R: xxxxxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
¹ Day is calculated as date - Date of first dose for dates prior to Baseline.  Otherwise, day is calculated as 
   date - Date of first dose + 1 for dates on or after Baseline. 
 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Start Date, End Date, and Adverse Event. 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 85 
Protocol Number: 926-202 
28Sep2017 
 
 
                       
Listing 16.2.8.1: Serum Pregnancy Test 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject    Age/Sex         Eval             Visit                 Date             Was the Specimen Collected       Results     
______________________________________________________________________________________________________________________________________ 
 
xxxxxx     xxxx            xxxxxxxx         xxxxxxxxxx            xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxxxxxx              xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx xxxxxxxxx        xxxxxxxxxx       xxx                              xxxxxxxx 
                              
xxxxxx     xxxx            xxxxxxxx         xxxxxxxxx             xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxxxxxx              xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx xxxxxxxxx        xxxxxxxxxx       xxx                              xxxxxxxx 
 
xxxxxx     xxxx            xxxxxxxx         xxxxxxxxxx            xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxxxxxx              xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx xxxxxxxxx        xxxxxxxxxx       xxx                              xxxxxxxx 
                              
xxxxxx     xxxx            xxxxxxxx         xxxxxxxxx             xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxxxxxx              xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xxx                              xxxxxxxx 
                                            xxxx x                xxxxxxxxxx       xx                               
                                            xxxx xxxxxxxxx        xxxxxxxxxx       xxx                              xxxxxxxx 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 86 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.8.2.1: Chemistry Laboratory Results  
                                                            Treatment Arm 
                                                           (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
______Reference Range______    Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units           Low      High   Indicator    Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT = ’Chemistry’ and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 87 
Protocol Number: 926-202 
28Sep2017 
 
 
 
Listing 16.2.8.2.2: Hematology Laboratory Results  
                                                            Treatment Arm 
                                                           (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
______Reference Range______    Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units           Low      High   Indicator    Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT=’Hematology’ and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 88 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.8.2.3: Urinalysis Laboratory Results  
                                                            Treatment Arm 
                                                           (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
Reference  
 
 
Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units          Range 
 
 Indicator     Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              xx - xx        xx    
xxxxxx         
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT=’Urinalysis’ and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 89 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
Listing 16.2.8.3.1: Abnormal Chemistry Results 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
______Reference Range______    Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units           Low      High   Indicator    Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT=’Chemistry’ and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 90 
Protocol Number: 926-202 
28Sep2017 
 
 
 
 
Listing 16.2.8.3.2: Abnormal Hematology Results 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
______Reference Range______    Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units           Low      High   Indicator    Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT=’Hematology’ and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 91 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.8.3.3: Abnormal Urinalysis Results 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
S: Subject    
A: Age/Sex      
 
 
 
 
 
 
 
 
 
Reference  
 
 
Clinically 
E: Eval      Test   
 
Visit 
 
Visit Date 
Results  
Units          Range 
 
 Indicator     Significant  
_____________________________________________________________________________________________________________________________________ 
 
S: xxxxxx    xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              xx - xx        xx    
xxxxxx         
A: xxxx        
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
E: xxxxxxxx                   xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
     xxxxxxxxx     
xxxxxxxxx    
xxxxxxxxxxxxx    xxx          xxx              x        xx  xxxxxx         xxx 
 
 
 
 
xxxxx 
 
xxxxxxxxxxxxx    xxx         xxxxxx           
 
 
 
xx 
xxxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
                              xxxxxx  
xxxxxxxxxxxxx    xxx         xxxxxx             
 
 
 
xx 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing filtered on LBCAT=Urinalysis and sorted by Subject Number, Test, Visit Date.  
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 92 
Protocol Number: 926-202 
28Sep2017 
 
 
     
 
Listing 16.2.8.4: Vital Signs 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
_____________________________________________________________________________________________________________________________________ 
 
Subject   Age/Sex  Eval       Visit                  Date              Vital Sign                  Result           Units 
_____________________________________________________________________________________________________________________________________ 
 
xxxxxx    xxxx     xxxxxxxx   xxxxxxxxx              xxxxxxxxxx        Diastolic Blood Pressure    xx               xxxx 
                                                                       Systolic Blood Pressure     xx               xxxx 
                                                                       Pulse                       xx               xxxxxxxxx 
                                                                       Respiration Rate            xx               xxxxxxxxxxx 
                                                                       Oral Temperature            xxxx             xxxxxxxxxx 
 
                              xxxxxxxx               xxxxxxxxxx        Diastolic Blood Pressure    xx               xxxx 
                                                                       Systolic Blood Pressure     xx 
                                                                       Pulse                       xx               xxxxxxxxx 
                                                                       Respiration Rate            xx               xxxxxxxxxxx 
                                                                       Oral Temperature            xxxx             x 
                                                                       Height                      xx               xxxxxxxxxxx 
                                                                       Weight                      xxx              xxx 
 
                              xxxx xx                xxxxxxxxxx        Weight                      xxx              xx 
 
                              xxxx xxxxxxxxx         xxxxxxxxxx        Diastolic Blood Pressure    xx               xxxx 
                                                                       Systolic Blood Pressure     xx               xxxx 
                                                                       Pulse                       xx               xxxxxxxxx 
                                                                       Respiration Rate            xx               xxxxxxxxxxx 
                                                                       Oral Temperature            xxxx             xxxxxxxxxx 
                                                                       Weight                      xxx              xxx 
 _____________________________________________________________________________________________________________________________________ 
 SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME) 
 
Listing sorted by Subject Number, Visit, Date, Vital Sign. 
 
 
 
 


 
 
 
 
Statistical Analysis Plan for PellePharm, Inc. 
Page 93 
Protocol Number: 926-202 
28Sep2017 
 
 
Listing 16.2.8.5: Clinical Evaluation 
                                                             Treatment Arm 
                                                            (Page xx of yy) 
 
______________________________________________________________________________________________________________________________________ 
 
Subject         Age/Sex    Eval        Visit  
Date 
 
Was Clinical 
 
 
Is the Subject Experiencing 
Evaluation Performed?  
Adequate Healing? / Specify if No 
______________________________________________________________________________________________________________________________________ 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx    xxxxxxxxxx     xx 
 
 
 
xxx 
 
 
 
xxxxxx          xxxx       xxxxxxxx    xxxxxxxxx    xxxxxxxxxx     xx 
 
 
 
xx / xxxxxxxxxxxxxxxxxx  xx 
 
 
 
 
 
 
______________________________________________________________________________________________________________________________________ 
SOURCE: USERNAME\SPONSOR\PROJECT\JOBNAME (DATE, TIME)