Clinical trial NCT02654483
Conditions: Epidermolysis Bullosa Pruritus
Sponsor: Jean Yuh Tang
Start date: 2016-08-31; Completion date: 2018-07-27
Enrollment: 14 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
Our goal is to determine whether daily oral administration of VPD-737 (5 mg) is effective and safe in treating moderate to severe pruritus in patients with Epidermolysis Bullosa (EB).

Detailed description:
Itch is the most common complaint reported by patients with EB of all subtypes, and there is no current effective treatment. Itch often triggers scratching that creates new wounds and increases EB disease severity.

This study aims to target the physiological mechanisms of pruritus (itch) in patients with EB. Substance P is a major mediator of pruritus and binds to the receptor neurokinin-1 (NK1), which is expressed in the central nervous system and the skin.

VPD-737 (serlopitant), a novel drug that inhibits the NK1 receptor, has been shown to reduce severe itch in a previous study of 257 adult patients with chronic pruritus.

Each patient will be un-blinded individually after completing 3 months of study period.

All patients who complete the study will be offered a 2-month period on active drug. Patient who received active drug in the first period will be contacted and asked if they would like to continue on active drug for an additional 2 months.

Patients who received placebo in the first period will be contacted and asked if they would like to repeat the study on open label for 2 months.

Arms: arm 1: 5 mg tablets of VPD-737 to be taken daily by mouth for 56 days arm 2: Placebo tablets to be taken daily by mouth for 56 days
Interventions: intervention 1: VPD-737 intervention 2: Placebo
Intervention descriptions: intervention 1: VPD-737 inhibits the receptor neurokinin-1. intervention 2: Matching tablets to VPD-737 tablets without active drug

Protocol documents:
Prot_SAP_001.pdf: 
Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               1 of 8 
 
1) Title: A Phase 2 trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in 
Epidermolysis Bullosa patients 
 
2) PI: Jean Tang, MD, PhD 
 
3) Funding: Epidermolysis Bullosa Research Partnership (SPO # 117763) 
 
4) Fund sources for this project: 
Epidermolysis Bullosa Research Partnership (EBRP) – provided funding 
Menlo Therapeutics, Inc. – provided study medication 
 
5) Specific aims: 
This study aims to determine the safety profile of Serlopitant (or VPD-737) in children (>13 y.o.) 
and adults with pruritus associated with epidermolysis bullosa (EB). This study also aims to 
determine the efficacy of Serlopitant (5 mg) compared with placebo on reducing EB itch as 
measured by patient self-reports using a numeric rating scale (NRS) for itch severity and the 
Stanford EB itch questionnaire. A secondary aim of this study is to determine whether 
Serlopitant when compared with placebo reduces EB wounds and promotes healing as 
determined by serial photographs. 
 It is our hope that Serlopitant will reduce itch in adolescents and adults with EB. 
 
6) General background: 
NK1 receptor antagonists (Aprepritant and Serlopitant) were originally tested in clinical trials as 
an antiemetic and a medication to treat overactive bladder. Since 2005, NK1 receptor antagonists 
have been evaluated in 13 completed Phase I studies and two Phase 2b studies (N=300 subjects). 
Serlopitant has been well tolerated in young men at a single dose of 400mg, and in the elderly at 
a dose of up to 20mg. Repeat dosing of up to 50 mg a day for 4 weeks was well tolerated in 
young adults and elderly males. There were no drug-related serious adverse events (SAE) in 
these Phase I studies. 
 
In a double-blinded, 6-week randomized trial of 257 adults with chronic pruritus, Serlopitant was 
shown to decrease itching significantly in a dose-dependent manner (submitted NEJM, 
clinicaltrials.gov NCT01951274). Participants in this study had chronic pruritus due to a variety 
of dermatologic, neuropathic, and psychogenic causes that was unresponsive to topical steroids 
and antihistamines. Serlopitant was well tolerated and safe in doses up to 5mg daily, and there 
were no treatment related serious adverse events. Based on this data, we have chosen to compare 
5mg serlopitant vs. placebo. When assessing pruritus with a visual analog scale (VAS), 
symptoms were reduced in a statistically significant manner in both the 1mg (p = 0.001) and the 
5mg (p = 0.002) dose groups when compared to placebo. After 6 weeks of treatment, nearly 70% 
of participants taking 5mg of serlopitant reported a 2-point reduction in VAS while nearly 50% 
of participants reported a 4-point decrease in VAS. 
 
In a Phase 2 dose-ranging study of 557 subjects examining treatment of overactive bladder, the 
most common clinical AEs across all treatment groups were headache (5.7%), diarrhea 
(5.2%), urinary tract infection (4.7%), dry mouth (4.5%), nasopharyngitis (4.5%), upper 
respiratory tract infection (4.3%), fatigue (4.1%), dizziness (3.8%), back pain (2.9%), nausea 
(2.3%), and peripheral edema (2.2%). 
 
 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               2 of 8 
 
 
 
 
 
Serlopitant has been more recently evaluated in a Phase 2 study of 257 adult subjects 18-65 years 
of age with chronic pruritus (TCP-101). In this study, treatment-emergent AEs (TEAEs) were 
reported in 25.4% of subjects in the placebo group and in 31.3%, 36.9%, and 37.5% of subjects 
in the serlopitant 0.25 mg, 1 mg, and 5 mg groups, respectively. Two severe TEAEs were 
reported: hypothyroidism in one subject in the serlopitant 0.25 mg group and peripheral 
neuropathy in one subject in the serlopitant 5 mg group. Both of these events were considered 
not related to study treatment. 
 
Treatment-related TEAEs were reported in 7.9% of TCP-101 subjects in the placebo group and 
in 10.9%, 13.8%, and 12.5% of subjects in the serlopitant 0.25 mg, 1 mg, and 5 mg groups, 
respectively. “Nervous systems disorders” was the SOC with the highest incidence of treatment-
related adverse events. 
 
One SAE (spontaneous abortion) occurred in a TCP-101 subject in the serlopitant 1 mg group. 
This event was considered not related to study treatment. Five subjects (three in the placebo 
group, one in the serlopitant 1 mg group, and one in the serlopitant 5 mg group) discontinued the 
study as a result of a TEAE. Two of these subjects had events considered not related to study 
treatment (pruritus in 2 subjects) and three subjects had events considered to be treatment related 
(headache in one subject; diarrhea in one subject; and symptoms of a panic attack in one 
subject). 
 
There was no evidence of meaningful trends in laboratory abnormalities or changes in vital signs 
in the TCP-101 study. Two subjects had a clinically significant change in an electrocardiogram 
(ECG): 1 subject in the serlopitant 0.25 mg group changed from normal to abnormal (clinically 
significant), and 1 subject in the serlopitant 5 mg group shifted from abnormal (not clinically 
significant) to abnormal (clinically significant). 
 
Overall, the nonclinical and clinical studies to date have demonstrated that serlopitant has an 
acceptable safety and pharmacokinetic profile, and they support the further evaluation of 
Serlopitant in pruritus-related indications.  
 
         Overall, headache was the most commonly reported TEAE in TCP-101, reported in 6.3% of 
subjects in the placebo group and in 1.6%, 4.6%, and 1.6% of subjects in the serlopitant 0.25 mg, 
1 mg, and 5 mg groups, respectively. Somnolence and diarrhea were the TEAEs with the highest 
overall incidence in the active-treatment groups. Somnolence was reported in 1.6% of subjects in 
the placebo group and in 1.6%, 4.6%, and 4.7% of subjects in the Serlopitant 0.25 mg, 1 mg, and 
5 mg groups, respectively. Diarrhea occurred in 1.6% of subjects in the placebo group and in 0%, 
6.2%, and 3.1% of subjects in the serlopitant 0.25 mg, 1 mg, and 5 mg groups, respectively. 
Most of the TCP-101 TEAEs were mild or moderate in severity. 
 
         Overall, the nonclinical and clinical studies to date have demonstrated that serlopitant has an 
acceptable safety and pharmacokinetic profile, and had been supported for further evaluation of 
Serlopitant in pruritus-related indications. Moreover, existing nonclinical data in adult animals 
and cumulative clinical data in adults support clinical trials in adolescents. 
 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               3 of 8 
7) Preliminary unpublished data:  
There is a trend towards greater reduction in itch severity as measured by daily self-reported NRS itch 
severity for serlopitant 5 mg as compared to placebo during the two month active trial period, though 
this was not statistically significant.    
 
 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               4 of 8 
8) Experimental design and data analysis: 
 
Inclusion criteria: 
1. Males or females who are at least 13 years of age. 
2. Willing and able to understand and sign informed assent/consent. Adolescents will need a 
parent or guardian willing and able to give consent. 
3. Clinical diagnosis of epidermolysis bullosa (DEB, EBS, or JEB). 
4. Subjects must have pruritus of at least 6 weeks duration 
5. History of chronic pruritus 
6. On the Stanford EB Itch survey, subject must have a Screening Visit (Visit 1) NRS 
pruritus score of at least 4 on either (1) average itch score in the past 24 hours or (2) 
bathing/dressing itch score in the past 24 hours. 
7. Female subjects must be of non-childbearing potential (ie, post-menopausal for at least 1 
year, had a hysterectomy, or had a tubal ligation) or, if of childbearing potential, must 
have a confirmed negative urine pregnancy test prior to study treatment and be willing to 
use effective contraception for the duration of the trial. Effective contraception is defined 
as follows: oral/implant/injectable/ transdermal contraceptives, intrauterine device, 
condom with spermicide, or diaphragm with spermicide. Abstinence or partner’s 
vasectomy is acceptable if the female agrees to use effective contraception if she decides 
to discontinue abstinence or to have sexual intercourse with a non-vasectomized partner. 
8. Judged to be in good health based upon the results of a physical examination, medical 
history, and safety laboratory tests. 
 
Exclusion criteria: 
1. Have any medical condition or disability that would interfere with the assessment of 
safety or efficacy in this trial or would compromise the ability of the subject to travel to 
Stanford or to undergo study procedures or to give informed consent. 
2. Have a history of sensitivity to any components of the study material. 
3. Are females of childbearing potential who are unwilling to use adequate contraception or 
who are breast feeding. 
4. Have any chronic or acute medical condition that, in the opinion of the investigator, 
might interfere with the study results or place the subject at undue risk. 
5. Have chronic renal disease, ie, serum creatinine greater than 2 times the upper limit of 
normal. 
6. Have chronic liver disease, ie, AST or ALT greater than 2 times the upper limit of 
normal. Subjects with hepatitis B and C who have normal liver function may be enrolled. 
7. Have a current malignancy (such as Hodgkin’s lymphoma, B or T cell lymphoma, or 
myeloma) or blood cell dyscrasia (eg, polycythemia or myelofibrosis) that would lead to 
systemic chronic pruritus. 
8. Subjects with untreated hyperthyroidism. 
9. Have pruritus of psychogenic etiology (delusions of parasitosis, obsessive compulsive 
disorder and major depression) or neuropathic etiology (due to shingles, spinal cord 
injury or with neurologic deficit). 
10. Have pruritus due to urticaria, drug allergy, or infection (such as pityriasis rosea or tinea 
or active human immunodeficiency virus [HIV]). Note: Subjects with HIV who have 
undetectable viral load, CD 4 counts >200 cells/cc, and stable retroviral therapy may 
enroll. 
11. Have taken investigational medications within 30 days prior to Screening. 
12. Are unwilling to discontinue specific pruritus medications for at least two weeks prior to initiation 
of study and throughout the study period (all severe CYP34A inhibitors - this 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               5 of 8 
includes miconazole, delavirdine, conivaptan, Clarithromycin, telithromycin, nefazodone, 
itraconazole, ketoconazole, indinavir, lopinavir, nelfinavir, ritonavir, saquinavir). 
13. Within in the past 12 months, have expressed suicidal ideation with some intent to act. 
14. Started or changed medications, creams, or emollients including over-the-counter (OTC) 
preparations or bath oil treatment for relief of pruritus within 3 months prior to 
Screening. 
15. Have any social or medical condition (e.g. alcoholism, drug dependency, psychotic state) 
that, in the investigator’s opinion, might interfere with the subject’s ability to comply 
with the requirements of the protocol. 
 
Statistical basis for the number of subjects to be enrolled:  
This 14 patient clinical trial is powered only to determine large differences in the placebo (N=7) 
vs. 5 mg (N=14) groups. In power calculations, we assumed a mean of 8 NRS for the placebo vs. 
5 NRS for the active with a SD of 3, and p-value of 0.05 (one sided). 
 
Two-sample t test with equal variances 
 
Obs 
Mean 
Std. Err. 
Std. Dev. 
[95% Conf. Interval] 
x 
14 
5 
0.8017837 
3 
3.267852 
6.732148 
y 
7 
8 
1.133893 
3 
5.225463 
10.77454 
combined 
21 
6 
0.7121396 
3.263434 
4.514503 
7.485497 
 
diff 
-3 
1.38873 
 
-5.906646 
-0.0933544 
 
Diff= mean (x) – mean (y) 
t = -2.1602 
Ho: diff = 0 
Degrees of freedom = 19 
Ha: diff < 0 
Ha: diff != 0 
Ha: diff > 0 
Pr(T < t) = 0.0219 
Pr(|T| > |t|) = 0.0437 
Pr(T > t) = 0.9781 
 
 
Statistical plan for analyzing at least the primary hypothesis:  
The sample size is powered to detect small differences in NRS score. Because itch is subjective 
and varies daily, subjects will be asked to keep an itch diary every day. For each subject, we will 
plot their average itch scores over time before and after drug intervention. For each dosage 
group, we will use linear regression methods with mix effects (GLM, SAS) to track the change in 
itch in each of the groups from baseline. This study will provide the effect size, standard 
deviation and range of itch scores in EB patients in order to properly power a larger phase 2 trial. 
This study can detect large differences in itch scores should Serlopitant decrease itch from severe 
to low-none at 1 and 2 months. At the 3 month follow up, we will measure the durability and 
length of response.  
 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               6 of 8 
Matrix showing procedure plan for each study visit: 
 
 
Schedule of Events for Phase 2 Serlopitant 
 
 
Screening period 
Treatment period 
Follow up 
 
 
Phone call 
Visit 1 
 
Visit 2 
Visit 3 
Phone call 
Early 
Term 
 
Screening 
call 
Screening 
visit 
 
Determina-
tion of 
Eligibility 
 
Month 1 
Month 
2/EOT 
Month 3 
 
Examination 
Day -60 to  
-30 
Day -44* to 
-1 
Months 6 
to 0 
Day 30 
±14 
Day 60 
±14 
Day 90 
±14 
 
Informed consent/ 
Photographic consent 
 
X 
 
 
 
 
 
Demographics 
 
X 
 
 
 
 
 
Medical history 
 
X 
Review  
 
 
 
 
Priora & current medications 
X 
X 
 
X 
X 
X 
X 
Abbreviated physical 
examination 
 
X 
 
 
X 
 
X 
Vital signs 
 
X 
 
X 
X 
 
X 
Urine pregnancy test (UPT)b 
 
X 
Review 
results 
X 
X 
 
X 
Inclusion/exclusion criteria 
X 
X 
 
 
 
 
 
Blood samples for clinical 
laboratory testsc 
 
Xc 
Review 
results 
Xc 
Xc 
 
Xc 
Randomization 
 
 
Xh 
 
 
 
 
NRSd & Stanford EB survey  
 
X 
 
X 
X 
Xe 
X 
Photograph the affected areaf 
 
X 
 
X 
X 
 
X 
Dispense study medication  
 
 
Xh 
X 
 
 
 
Dispense subject diary 
 
X 
Xh 
X 
Xg 
 
 
Review/collect diary with 
subject 
 
 
 
X 
X 
X 
X 
Review and record adverse 
events 
 
 
 
X 
X 
X 
X 
 
Note: NRS = numeric rating score.  
* Includes up to 30 day washout from prohibited medications.  The study medication and diary dispensation are to 
occur no more than 1-2 weeks after Screening Visit. 
 
a: 
all medications taken within 30 days prior to screening. 
b: for females of child-bearing potential. 
c: 
all subjects: hematology, serum chemistry will be measured at Screening, Month 1, and Month 2/EOT or early 
termination (ET). 
d:    NRS recorded by subject daily, using diary, from screening visit through the end of the study; NRS is included in 
the Stanford EB Itch survey. 
e: 
the Stanford EB survey will be administered over the phone by a member of the research staff.      
f: 
photographs taken only if patient has signed photography consent 
g:    a return envelope will be provided for the patient to return the study diary to Stanford after the follow up phone  
       call  


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               7 of 8 
 
Schedule of Events for Phase 2 Serlopitant 
 
 
Screening period 
Treatment period 
Follow up 
 
 
Phone call 
Visit 1 
 
Visit 2 
Visit 3 
Phone call 
Early 
Term 
h:    Patient letter of enrollment and study medication will be mailed to patient after determination of eligibility  
       following the Screening Visit 
 
 
Each subject will be un-blinded individually after completing 3 months of study period. All patients who complete 
the study will be offered a 2-month trial on active drug. Patient who received active drug in the first phase will be 
contacted and asked if they would like to continue on active drug for an additional 2 months. Patients who received 
placebo in the first phase will be contacted and asked if they would like to repeat the study on open label for 2 
months. 
 
When this occurs, the schedule of events will be as follows: 
 
Patients who would like to continue with the treatment for the open-label extension portion of the study will be 
provided with the research medication and research daily diary. They will be followed up by phone at baseline, 1 
month, 2 months, and 3 months (wash out). In addition, they will go to an external lab near their home for lab 
monitoring that will occur once a month for the two months they are on active drug. 
 
 
 


Serlopitant Itch Clinical Protocol (IRB #: 34182, IND: 129320) 
 
 
 
 
 
Stanford Dermatology, PI: Jean Tang, MD, PhD  
    
Version: 09 Jan, 2017 
                                               8 of 8 
Data Monitoring Plan: 
 
The Principal Investigator will provide oversight on monitoring safety/AEs/SAEs at this site. 
While there is not Data Safety Monitoring Board, the PI will act as the medical expert who 
answers safety/medical questions and who will provide medical expert opinion on this study. All 
AEs will be regularly reviewed and evaluated. The investigator will assess whether the AE 
represents an unanticipated problem (UP). If the investigator determines that the AE represents a 
UP, the investigator will promptly report the AE to the IRB. If the adverse event does not qualify 
as a UP, the adverse event should be reported at continuing review.  
 
FDA Reporting: 
 
In this study, the occurrence of any SAE from the time of informed consent until 4 weeks after 
the last dose of study drug will be reported to the Stanford IRB and the FDA within 24 hours of 
coming to the attention of the investigator. 
 
Reporting any unexpected fatal or life-threatening suspected adverse reactions to the FDA will 
occur no later than 7 calendar days after initial receipt of the information.  
 
Reporting any serious, unexpected suspected adverse reactions, findings from other clinical, 
animal or in-vitro studies that suggest significant human risk, and (3) a clinically important 
increase in the rate of a serious suspected adverse reaction to the FDA and all investigators will 
occur no later than 15 calendar days after determining that the information qualifies for 
reporting.  
 
Submission of annual progress reports will occur within 60 days of the anniversary of the date 
that the IND went into effect (date that clinical studies were permitted to begin). 
 
9) Significance: 
Management of chronic pruritus is notoriously challenging for patients with EB. In a 
comprehensive survey of 146 EB patients, itch was reported as the most bothersome EB 
symptom, ranking higher than acute or chronic pain or problems eating.1 Furthermore, pruritus 
can induce an itch-scratch-blister cycle, which can exacerbate existing wounds or induce new 
wounds.2 There have been no new drugs for chronic pruritus, and the current management of 
pruritus in EB with anti-histamines and topical steroids only minimally relieve itch.3 
 
It is our hope that serlopitant will reduce itch in EB adolescents and adults. However, in the 
process of conducting this clinical trial, we will obtain critical FDA feedback on the 
development of novel itch endpoints, valid instruments to measure itch, and an orphan drug 
disease product designation – all of these important milestones will reduce the regulatory hurdles 
for future clinical trials in EB itch and stimulate more drug development in this unmet area. 
 
10) Key references: 
1. Danial C AR, Gorell ES, Lucky AW, Paller AS, Bruckner A et al. Prevalence and 
Characterization of Pruritus in Epidermolysis Bullosa. Pediatric Dermatology 2015;32:6. 
2. Goldschneider KR, Lucky AW. Pain management in epidermolysis bullosa. Dermatol Clin 
2010;28:273-82, ix. 
3. Danial C AR, Gorell ES, Lucky AW, Paller AS, Bruckner A et al. Evaluation of Treatments 
for Pruritus in Epidermolysis Bullosa. Pediatric Dermatology 2014. 

ICF_000.pdf: 
 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 1 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
 
Please check all that are applicable: 
 
 I am an adult participant in this study. 
 
Print your name here: _______________________________________________ 
 
 
 I am the parent or guardian granting permission for a child in this study (the use of 
"you" refers to "your child" or “your ward.”) 
  
Print child’s name here: _____________________________________________ 
 
************* 
 
Are you participating in any other research studies? _____ Yes _____No 
 
PURPOSE OF RESEARCH 
 
You are invited to participate in a research study of chronic pruritus or long-lasting itch 
associated with epidermolysis bullosa (EB).  We hope to learn if a research medication 
known as Serlopitant is effective and safe to use in patients with EB and chronic pruritus. 
You were selected as a possible participant in this study because you have been 
diagnosed with EB and chronic pruritus (itch). 
 
Researchers want to find out more about an investigational drug called Serlopitant. An 
“investigational drug” is a drug that is being tested and is not approved for sale in the 
United States by the U.S. Food and Drug Administration (FDA). 
 
In addition, this study will compare doses of Serlopitant (5 mg) with a placebo to see if 
taking Serlopitant is better than taking a placebo. The placebo is a tablet that looks like a 
drug but has no drug or other active ingredient in it. Be aware that this form refers to 
Serlopitant as “study drug.” 
 
If you decide to terminate your participation in this study, you should notify Dr. Albert 
Chiou at (650) 721-8418. 
 
This research study is looking for 40 to 50 patients with EB and chronic itch. Enrollment 
will occur in the United States. Stanford University expects to enroll all research study 
participants.  
 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 2 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
VOLUNTARY PARTICIPATION  
 
Your participation in this study is entirely voluntary.  Your decision not to participate will 
not have any negative effect on you or your medical care.  You can decide to 
participate now but withdraw your consent later and stop being in the study without 
any loss of benefits or medical care to which you are entitled. 
 
DURATION OF STUDY INVOLVEMENT  
 
This research study is expected to take approximately 16 weeks, with up to 4 weeks of 
screening, 8 weeks of active treatment, and a follow up period of 4 weeks. A washout 
period of at least 30 days may be required for patients currently taking medication that 
is not allowed during the study. Enrollment for the entire study may take up to 16 
months. Stanford University will be responsible for analysis of study data. 
 
If you decide to be in this study and the study doctor says you can be in the study, 
your participation will last up to 16 weeks. 
 
You will visit the study center 2 times (Screening Visit and Month 2 after starting study 
medication) to have the procedures and tests described in this form. Your visits will be 
followed up by monthly calls (Month 1 and Month 3 after starting study medication) by 
a research coordinator or research team member in order to ask how you are doing and 
if you have any problems during the course of study. Ask the study doctor or study staff 
about your study visit schedule. 
 
PROCEDURES 
 
If you choose to participate, Dr. Chiou and his research study staff will start by 
screening you to see if you are eligible to participate in the study. 
 
Phone Screen (Day -60 to -30) 
• A member of the research team may call you to introduce this study. 
• We will ask questions to determine your eligibility. 
• If eligible and interested, you may give your contact information to us. A read-
only version of this consent form and a medical release form (if you are not a 
Stanford patient) will be mailed to you. 
• After the phone screen, we will review your medical history to make sure you 
meet eligibility requirements.  
• If you meet study criteria, we will contact you to schedule a Screening Visit at 
Stanford. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 3 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
Screening Visit (Day -30 at Stanford): screening procedures should be completed 
within 30 days prior to Baseline/Day 1 of study medication. This may be extended by at 
least 14 days if a medication washout is required. The following procedures are to be 
performed: 
• The study information will be reviewed with you and written consent will be 
obtained.  
• Inclusion/exclusion criteria will be reviewed in depth. 
• Demographic and background information will be collected; this includes your 
age, gender, race and ethnicity. 
• We will review your medical history including history of EB and allergies. 
• Information on all concurrent medications will be collected.  
• An abbreviated physical examination, including assessments of weight, height 
and skin will be performed.  
• Vital signs (blood pressure, pulse, temperature) will also be recorded. 
• If you are a female of childbearing potential, a urine sample will be collected for 
a pregnancy test. 
• Blood (up to 2 tablespoons) and urine samples for safety testing will be 
collected.  
• You will be provided with an itch diary where you will record your itch severity 
nightly until you receive the study medication in the mail. You will also receive a 
return envelope to send the itch diary back to us when you receive a new itch 
diary in the mail. 
• Photographs: If you give the study center permission to take your photograph, a 
member of the study staff will take photos of the affected area. We will 
designate a target wound area and photograph it. 
• Questionnaires: You will be asked to complete questionnaires about your quality 
of life, including your day to day activities. Questionnaires may be completed on 
paper or online. 
• In the case that you are eligible, you will be instructed on how and when to take 
the study medication, which will be mailed to you. 
• We will contact you when we can determine if you are eligible or not eligible for 
the study. 
 
The screening period may be extended by at least 14 days if a washout from 
medications which are not allowed while on study is required. If you decide to be in this 
study, you may have to stop taking your regular medication during the entire study.  
 
Make sure to tell the study staff about all of the medicines you take so you will know if 
any must be stopped. If you stop your regular medication to be on the study, your 
symptoms might come back or your health might get worse. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 4 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
Any blood or urine samples taken during this study will not be saved for future 
research. 
 
Baseline (Day 1 of treatment) 
During the screening period, if the exams, tests and procedures show that you can be 
in the study, and you choose to take part, then you will be mailed home the study 
medication and an Itch Diary to record your itch score. The medication package will 
include detailed instructions on how and when to take the study medication. Once you 
receive a new Itch Diary, you will mail back the previous Itch Diary in the return 
envelope provided during the Screening Visit (if applicable). 
 
Screening and Baseline visits may be combined. In this case you will be given an itch 
diary to record your nightly itch score for at least 7 days before starting Study Drug. 
 
Eligible patients will be randomly (like tossing a coin) assigned to receive one of the two 
different patterns of therapy. There will be a 50% chance of receiving either pattern of 
therapy. 
1. Approximately one half of the patients (20 -25) will receive Serlopitant: 5 mg 
once daily by mouth following a 3-tablet loading dose on Day 1. 
2. Approximately one half of the patients (20-25) will receive placebo (no active 
ingredient): Once daily by mouth, following a 3-tablet loading dose on Day 1. 
 
You have an equal chance of being in either of the study groups. 
 
Neither you nor the study doctor or the study staff will be able to pick which study 
group you are in. You will not know and the study doctor or study staff will not know 
which group you are in. The study doctor and study staff can find out if it is necessary 
to know for your health. If this happens, the study doctor or study staff may not be 
able to tell you which study group you were in until everyone finishes the study (which 
may be years in some cases). 
 
At the Baseline day, the following procedures are to be performed: 
• You will take a loading does of 3 tablets. Thereafter, you will take 1 tablet every 
day at bedtime for 8 weeks.  
• You will continue to fill out an itch diary where you will record your itch severity 
nightly and the time that you take your study medication.  
• Mail in Itch Diary. 
• If Screening and Baseline visits are combined, you will be given an itch diary to 
record your nightly itch score for 1 week (7 days) before starting Study Drug. 
 
 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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Phone Call at Month 1 
• You will be asked about any side effects you may have had and any medications 
you may have taken. 
• The study doctor or study staff will administer the questionnaires verbally and 
record your responses. Alternatively, questionnaires may be completed online. 
• You will receive a new itch diary. 
• If you are a female of childbearing potential, a urine sample will be collected for 
a pregnancy test. 
• Blood and urine samples will be collected for safety laboratory tests. Testing will 
be completed at a lab close to your home. 
• Additional medication will be dispensed after laboratory test results have been 
reviewed. 
 
End of Treatment Visit (Month 2, at Stanford) 
• You will be asked about any side effects, you may have had and any 
medications you may have taken. 
• Vital signs (blood pressure, pulse, temperature) will be assessed 
• An abbreviated physical examination, including assessments of weight, height, 
skin and lymph nodes will be performed. 
• Photographs will be taken (if you consented to photography). 
• You will be asked to complete your questionnaires. Questionnaires may be 
completed on paper or online. 
• Your itch diary will be reviewed and collected. 
• You will receive a new Itch Diary and a return envelope to send the itch diary 
back to us after the follow-up phone call at the end of Month 3. 
• Your study medication bottle will be collected. 
• If you are a female of childbearing potential, a urine sample will be collected for 
a pregnancy test. 
• Blood and urine samples will be collected for safety tests. 
 
Follow-up Period Phone visit at Month 3 
• You will be asked about any side effects, you may have had and any medications 
you may have taken. 
• The study doctor or study staff will administer the questionnaires verbally and 
record your responses. Alternatively, questionnaires may be completed online. 
• You will mail in your Itch Diary for the previous month in an envelope provided 
to you by the study staff. 
 
You will find out if you were on active drug or on placebo after the last study 
patient completes study treatment. At that time, you will be given the option to 
continue your study participation by taking active drug for 12 months.  
 


 
 
 
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If you would like to continue with the treatment,  
• you will be provided with the research medication and  
• research daily itch diary.  
• you will be followed up by phone at baseline, 1 month, 2 months, 6 months and 
12 months.  
• you will have no on-site visits. 
• you are required to perform lab tests at baseline, 1 month, 2 months, 6 months 
and 12 months while on the extension at a lab convenient to you. 
 
If you decide not to continue on active drug, the study doctor or study staff will call you 
for your follow up phone call and you will have completed the study. 
 
Women of Childbearing Potential 
If you are a woman who is able to become pregnant, it is expected that you will use an 
effective method of birth control to prevent exposing a fetus to a potentially dangerous 
agent with unknown risk.  If you are pregnant or currently breast feeding, you may not 
participate in this study.  You understand that if you are pregnant, if you become 
pregnant, or if you are breast-feeding during this study, you or your child may be 
exposed to an unknown risk. 
 
To confirm to the extent medically possible that you are not pregnant, you agree to have 
a pregnancy test done before beginning this research study.   
You must agree to avoid sexual intercourse or use a birth control method judged to be 
effective by the investigator and which will not interfere with the proposed investigation.   
 
Effective contraception is defined as follows: oral/implant/injectable/transdermal 
contraceptives, intrauterine device, condom with spermicide, or diaphragm with 
spermicide. Abstinence or partner’s vasectomy is acceptable if the female agrees to use 
effective contraception if she decides to discontinue abstinence or to have sexual 
intercourse with a non-vasectomized partner. 
 
You must accept the risk that pregnancy could still result despite the responsible use of 
reliable method of birth control.  You agree to notify the investigator as soon as possible 
of any failure of proper use of your birth control method, or if you become pregnant, 
either of which may result in your being withdrawn from  
the study. 
 
PHOTOGRAPHY 
 
If the skin on the area of your body that itches has redness, scratches, nodules, or 
lesions, you will be asked to allow photographs to be taken of the affected area(s). If 
you agree, photographs will be taken. This photography is optional. You can still be in 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
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the study even if you do not want the study doctor or study staff to take photographs 
of your affected areas of itch. Be aware that photographs taken during this study will be 
used for information purposes only. Your name will not be used if the photographs are 
shared at meetings or in publications. 
 
We will use these photographs to record changes in the affected area if the itching 
lessens or stops. All reasonable efforts will be made to protect your identity, including 
blocking your eyes with a black bar, or using only partial face photographs if the 
affected area is the face. 
 
CONSENT FOR PHOTOGRAPHY 
 
I agree to have photographs taken for the study. I further agree that any pictures taken 
of me during this study may be used for information purposes only, if considered 
appropriate, unless I notify the study doctor in writing that she is not to use these 
photographs prior to publication. All reasonable efforts will be made to protect my 
identity, including blocking of my eyes, or using only partial face photographs. Please 
check one box below: 
 
q Yes, I agree to allow the study doctor or study staff to photograph the area(s) of 
my skin affected by itching as described in this form. I also agree to the release 
and uses of the photographs as described above. 
 
q No, I do not agree to allow the study doctor or study staff to photograph the 
areas of my skin affected by itching during the study. I can still be in the rest of 
the study. 
 
PARTICIPANT RESPONSIBILITIES           
 
As a participant, your responsibilities include:  
• Follow the instructions of the Protocol Director and study staff. 
• Take the study drug as instructed – at bedtime without food, at least 2 
hours after your evening meal. 
• Complete your questionnaires as instructed. 
• You should avoid eating grapefruit while participating in this study. 
• Complete your itch diaries as instructed. 
• Have your blood drawn and urine collected for safety testing. Additional 
research drug cannot be dispensed until laboratory results are reviewed by the 
study doctor. 
• Keep your study appointments.  If it is necessary to miss an appointment, please 
contact the Protocol Director or research study staff to reschedule as soon as 
you know you will miss the appointment. 


 
 
 
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• Tell the Protocol Director or research study staff about any side effects, doctor 
visits, or hospitalizations that you may have. 
• Tell the Protocol Director or research staff if you believe you might be pregnant 
or gotten your partner pregnant. 
• Keep the study drug in a safe place, away from children and for your use only. 
• Ask questions as you think of them. 
• Tell the Protocol Director or research staff if you change your mind about staying 
in the study. 
 
WITHDRAWAL FROM STUDY 
 
If you first agree to participate and then you change your mind, you are free to 
withdraw your consent and discontinue your participation at any time.  Your decision 
will not affect your ability to receive medical care for your disease and you will not lose 
any benefits to which you would otherwise be entitled.  
 
If you decide to withdraw your consent to participate in this study, you should notify Dr. 
Chiou at (650) 721-8418. 
 
We will perform the following procedures if you decide to withdraw from this study: 
• You will be asked about any side effects, you may have had and any medications 
you may have taken 
• Vital signs (blood pressure, pulse, temperature) will be assessed 
• Photographs will be taken (if you consented to photography) 
• You will be asked to complete your questionnaires 
• Your itch diary will be reviewed and collected  
• If you are a female of childbearing potential, a urine sample will be collected for a 
pregnancy test (if leaving study prior to Month 1 visit)  
• Blood and urine samples will be collected for safety laboratory tests.  
• Blood samples will also be taken to assess the amount of research medication you 
have in your blood (if leaving study prior to Month 1 visit). 
• Your study medication bottle will be collected 
 
The Protocol Director may also withdraw you from the study and the study medication 
may be stopped, without your consent for one or more of the following reasons:   
o Failure to follow the instructions of the Protocol Director and study staff. 
o The Protocol Director decides that continuing your participation could be harmful 
to you. 
o Pregnancy. 
o You need treatment not allowed in the study. 
o The study is cancelled.  
o Other administrative reasons. 


 
 
 
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o Unanticipated circumstances. 
 
POSSIBLE RISKS, DISCOMFORTS, AND INCONVENIENCES  
 
There are risks, discomforts, and inconveniences associated with any research study.  
These deserve careful thought.  You should talk with the Protocol Director if you have 
any questions. 
 
You may have side effects from the drugs or procedures used in this study, and they 
will vary from person to person. Everyone taking part in the study will be watched 
carefully for any side effects. However, doctors do not know all the side effects that 
may happen, and there may be unknown side effects that occur. 
 
During this study, your blood will be drawn to perform a variety of tests. The risks of 
drawing blood include temporary discomfort from the needle in your arm, bruising, 
swelling at the needle site and, in rare instances, infection. 
 
To date, over 1000 subjects have been studied on Serlopitant in previous studies. Most 
of the side effects were mild or moderate.  
 
The most commonly reported side effect was headache. 
 
Other most commonly reported (>2% of patients) side effects include: 
diarrhea 
dizziness 
urinary tract infection 
nausea 
drowsiness 
cough, upper respiratory 
tract infection 
dry mouth 
tiredness 
back pain 
irritation of the nose and 
mouth 
high blood pressure 
irritation of the sinuses 
 
 
Less commonly reported (<2% of patients) side effects include: 
vomiting 
disturbance in attention 
pain in the nose and 
mouth 
abdominal pain 
irritation of the eye 
joint pain 
heart burn 
itch 
bone pain 
 
The following rare but serious side effects were reported; 
• spontaneous abortion or miscarriage occurred in one patient taking a low dose of 
Serlopitant (5 times lower dose than in this study). This event was considered 
not related to study treatment. 


 
 
 
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Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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• hypothyroidism (under active thyroid gland) in one patient taking Serlopitant at a 
very low dose (20 times lower than in this study) and 
• peripheral neuropathy (weakness, numbness and pain from nerve damage) in 
one patient taking Serlopitant at the same dose as in this study. Both of these 
events were considered not related to study treatment. 
• recurrent depression with suicidal thoughts, was reported in July 2015, that was 
not related to the drug and caused the patient to be hospitalized for a long 
period of time.  
In a recent study conducted at Stanford the following mild or moderate side effects 
were reported in patients treated with Serlopitant at 5 mg per day; headache, nausea, 
dizziness, skin infection, chest infection, diarrhea, cold, cough, pain, toothache and flu-
like symptoms. 
An unexpected serious side effect of pulmonary embolism (blood clot in the lung) was 
reported in May 2018, which was determined not to be related to the drug, and 
resulted in death. 
Data collected to date in young adult animals and in adult clinical trial patients taking 
Serlopitant support clinical trials in adolescent patients. 
If you decide to enroll in this study, you may need to stop taking certain medications 
prior to starting the study. Additionally, once you start taking the study medication, you 
should not change which medications you are taking, or how much of your medications 
you are taking. If you feel that you must change your medications while enrolled in the 
study, we may need to end your participation.   
 
Please tell the study doctor or study staff right away if you have any side 
effects. Please tell them if you have any other problems with your health or 
the way you feel during the study, whether or not you think these problems 
are related to the study drug. 
 
Sometimes people have allergic reactions to drugs or product ingredients. If you have a 
very bad allergic reaction, you could die. Some things that happen during an allergic 
reaction that could be a sign or a symptom of a life-threatening allergic reaction 
(anaphylaxis) are: 
• A rash 
• Having a hard time breathing 
• Wheezing 
• A sudden drop in blood pressure (making you feel dizzy or lightheaded) 
• Swelling around the mouth, throat, or eyes 
• A fast pulse 
• Sweating 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
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Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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You should get medical help and contact the study doctor or study staff if you have any 
of these or any other side effects during the study. 
 
If you decide to be in this study, you might have to continue to take your current 
medications and you may not be able to add any additional medications. Make sure to 
tell the study staff about all the medicines you take so you will know if any must 
continue throughout the study. If you stop your regular medication to be in the study, 
your symptoms might come back or your health might get worse. 
 
Some people in the study will get placebo instead of Serlopitant. Taking placebo is the 
same as not taking anything for your chronic itch. If you take placebo during this study, 
it is possible that your chronic itch may get worse. 
 
Completing questionnaires could lead you to feel uncomfortable or upset. Please tell the 
study doctor or study staff if you feel uncomfortable or upset while completing the 
questionnaires. You have the right to refuse to answer any question.  
 
You should talk to the study doctor or research staff if you have any questions. 
 
POTENTIAL BENEFITS                              
 
Taking part in this study may or may not cause your health to improve. Information 
from this study may help doctors learn more about Serlopitant and the treatment of 
chronic itch in patients with EB. This information may benefit other patients with 
chronic itch related to EB. 
 
We cannot and do not guarantee or promise that you will receive any benefits from this 
study. 
 
ALTERNATIVES 
 
You do not have to be in this study to get help for your chronic itch. Although most 
common treatments for itch have not worked for you, some other things you may be 
able to consider are: 
 
• Anticonvulsants and gabapentin 
• Opioid receptor agonists and antagonists 
• Antidepressants such as SSRIs 
• Alternative wound care dressings 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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You should discuss your alternatives to participating in research with the study doctor 
or study staff. In addition, you may discuss your options with your regular health care 
provider. 
 
PARTICIPANT’S RIGHTS 
 
You should not feel obligated to agree to participate.  Your questions should be 
answered clearly and to your satisfaction.  If you decide not to participate, tell the 
Protocol Director.   
 
You will be told of any important new information that is learned during the course of 
this research study, which might affect your condition or your willingness to continue 
participation in this study. 
 
ClinicalTrials.gov 
 
A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as 
required by U.S. Law.  This Web site will not include information that can identify 
you.  At most, the Web site will include a summary of the results.  You can search this 
Web site at any time. 
 
CONFIDENTIALITY 
 
The results of this research study may be presented at scientific or medical meetings or 
published in scientific journals.  Your identity and/or your personal health information 
will not be disclosed except as authorized by you or as required by law.  However, there 
is always some risk that even de-identified information might be re-identified.  
 
Patient information may be provided to Federal and other regulatory agencies as 
required.  The Food and Drug Administration (FDA), for example, may inspect research 
records and learn your identity if this study falls within its jurisdiction. 
 
The purpose of this research study is to obtain data or information on the safety and 
effectiveness of Serlopitant; the results will be provided to the sponsor, the Food and 
Drug Administration and other federal and regulatory agencies as required. 
 
We may schedule blood tests for you at a local lab to run safety lab tests. We will need 
to provide information on your identity (name, date of birth, address, etc.) to order 
these tests. 
 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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Authorization To Use Your Health Information For 
Research Purposes  
Because information about you and your health is personal and private, it 
generally cannot be used in this research study without your written 
authorization.  If you sign this form, it will provide that authorization.  The 
form is intended to inform you about how your health information will be 
used or disclosed in the study.  Your information will only be used in 
accordance with this authorization form and the informed consent form 
and as required or allowed by law.  Please read it carefully before signing 
it.   
 
What is the purpose of this research study and how will my health 
information be utilized in the study? 
The purpose of this study is to learn if a research medication known as 
Serlopitant is effective and safe to use in patients with EB and chronic itch. 
The study doctor and study staff may carry out certain statistical tests on 
your information, along with the information collected from the other 
patients who entered the study. The study doctor may report results at 
medical meetings and in medical magazines, so that other doctors can find 
out about the results of the study. The results of this study may be used 
for future medical research.  
 
Do I have to sign this authorization form? 
You do not have to sign this authorization form.  But if you do not, you will 
not be able to participate in this research study, including receiving any 
research-related treatment.  Signing the form is not a condition for 
receiving any medical care outside the study. 
 
If I sign, can I revoke it or withdraw from the research later? 
If you decide to participate, you are free to withdraw your authorization 
regarding the use and disclosure of your health information (and to 
discontinue any other participation in the study) at any time.  After any 
revocation, your health information will no longer be used or disclosed in 
the study, except to the extent that the law allows us to continue using 
your information (e.g., necessary to maintain integrity of research).  If you 


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
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Albert Chiou MD/MBA 
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wish to revoke your authorization for the research use or disclosure of your 
health information in this study, you must write to: Dr. Albert Chiou,  
Stanford University, Dermatology Dept., 450 Broadway St. Pavilion C 
MS:5334, Redwood City, CA 94063 
 
What Personal Information Will Be Obtained, Used or Disclosed? 
Your health information related to this study, may be used or disclosed in 
connection with this research study, including, but not limited to, your 
name, address, phone number, or social security number. Your records 
include: 
• Medical records 
• Medical history 
• Physical exams 
• Laboratory results 
• Photographs (if you agreed to participate in the study photography) 
• Interviews and/or questionnaires 
• Information from other procedures you have as part of the study 
 
Who May Use or Disclose the Information? 
The following parties are authorized to use and/or disclose your health 
information in connection with this research study: 
• The Protocol Director, Dr. Albert Chiou 
• The Stanford University Administrative Panel on Human Subjects in 
Medical Research and any other unit of Stanford University as 
necessary 
• Research Staff 
 
Who May Receive or Use the Information? 
The parties listed in the preceding paragraph may disclose your health 
information to the following persons and organizations for their use in 
connection with this research study:  
• The Office for Human Research Protections in the U.S. Department of 
Health and Human Services 
• The funding agency, Epidermolysis Bullosa Research Partnership, and 
anyone acting on its behalf 


 
 
 
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Protocol Director: 
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Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
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• Menlo Therapeutics Inc., the company providing the study 
medication 
• The Food and Drug Administration 
• Quest Diagnostics 
Your information may be re-disclosed by the recipients described above, if 
they are not required by law to protect the privacy of the information. 
 
When will my authorization expire? 
Your authorization for the use and/or disclosure of your health information 
will end on December 31, 2045 or when the research project ends, 
whichever is earlier.  
 
Will access to my medical record be limited during the study? 
To maintain the integrity of this research study, you may not have access 
to any health information developed as part of this study until it is 
completed.  At that point, you would have access to such health 
information if it was used to make a medical or billing decision about you 
(e.g., if included in your official medical record).  
 
 
________________________________             
  ______________ 
Signature of Adult Participant                                        Date 
 
 
________________________________             
 
Print Name of Adult Participant                                         
 
 
_______________________________                       ______________ 
Signature of Legally Authorized Representative (LAR)          Date 
(e.g., parent, guardian or conservator) 
 
 
________________________________      ____________________________      
Print Name of LAR                                    LAR’s Authority to Act for Participant  
 
   


 
 
 
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FINANCIAL CONSIDERATIONS 
 
Payment/Reimbursement 
The study will cover reasonable costs for transportation and housing. If you stay 
overnight for a research visit, the study will also reimburse you per diem for food 
(about $50 per day per person). 
 
You will be paid $75 per visit to help support your participation in this trial. The 
maximum you may receive is $225. 
 
Payments may only be made to U.S. citizens, legal resident aliens, and those who have 
a work eligible visa. You may need to provide your social security number to receive 
payment.   
 
 
Costs 
If you participate in this study, the study will pay for those services, supplies, 
procedures, and care associated with the study that are not a part of your routine 
medical care.  However, there may be additional costs to you.  These include basic 
expenses like transportation and the personal time it will take to come to the study 
visits.  You and/or your health insurance must pay for services, supplies, procedures, 
and care that are required during this study for routine medical care.  You will also be 
responsible for any co-payments and/or deductibles as required by your 
insurance.  Participation in this study is not a substitute for health insurance.   
 
Sponsor 
EB Research Partnership is providing financial support and Menlo Therapeutics Inc. is 
providing study drug for this study.  
 
COMPENSATION for Research-Related Injury 
 
All forms of medical diagnosis and treatment – whether routine or experimental – 
involve some risk of injury.  In spite of all precautions, you might develop medical 
complications from participating in this study.  If such complications arise, the Protocol 
Director and the research study staff will assist you in obtaining appropriate medical 
treatment.  In the event that you have an injury or illness that is directly caused by 
your participation in this study, reimbursement for all related costs of care first will be 
sought from your insurer, managed care plan, or other benefits program.  You will be 
responsible for any associated co-payments or deductibles as required by 
your insurance. 
 
If costs of care related to such an injury are not covered by your insurer, managed care 
plan or other benefits program, you may be responsible for these costs.  If you are 


 
 
 
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unable to pay for such costs, the Protocol Director will assist you in applying for 
supplemental benefits and explain how to apply for patient financial assistance from the 
hospital. 
 
You do not waive any liability rights for personal injury by signing this form. 
 
CONTACT INFORMATION  
 
Independent Contact:  If you are not satisfied with how this study is being conducted, 
or if you have any concerns, complaints, or general questions about the research or 
your rights as a participant, please contact the Stanford Institutional Review Board 
(IRB) to speak to someone independent of the research team at (650)-723-5244 or toll 
free at 1-866-680-2906.  You can also write to the Stanford IRB, Stanford University, 
3000 El Camino Real, Five Palo Alto Square, 4th Floor, Palo Alto, CA 94306. 
 
Appointment Contact: If you need to change your appointment, please contact Melissa 
Barriga at 650-736-8033.  
 
Alternate Contact:  If you cannot reach the Protocol Director, please contact Irene 
Bailey at 650-721-7149. 
 
Questions, Concerns, or Complaints:  If you have any questions, concerns or complaints 
about this research study, its procedures, risks and benefits, or alternative courses of 
treatment, you should ask the Protocol Director, Dr. Albert Chiou at (650) 721-8418. 
You should also contact him at any time if you feel you have been hurt by being a part 
of this study.  
 
EXPERIMENTAL SUBJECT’S BILL OF RIGHTS 
 
As a research participant you have the following rights.  These rights include but are 
not limited to the participant's right to: 
• 
be informed of the nature and purpose of the experiment;  
• 
be given an explanation of the procedures to be followed in the medical 
experiment, and any drug or device to be utilized;  
• 
be given a description of any attendant discomforts and risks reasonably to be 
expected;  
• 
be given an explanation of any benefits to the subject reasonably to be 
expected, if applicable;  
• 
be given a disclosure of any appropriate alternatives, drugs or devices that might 
be advantageous to the subject, their relative risks and benefits; 
• 
be informed of the avenues of medical treatment, if any available to the subject 
after the experiment if complications should arise;  


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 18 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
• 
be given an opportunity to ask questions concerning the experiment or the 
procedures involved;  
• 
be instructed that consent to participate in the medical experiment may be 
withdrawn at any time and the subject may discontinue participation without 
prejudice;  
• 
be given a copy of the signed and dated consent form; and  
• 
be given the opportunity to decide to consent or not to consent to a medical 
experiment without the intervention of any element of force, fraud, deceit, 
duress, coercion or undue influence on the subject's decision. 
 
May we contact you about future studies that may be of interest to you?   £ Yes £ No 
 
Signing your name means you agree to be in this study and that you will receive a copy 
of this signed and dated consent form.  
 
 
_____________________________________                ____________ 
Signature of Adult Participant 
 
 
 
   
   Date 
 
_____________________________________             
Print Name of Adult Participant                                         
 
_____________________________________                ____________         
Signature of Legally Authorized Representative (LAR)  
  Date  
(e.g., parent, guardian or conservator)    
 
____________________________________             ______________________________ 
Print Name of LAR                                          
LAR’s Authority to Act for Participant  
 
 
 
 
 
 
 
 
(e.g., parent, guardian or conservator) 
 
The IRB determined that the permission of two parents is recommended in accordance with 21 CFR 50.55 
unless one parent is deceased, unknown, incompetent, not reasonably available, or only one parent has legal 
responsibility for the care and custody of the child.  Not reasonably available means that the other parent is 
not present during the consenting process, or will not be available prior to the start of research procedures. 
 
 
_____________________________________                ____________  
(If available) Signature of Other Parent or Guardian               Date                         
 
_____________________________________           ______________________________ 
Print Name of Other Parent or Guardian                    Authority to Act for Participant  
 
 
 
 
                     


 
 
 
STANFORD UNIVERSITY   Research Consent Form 
Protocol Director: 
Albert Chiou MD/MBA 
Protocol Title:  A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Patients with Epidermolysis Bullosa  
 
 
Page 19 of 19 
SU_MainICF_HIPAA rev 041218 
IRB Use Only 
Approval Date:   April 17, 2019 
Expiration Date:  February 5, 2020 
Participant ID: 
 
STANFORD PERSONNEL ONLY 
 
 
 
_____________________________________                ____________  
Signature of Person Obtaining Consent  
 
   
  Date 
 
 
_____________________________________             
Print Name of Person Obtaining Consent 
 
 
 
 
The following witness line is to be signed only if the consent is provided as a summary form and 
accompanied by a short form foreign language consent. 
 
 
_____________________________________                     ____________ 
Signature of Witness                                                              Date 
 
 
__________________________________              
Print Name of Witness                                         
 
(e.g., staff, translator/interpreter, family member) 
 
• Translated short form must be signed and dated by both the participant (or their LAR) AND 
the witness. 
• The English consent form (referred to as the "Summary Form" in the regulations): 
- Must be signed by the witness AND the Person Obtaining Consent (POC). 
- The non-English speaking participant/LAR does not sign the English consent. 
- The non-English speaking participant/LAR should not sign the HIPAA participant line 
- If the participant or the LAR is non-English speaking, the Person Obtaining Consent (POC) 
must ensure that 1) the LAR's Description of Authority is completed and 2) that any 
questions or options presented by the consent form are documented and initialed by the 
POC on the Summary Form, per the participant's wishes, as they are understood during the 
consent process.