Clinical trial NCT03495817
Conditions: Androgenetic Alopecia
Sponsor: Aclaris Therapeutics, Inc.
Start date: 2018-03-22; Completion date: 2019-10-30
Enrollment: 31 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
Open label study to assess safety, tolerability, and efficacy of ATI-50002 in male and female subjects with androgenetic alopecia.

Detailed description:
This is an open-label study designed to evaluate the safety and efficacy of ATI-50002 Topical Solution in male and female subjects with androgenetic alopecia.

Arms: arm 1: This is an open-label phase 2 study designed to evaluate the safety and efficacy of ATI- 50002 Topical Solution, 0.46% in male and female subjects with androgenetic alopecia.

Subjects will be required to apply ATI-50002 study medication to their scalp twice a day for a total of 26 weeks.
Interventions: intervention 1: ATI-50002
Intervention descriptions: intervention 1: ATI-50002 Topical Solution

Protocol documents:
SAP_002.pdf: 
STATISTICA工ANAIIYS萱S PエAN¯○○ MOCK UP DISPLAYS
Title: An Open­LabeI Safety, ToIerability, and E鮪cacy Study in Male and Female
Subjects with Androgenetic AIopeda Treated with ATI­50002 Topical Solution
¯Protoc 61三　　　ATIこ50 0 02こAGAニ20】「
S叫D剛g: ATト5000之
Spon§Or:　Aclaris Therapentics, Inc.
臆Da紅:　　　2う­Apri1 20帆
Statu§:　　　Fina1 1.0
PreDared bv:
圏国圏堅田
Da書e‥幽仔


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
2 
END-OF-TEXT TABLES 
 
Number 
Title 
Table 14.1.1.1 
 Study Population and Patient Disposition 
Table 14.1.1.2 
 Reasons for Discontinuation from Study ITT Population 
Table 14.1.2 
 Patient Demographics and Other Baseline Characteristics ITT Population 
Table 14.1.3 
 Diagnosis and Prior Treatments for Androgenetic Alopecia Safety 
Population 
Table 14.1.4 
 Exposure to Study Medication Safety Population 
Table 14.1.5 
 Exposure to Study Medication Safety Population 
Table 14.2.1.1 
 Non-Vellus Target Area Hair Count (TAHC): Mean and Mean Change 
from Baseline (hairs/cm2) ITT Population 
Table 14.2.1.2 
 Vellus Target Area Hair Count: Mean and Mean Change from Baseline 
(hairs/cm2) ITT Population 
Table 14.2.1.3 
 Total (Non-Vellus + Vellus) Target Area Hair Count: Mean and Mean 
Change from Baseline (hairs/cm2) ITT Population 
Table 14.2.1.4 
 Non-Vellus Target Area Hair Width: Mean and Mean Change from 
Baseline (cm) ITT Population 
Table 14.2.1.5 
 Vellus Target Area Hair Width: Mean and Mean Change from Baseline 
(cm) ITT Population 
Table 14.2.1.6 
 Total (Non-Vellus + Vellus) Target Area Hair Width: Mean and Mean 
Change from Baseline (cm) ITT Population 
Table 14.2.1.7 
 Average Non-Vellus Hair Width: Mean and Mean Change from Baseline 
(cm) ITT Population 
Table 14.2.2.1 
 Investigator Global Assessment ITT Population 
Table 14.2.2.2 
 Subject Self-Assessment ITT Population 
Table 14.2.3.1 
 Investigator Global Assessment: Patients with Slightly Increased Hair 
Growth or Better (Grade +1 to +3)  ITT Population 
Table 14.2.3.2 
 Subject Self-Assessment: Patients with Slightly Increased Hair Growth or 
Better (Grade +1 to +3) ITT Population 
Table 14.2.4 
 Improvement from Baseline in Norwood-Hamilton Scale in Males ITT 
Population 
Table 14.2.5 
 Improvement from Baseline in Sinclair Scale in Females Safety 
Population 
Table 14.2.6 
 Correlation of Selected Effiacy Measures  ITT Population 
Table 14.2.7 
 Correlation of Nonvellus Hair Count and Width Measures with Time 
Since Diagnosis of Androgenetic Alopecia  ITT Population 
Table 14.3.1 
 Summary of Treatment-Emergent Adverse Events  Safety Population 
Table 14.3.1.1 
 Treatment-Emergent Adverse Events by System Organ Class and 
Preferred Term  Safety Population 
Table 14.3.1.2 
 Treatment-Emergent Study Treatment-Related Adverse Events by System 
Organ Class and Preferred Term Safety Population 
Table 14.3.1.3 
 Treatment-Emergent Adverse Events Resulting in Discontinuation of 
Study Treatment by System Organ Class and Preferred Term Safety 
Population 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
3 
Table 14.3.1.4 
 Treatment-Emergent Serious Adverse Events by System Organ Class and 
Preferred Term Safety Population 
Table 14.3.1.5 
 Treatment-Emergent Adverse Events by Descending Order of Frequency 
for ATI-50002  Safety Population 
Table 14.3.1.6 
 Treatment-Emergent Adverse Events by Maximum Severity Safety 
Population 
Table 14.3.1.7 
 Treatment-Emergent Study Medication Related Adverse Events by 
Maximum Severity Safety Population 
Table 14.3.1.8 
 Local Skin Reactions – Highest Grade Any Time Post-Baseline 
Safety Population 
Table 14.3.1.9 
 Treatment-Emergent Adverse Events by System Organ Class and 
Preferred Term in Patients Who Discontinued in First 26 Weeks Safety 
Population 
Table 14.3.1.10 
 Treatment-Emergent Adverse Events by System Organ Class and 
Preferred Term in Patients Who Completed 26 Weeks and Did not Enter 
Six Month Extension Safety Population 
Table 14.3.1.11 
 Treatment-Emergent Adverse Events by System Organ Class and 
Preferred Term in Patients Who Completed 26 Weeks and Entered Six 
Month Extension Safety Population 
Table 14.3.2.1 
 Vital Signs: Systolic Blood Pressure (mmHg) Safety Population 
Table 14.3.2.2  
 Vital Signs:  Diastolic Blood Pressure (mmHg) Safety Population 
Table 14.3.2.3  
 Vital Signs:  Heart Rate (bpm) Safety Population 
Table 14.3.2.4  
 Vital Signs:  Respiratory Rate (bpm) Safety Population 
Table 14.3.2.5  
 Vital Signs:  Temperature (Degrees C) Safety Population 
Table 14.3.3 
 ECG Interpretation: Shift from Baseline to Worst Assessment Post 
Baseline  Safety Population 
Table 14.3.4.1 
 Descriptive Statistics for Chemistry Parameters Safety Population 
Table 14.3.4.2 
 Descriptive Statistics for Hematology Parameters Safety Population 
Table 14.3.4.3 
 Descriptive Statistics for Hormone Parameters Safety Population 
Table 14.3.5 
 Number and Percent of Patients Meeting Criteria for Hepatobiliary 
Laboratory Abnormalities  Safety Population 
Table 14.3.6 
 Number and Percent of Patients Meeting Laboratory Criteria for Potential 
Renal Impairment Safety Population 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
4 
 
 
 
END-OF-TEXT FIGURES 
 
Number 
Title 
 
Figure 14.1 
 Association of Selected Efficacy Measures with Fit Linear Regression 
Line 
Figure 14.2  
Association of Selected Efficacy Measures with Fit Linear Regression 
Line, ITT Population: Visit=V7 Week 16 
Figure 14.3  
Association of Selected Efficacy Measures with Fit Linear Regression 
Line, ITT Population: Visit=V10 Week 26 
Figure 14.4  
Association of Selected Efficacy Measures with Fit Linear Regression 
Line, ITT Population: Visit=V16 Week 52 
Figure 14.5  
Association of Change in Nonvellus Target Area Average Hair Width with 
Investigator Global Assessment, ITT Population 
Figure 14.6  
Association of Change in Nonvellus Target Area Average Hair Width with 
Subject Self Assessment, ITT Population 
Figure 14.7  
Association of Change in Nonvellus Target Area Hair Count with Time 
Since Diagnosis of Androgenetic Alopecia, ITT Population 
Figure 14.8  
Association of Change in Nonvellus Target Area Hair Width with Time 
Since Diagnosis of Androgenetic Alopecia, ITT Population 
Figure 14.9  
Association of Change in Nonvellus Target AreaAverage Hair Width with 
Time Since Diagnosis of Androgenetic Alopecia, ITT Population 
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
5 
PATIENT DATA LISTINGS 
 
Number 
Title 
 
Appendix 16.2.1.1 
 Disposition 
Appendix 16.2.1.2 
 Demographics 
Appendix 16.2.1.3 
 History of Androgenetic Alopecia: Diagnosis and Prior Treatment and 
Therapies 
Appendix 16.2.1.4 
 Medical History 
Appendix 16.2.1.5 
 Prior and Concomitant Therapies 
Appendix 16.2.1.6 
 ATI-50002 Accountability 
Appendix 16.2.1.7 
 ATI-50002 Accountability Comments 
Appendix 16.2.1.8 
 ATI-50002 Application Changes and Missed Doses 
Appendix 16.2.1.9 
 Eligibility Criteria 
Appendix 16.2.1.10 
 Protocol Violations 
Appendix 16.2.2.1 
 Target Area Hair Count and Width 
Appendix 16.2.2.2 
 Investigator and Subject Global Assessments 
Appendix 16.2.2.3 
 Norwood-Hamilton Scale in Males 
Appendix 16.2.2.4 
 Sinclair Scale in Females 
Appendix 16.2.3.1  
 All Adverse Events 
Appendix 16.2.3.2 
Serious Adverse Events 
Appendix 16.2.3.3 
Adverse Events Resulting in Discontinuation of Study Treatment 
Appendix 16.2.3.4 
Adverse Events Resulting in Outcome of Death 
Appendix 16.2.3.5 
 Local Skin Reactions 
Appendix 16.2.3.6 
 Physical Exam 
Appendix 16.2.3.7 
 Vital Signs 
Appendix 16.2.3.8 
 Electrocardiogram – Evaluator Interpretation 
Appendix 16.2.3.9 
 Clinical Laboratory Assessments: Hematology 
Appendix 16.2.3.10 
 Clinical Laboratory Assessments: Biochemistry 
Appendix 16.2.3.11 
 Clinical Laboratory Assessments: Hormones 
Appendix 16.2.3.12 
 Clinical Laboratory Assessments: Urinalysis 
Appendix A 
 MedDRA System Organ Class/Preferred Term and CRF Verbatim Terms 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
6 
 
MOCKUPS OF TABLES, AND PATIENT LISTINGS 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
7  
Table 14.1.1.1 
 
Study Population and Patient Disposition 
 
 
 
 
Female 
n (%) 
 
Male 
n   (%) 
 
Total 
n   (%) 
 
 
 
 
 
Population/Group 
 
 
 
 
Intent-to-Treat (ITT) Population 
 
xx (100) 
xx (100 
xx (100 
Safety Population 
 
xx (100) 
xx (100 
xx (100 
 
 
 
 
 
Entered 6 Month Extension 
 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Percentages based on the number of patients receiving at least 1 treatment application  


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
8  
Table 14.1.1.2 
 
Reasons for Discontinuation from Study 
ITT Population 
 
 
Female 
(N=xxx) 
n (%) 
Male 
(N=xxx) 
n   (%) 
Total 
(N=xxx) 
n   (%) 
 
 
 
 
ENTIRE STUDYa: 
 
 
 
 
 
 
 
Number (%) of Patients Discontinued 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Protocol Violation 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        AE/SAE 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Lost to Follow-Up 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Withdrew Consent 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Pregnancy 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Other 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Completed 26 Week Study 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Completed Optional 6 Month Extension 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Ongoing as of “cut-off date” 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
 
 
 
FIRST 26  WEEKS: 
 
 
 
 
 
 
 
Number (%) of Patients Discontinued 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Protocol Violation 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        AE/SAE 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Lost to Follow-Up 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Withdrew Consent 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Pregnancy 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Other 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Completed  
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Ongoing as of “cut-off date” 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
a Includes all discontinuations/completions from the first 26 weeks  and optional 6 month extension.  Patents completing both the initial 26 
weeks and the 6 month extension periods are counted on both corresponding rows. 
b Used as denominator for percentages calculated under 6 month extension. 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
9  
Table 14.1.1.2 
 
Reasons for Discontinuation from Study 
ITTSafety Population 
 
 
Female 
(N=xxx) 
n (%) 
Male 
(N=xxx) 
n   (%) 
Total 
(N=xxx) 
n   (%) 
 
 
 
 
6 MONTH EXTENSION: 
 
 
 
 
 
 
 
Number of Patients Enteringb 
xx 
xx 
xx 
 
 
 
 
Number (%) of Patients Discontinued 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Protocol Violation 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        AE/SAE 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Lost to Follow-Up 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
        Withdrew Consent 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Pregnancy 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
Other 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Completed  
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
Ongoing as of “cut-off date” 
xx  (xx.x) 
xx  (xx.x) 
xx  (xx.x) 
 
 
 
 
 
a Includes all discontinuations/completions from the first 26 weeks and optional 6 month extension.  Patents completing both the initial 26 
weeks and the 6 month extension periods are counted on both corresponding rows. 
b Used as denominator for percentages calculated under 6 month extension. 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
10  
  
Table 14.1.2 
 
Patient Demographics and Other Baseline Characteristics 
ITT Population 
 
 
Female 
(N=xxx) 
Male 
(N=xxx) 
Total 
(N=xxx) 
 
 
 
 
Age (years) 
 
 
 
       n  
xx 
xx 
xx 
       Mean 
xx.x 
xx.x 
xx.x 
       SD 
xx.xx 
xx.xx 
xx.xx 
       Median 
xx.x 
xx.x 
xx.x 
      Min, Max 
xx,xx 
xx,xx 
xx,xx 
 
 
 
 
Age Category (years), n (%) 
 
 
 
      < 35 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
      ≥ 35 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Gender, n (%) 
 
 
 
 
Male 
 
 
xx (xx.x) 
 
Female 
 
 
xx (xx.x) 
 
 
 
 
Race, n (%) 
 
 
 
 
White 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Black or African American 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Asian 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
American Indian or Alaska Native 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Native Hawaiian or Other Pacific Islander 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Other 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Ethnicity, n (%) 
 
 
 
      Hispanic or Latino 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
      Non-Hispanic or Latino 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
      Not Reported 
 
 
 
                     


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
11  
Table 14.1.2 
Patient Demographics and Other Baseline Characteristics 
ITT Population 
 
 
Female 
(N=xxx) 
Male 
(N=xxx) 
Total 
(N=xxx) 
 
 
 
 
Height (cm) 
 
 
 
      n 
xx 
xx 
xx 
      Mean 
xx.x 
xx.x 
xx.x 
      SD 
xx.xx 
xx.xx 
xx.xx 
      Median 
xx.x 
xx.x 
xx.x 
      Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
Weight (kg) 
 
 
 
 
N 
xx 
xx 
xx 
 
Mean  
xx.x 
xx.x 
xx.x 
       SD 
xx.xx 
xx.xx 
xx.xx 
       Median 
xx.x 
xx.x 
xx.x 
       Min, Max 
 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
Fitzpatrick Skin Type, n (%) 
 
 
 
        1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
        2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
        3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
        4 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
        5 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
        6 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Sinclar Grade (Females), n (%) 
 
 
 
        2 
xx (xx.x) 
 
 
        3 
xx (xx.x) 
 
 
        4 
xx (xx.x) 
 
 
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
12  
Table 14.1.2 
Patient Demographics and Other Baseline Characteristics 
ITT Population 
 
 
Female 
(N=xxx) 
Male 
(N=xxx) 
Total 
(N=xxx) 
 
 
 
 
Norwood-Hamilton Classificiation (Males), n (%) 
 
 
 
        III vertex 
 
xx (xx.x) 
 
        IV 
 
xx (xx.x) 
 
        IVa 
 
xx (xx.x) 
 
        V 
 
xx (xx.x) 
 
 
 
 
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
13  
  
Table 14.1.3 
 
Diagnosis and Prior Treatments for Androgenetic Alopecia 
Safety Population 
 
 
Female 
(N=xx) 
Male 
(N=xx) 
Total 
(N=xx) 
 
 
 
 
Prior Therapies for Androgenetic Alopecia, n (%) 
 
 
 
      ANY therapy 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Minoxidil 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Finasteride 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Device 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Other 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
{Will need to review for appropriate categories} 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Time since Diagnosis of Androgenetic Alopecia  (Weeks)a 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
Age at Diagnosis of Androgenetic Alopecia  (Years) 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
a (date of first application of study medication – date of diagnosis)/7.


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
14  
 
  
Table 14.1.4 
 
Exposure to Study Medication 
Safety Population 
 
 
Parameter 
Female 
(N=xx) 
Male 
(N=xx) 
Total 
(N=xx) 
 
 
 
 
Duration of Treatment (Weeks)a 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
Total Weight Administered (gm) 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
Total Volume Administered (mL) 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
a (Date of last application of study medication – date of first application of study medication +1 ) / 7. Includes exposure in optional six month extension, 
if applicable. 
b Each patient’s average volume across all applications is calculated first. 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
15  
Table 14.1.5 
 
Exposure to Study Medication 
Safety Population 
 
 
Parameter 
Female 
(N=xx) 
Male 
(N=xx) 
Total 
(N=xx 
 
 
 
 
Total Number of Applications (Total of Each Patient) 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
       Total Applications (All Patients Total) 
xxxx 
xxxx 
xxxx 
 
 
 
 
Average Volume Per Application (mL)b 
 
 
 
        N 
xx 
xx 
xx 
        Mean 
xx.x 
xx.x 
xx.x 
        SD 
xx.xx 
xx.xx 
xx.xx 
        Median 
xx.x 
xx.x 
xx.x 
        Min, Max 
xx, xx 
xx, xx 
xx, xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a (Date of last application of study medication – date of first application of study medication +1 ) / 7. Includes exposure in optional six month extension, 
if applicable. 
b Each patient’s average volume across all applications is calculated first. 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
16  
Table 14.2.1.1 
 
Non-Vellus Target Area Hair Count (TAHC): Mean and Mean Change from Baseline (hairs/cm2) 
ITT Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
  
 
  
 
Baselinea 
n 
xx 
 
 
xx 
 
 
xx 
 
 
Mean (SD) 
xx.x (xx.xx)  
 
 
xx.x (xx.xx)  
 
 
xx.x (xx.xx)  
 
 
SE 
x.xx 
 
 
x.xx 
 
 
x.xx 
 
 
Median 
xxx.x 
 
 
xxx.x 
 
 
xxx.x 
 
 
(Min, Max) 
(xxx ,xxx) 
 
 
(xxx ,xxx) 
 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
 
V5 – Week 8 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
SE 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
[95% CI]b 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
 
 
 
 
 
 
 
 
V7– Week 16 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
SE 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
[95% CI]b 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Confidence interval for the change from baseline. 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
17  
 
Table 14.2.1.1 
 
Non-Vellus Target Area Hair Count: Mean and Mean Change from Baseline (hairs/cm2) 
ITT Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
  
 
  
 
V10–Week 26 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
SE 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
[95% CI]b 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
 
 
 
 
 
 
 
 
V16–Week 52 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
xx.x (xx.xx)  
xx.x (xx.xx)  
 
SE 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
x.xx 
x.xx 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
[95% CI]b 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
[xx.x, xx.x] 
 
 
 
 
 
 
 
 
 
 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Confidence interval for the change from baseline. 
 
Repeat Format for 
Table 14.2.1.2 
 Vellus Target Area Hair Count: Mean and Mean Change from Baseline (hairs/cm2) ITT Population 
Table 14.2.1.3 
 Total (Non-Vellus + Vellus) Target Area Hair Count: Mean and Mean Change from Baseline (hairs/cm2) ITT Population 
Table 14.2.1.4 
 Non-Vellus Target Area Hair Width: Mean and Mean Change from Baseline (cm) ITT Population 
Table 14.2.1.5 
 Vellus Target Area Hair Width: Mean and Mean Change from Baseline (cm) ITT Population 
Table 14.2.1.6 
 Total (Non-Vellus + Vellus) Target Area Hair Width: Mean and Mean Change from Baseline (cm) ITT Population 
Table 14.2.1.7 
 Average Non-Vellus Hair Width: Mean and Mean Change from Baseline (cm) ITT Population 
Footnote for 14.2.1.7:  Note: Average non-vellus hair width is calculated for each patient as the non-vellus target area count divided by the non-vellus 
target area hair width 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
18  
Table 14.2.2.1 
 
Investigator Global Assessment 
ITT Population 
Visit  
Statistic 
Female 
 
Male 
 
Total 
 
 
 
  
 
 
V5 – Week 8 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
V7– Week 16 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
V10–Week 26 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
V13–Week 39 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
V16 – Week 52 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
FU/Early Termination 
n 
xx 
 
xx 
 
xx 
 
Mean (SD) 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
xx.x (xx.xx)  
 
Median 
xxx.x 
 
xxx.x 
 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
 
(xxx ,xxx) 
 
(xxx ,xxx) 
Repeat Format for 
Table 14.2.2.2 
 Subject Self-Assessment ITT Population 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
19  
Table 14.2.3.1 
 
Investigator Global Assessment: Patients with Slightly Increased Hair Growth or Better (Grade +1 to +3)  
ITT Population 
 
 
Visit 
 
Statistic 
 
Female 
 
Male 
 
Total 
 
 
 
 
 
V5 – Week 8 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
V7– Week 16 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
V10–Week 26 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
V13–Week 39 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
V16 – Week 52 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
FU/Early Termination 
Na     n   (%) 
xx   xx (xx.x) 
xx   xx (xx.x) 
xx   xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a The number of patents with an assessement at the visit. Used as denominator for percentages. 
 
Repeat Format for 
Table 14.2.3.2 
 Subject Self-Assessment: Patients with Slightly Increased Hair Growth or Better (Grade +1 to +3) ITT Population 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
20  
Table 14.2.4 
 
Improvement from Baseline in Norwood-Hamilton Scale in Males 
ITT Population 
 
Visit 
     Shift Detaila 
 
Nb 
 
Improved by ≥ 1 Level 
n (%) 
 
Improved by ≥ 2 Levels  
n (%) 
 
  
 
 
 
  
 
 
 
 
  
V7 – Week 16 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
V10 – Week 26 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
V16 – Week 52 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
FU/Early Terminationc 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
Early Term, 1st 26 Weeks 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
 
a The Baseline Score →Timepoint Score combination that met the ≥ 1 level improvement criteria. 
b The number of patients with a non-missing score baseline and at the visit indicated. Used as denominator for percentages. 
c Includes early terminations or follow-ups collected during the first 26 Weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   Additional rows provided to further sub-divide based on the period (1st 26 Weeks vs 6 Month Extension) 
and termination status (early termination vs follow-up). 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
21  
Table 14.2.4 
 
Improvement from Baseline in Norwood-Hamilton Scale in Males 
ITT Population 
 
Visit 
     Shift Detaila 
 
Nb 
 
Improved by ≥ 1 Level 
n (%) 
 
Improved by ≥ 2 Levels  
n (%) 
 
  
 
 
 
  
 
 
 
 
  
Follow-Up 1st 26 Weeks 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
Early Term, 6 Month Extension 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
Follow-Up, 6 Month Extension 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – IIIa 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
IV – III vertex 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
 
a The Baseline Score →Timepoint Score combination that met the ≥ 1 level improvement criteria. 
b The number of patients with a non-missing score baseline and at the visit indicated. Used as denominator for percentages. 
c Includes early terminations or follow-ups collected during the first 26 Weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   Additional rows provided to further sub-divide based on the period (1st 26 Weeks vs 6 Month Extension) 
and termination status (early termination vs follow-up). 
 
PROGRAMMING NOTE: Add rows for each combination that shows an improvement starting from greatest improvement and highest baseline score.  


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
22  
 
 
 
Table 14.2.5 
 
Improvement from Baseline in Sinclair Scale in Females 
Safety Population 
 
Visit 
     Shift Detaila 
 
Nb 
 
Improved by ≥ 1 Level 
n (%) 
 
Improved by ≥ 2 Levels  
n (%) 
 
  
 
 
 
  
 
 
 
 
  
V7 – Week 16 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 3 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
V10 – Week 26 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 3 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
V16 – Week 52 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 3 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
FU/Early Terminationc 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 3 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
Early Term, 1st 26 Weeks 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 3 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 etc 
 
 
 
 
 
 
  
 
 
a The Baseline Score →Timepoint Score combination that met the ≥ 1 level improvement criteria. 
b The number of patients with a non-missing score baseline and at the visit indicated. Used as denominator for percentages. 
c Includes early terminations or follow-ups collected during the first 26 Weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   Additional rows provided to further sub-divide based on the period (1st 26 Weeks vs 6 Month Extension)  
and termination status (early termination vs follow-up). 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
23  
Table 14.2.5 
 
Improvement from Baseline in Sinclair Scale in Females 
Safety Population 
 
Visit 
     Shift Detaila 
 
Nb 
 
Improved by ≥ 1 Level 
n (%) 
 
Improved by ≥ 2 Levels  
n (%) 
 
  
 
 
 
  
 
 
 
 
  
Follow-Up 1st 26 Weeks 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
Early Term, 6 Month Extension 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
Follow-Up, 6 Month Extension 
xx 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 3 
 
 
xx (xx.x) 
 
xx (xx.x) 
 
  
 
 
4 - 2 
 
 
xx (xx.x) 
 
 
 
  
 
 
etc 
 
 
 
 
 
 
  
 
 
a The Baseline Score →Timepoint Score combination that met the ≥ 1 level improvement criteria. 
b The number of patients with a non-missing score baseline and at the visit indicated. Used as denominator for percentages. 
c Includes early terminations or follow-ups collected during the first 26 Weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   Additional rows provided to further sub-divide based on the period (1st 26 Weeks vs 6 Month 
Extension) and termination status (early termination vs follow-up). 
 
PROGRAMMING NOTE: Add rows for each combination that shows an improvement starting from greatest improvement and highest baseline score.


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
24  
 
Table 14.2.6 
 
Correlation of Selected Effiacy Measures  
ITT Population 
 
 
Visit 
 
 
Parameter → 
↓ 
 
Change in 
Nonvellus Target 
Area Hair Count 
 
Change in 
Nonvellus Target 
Area Hair Width 
Change in 
Nonvellus Target 
Average Area 
Hair Width 
 
Investigator 
Global 
Assessment 
 
 
Subject Self 
Assessment 
 
 
 
 
 
 
 
V5 – Week 8 
Change in Nonvellus Target 
Area Hair Count 
Corr=1 
Corr=0.xx 
Corr=0.xx 
Corr=0.xx 
Corr=0.xx 
 
 
P-Value=0.xxxx 
P-Value=0.xxxx 
P-Value=0.xxxx P-Value=0.xxxx 
 
 
 
 
 
 
 
 
Change in Nonvellus Target 
Area Hair Width 
 
Corr=1 
Corr=0.xx 
Corr=0.xx 
Corr=0.xx 
 
 
 
P-Value=0.xxxx 
P-Value=0.xxxx P-Value=0.xxxx 
 
 
 
 
 
 
 
 
Change in Nonvellus Target 
Area Average  Hair Width 
 
 
Corr=1 
Corr=0.xx 
Corr=0.xx 
 
 
 
 
P-Value=0.xxxx P-Value=0.xxxx 
 
 
 
 
 
 
 
 
Investigator Global 
Assessment 
 
 
 
Corr=1 
Corr=0.xx 
 
 
 
 
 
P-Value=0.xxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
V7–Week 16 
 
 
 
 
 
 
V10–Week 26 
 
 
 
 
 
 
V16 – Week 52 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Note: Correaltions are Pearson Correaltions 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
25  
 
Table 14.2.7 
 
Correlation of Nonvellus Hair Count and Width Measures with Time Since Diagnosis of Androgenetic Alopecia  
ITT Population 
 
 
 
Visit 
 
Parameter → 
↓ 
 
 
Time Since Diagnosis of 
Androgenetic Alopecia 
 
 
 
 
 
 
V5 – Week 8 
Change in Nonvellus Target Area 
Hair Count 
 
Corr=0.xx 
 
 
 
P-Value=0.xxxx 
 
 
 
 
 
 
 
Change in Nonvellus Target Area 
Hair Width 
 
Corr=0.xx 
 
 
 
P-Value=0.xxxx 
 
 
 
 
 
 
 
Change in Nonvellus Target Area 
Average  Hair Width 
 
Corr=0.xx 
 
 
 
P-Value=0.xxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
V7–Week 16 
 
 
 
 
V10–Week 26 
 
 
 
 
V16 – Week 52 
 
 
 
 
 
 
 
 
 
 
Note: Correaltions are Pearson Correaltions 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
26  
Table 14.3.1 
 
Summary of Treatment-Emergent Adverse Events  
Safety Population 
 
 
 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
Total 
(N=xx)  
   n   (%) 
 
 
 
 
Any Adverse Event 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Any Treatment-Related Adverse Event 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Any Serious Adverse Event 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
Any Adverse Event Resulting in Study Discontinuation 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
n = number of patients with treatment emergent adverse event(s). 
NOTE: Patients with multiple occurrences of a preferred term are counted only once for that term.  


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
27  
 
 
Table 14.3.1.1 
 
Treatment-Emergent Adverse Events by System Organ Class and Preferred Term  
Safety Population 
 
 
MedDRA SOC 
    Preferred Term 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
Total 
(N=xx)  
   n   (%) 
 
 
 
 
Any Adverse Event 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
SOC Term 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
SOC Term 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
n = number of patients with treatment emergent adverse event(s). 
NOTE: Patients with multiple occurrences of a preferred term are counted only once for that term.  
 
PROGRAMMING NOTE:  to be carried out for:  
Table 14.3.1.2 
 Treatment-Emergent Study Treatment-Related Adverse Events by System Organ Class and Preferred Term Safety Population 
Table 14.3.1.3 
 Treatment-Emergent Adverse Events Resulting in Discontinuation of Study Treatment by System Organ Class and Preferred Term 
Safety Population 
Table 14.3.1.4 
 Treatment-Emergent Serious Adverse Events by System Organ Class and Preferred Term Safety Population 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
28  
 
Table 14.3.1.5 
 
Treatment-Emergent Adverse Events by Descending Order of Frequency for ATI-50002  
Safety Population 
 
 
 
Preferred Term 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
Total 
(N=xx)  
   n   (%) 
 
 
 
 
 
AE Preferred Term 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
n = number of patients with treatment emergent adverse event(s). 
NOTE: Events presented in descending order of frequency (i.e. percentage of patients with the event) in the Total AT-50002 treatment group.  Patients 
with multiple occurrences of a preferred term are counted only once for that term.  
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
29  
Table 14.3.1.6 
 
Treatment-Emergent Adverse Events by Maximum Severity 
Safety Population 
 
 
Female,  n  (%) a 
 
MedDRA SOC 
 
Preferred Term 
 
Severity 
Mild 
 
Moderate 
 
Severe 
 
Missinga 
 
Total 
 
 
 
 
 
 
 
 
 
 
Total Adverse Events 
xx (xx.x) 
 
xx (xx.x) 
 
xx (xx.x) 
 
xx (xx.x) 
 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
SOC Term 1 
 
Preferred Term 1 
 
Preferred Term 2 
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) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
a Missing is referring to an event that has missing severity and no other event with severity provided for the same preferred term. 
 
NOTE: If a patient had more than one adverse event within an SOC, the patient was counted in the category of greatest known severity.  
              If a patient had multiple occurrences of the same adverse event, the patient was counted in the category of greatest known severity. 
 
Table 14.3.1.7  Treatment-Emergent Study Medication Related Adverse Events by Maximum Severity Safety Population 
 
PROGAMMING NOTE: Separate pages for Female. Male and Total. 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
30  
Table 14.3.1.8 
 
Local Skin Reactions – Highest Grade Any Time Post-Baseline  
Safety Population 
 
 
 
Highest Severity, n  (%) 
 
 
Female 
 
Male 
Assessor 
Sign 
Mild 
Moderate 
Severe 
Total 
 
Mild 
Moderate 
Severe 
Total 
 
 
 
 
 
 
 
 
 
 
 
Any 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
Investigator 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Erythema 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Scaling 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Dryness 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
Subject 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Stinging/burning 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
Itching 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
a Percentages are based on the total number of patients safety population for the group. 
NOTE: Patients are counted only once foreach sign at the highest severity grade.  
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
31  
 
Table 14.3.1.8 
 
Local Skin Reactions – Highest Grade Any Time Post-Baseline   
Safety Population 
 
 
 
                            Highest Severity, n  (%) 
 
 
Total 
 
 
Assessor 
Sign 
Mild 
Moderate 
Severe 
Total 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Any 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Investigator 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Erythema 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Scaling 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Dryness 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
Subject 
Any 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Stinging/burning 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Itching 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Percentages are based on the total number of patients safety population for the group. 
NOTE: Patients are counted only once foreach sign at the highest severity grade.  


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
32  
 
Table 14.3.1.9 
 
Treatment-Emergent Adverse Events by System Organ Class and Preferred Term in Patients Who Discontinued in First 26 Weeks 
Safety Population 
 
 
MedDRA SOC 
    Preferred Term 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
Total 
(N=xx)  
   n   (%) 
 
 
 
 
Any Adverse Event 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
SOC Term 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term 1 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       AE Preferred Term 3 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
SOC Term 2 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
AE Preferred Term _ 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
n = number of patients with treatment emergent adverse event(s). 
NOTE: Patients with multiple occurrences of a preferred term are counted only once for that term.  
 
PROGRAMMING NOTE:  to be carried out for:  
Table 14.3.1.10  Treatment-Emergent Adverse Events by System Organ Class and Preferred Term in Patients Who Completed 26 Weeks and Did not 
Enter Six Month Extension Safety Population 
Table 14.3.1.11  Treatment-Emergent Adverse Events by System Organ Class and Preferred Term in Patients Who Completed 26 Weeks and Entered 
Six Month Extension Safety Population 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
33  
Table 14.3.2.1 
 
Vital Signs: Systolic Blood Pressure (mmHg) 
Safety Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
 
 
 
 
 
 
Baselinea 
n 
xx 
 
 
xx 
 
 
xx 
 
 
Mean (SD)  
xx.x (xx.xx) 
 
 
xx.x (xx.xx) 
 
 
xx.x (xx.xx) 
 
 
Median 
xxx.x 
 
 
xxx.x 
 
 
xxx.x 
 
 
(Min, Max) 
(xxx ,xxx) 
 
 
(xxx ,xxx) 
 
 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
 
V3 – Week 3 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V4 – Week 4 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V5 – Week 8 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V6 – Week 12 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Includes early terminations or follow-ups collected during the first 26 weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.    


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
34  
Table 14.3.2.1 
 
Vital Signs:  Systolic Blood Pressure (mmHg) 
Safety Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
  
 
 
 
 
V7 – Week 16 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V8 – Week 20 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V9 – Week 24 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V10 – Week 26 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V10 – Week 26 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Includes early terminations or follow-ups collected during the first 26 weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
35  
Table 14.3.2.1 
 
Vital Signs:  Systolic Blood Pressure (mmHg) 
Safety Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
  
 
 
 
 
V10 – Week 26 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V11 – Week 30 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V12 – Week 35 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V13 – Week 39 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V14 –Week 44 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Includes early terminations or follow-ups collected during the first 26 weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.   


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
36  
Table 14.3.2.1 
 
Vital Signs:  Systolic Blood Pressure (mmHg) 
Safety Population 
 
 
 
Female 
 
Male 
 
Total 
Visit  
Statistic 
Value 
Change 
 
Value 
Change 
 
Value 
Change 
 
 
 
 
  
 
 
 
 
V15 – Week 48 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
V16 – Week 52 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
FU/Early Terminationb 
n 
xx 
xx 
 
xx 
xx 
 
xx 
xx 
 
Mean (SD)  
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
xx.x (xx.xx) 
xx.x (xx.xx) 
 
Median 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
xxx.x 
xxx.x 
 
(Min, Max) 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
(xxx ,xxx) 
(xxx ,xxx) 
 
 
 
 
 
 
 
 
 
 
 
 
a Baseline is selected as the last measurement prior to the first application of study medication. 
b Includes early terminations or follow-ups collected during the first 26 weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension.  


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
37  
 
 
PROGRAMMING NOTE: to be repeated for the following: 
Table 14.3.2.2   Vital Signs:  Diastolic Blood Pressure (mmHg) Safety Population    
Table 14.3.2.3   Vital Signs:  Heart Rate (bpm) Safety Population    
Table 14.3.2.4   Vital Signs:  Respiratory Rate (bpm) Safety Population 
Table 14.3.2.5   Vital Signs:  Temperature (Degrees C) Safety Population    


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
38  
Table 14.3.3 
 
ECG Interpretation: Shift from Baseline to Worst Assessment Post Baseline  
Safety Population 
 
Group 
    Baseline Interpretation 
Worst Post-Baseline Interpretation, n (%)  
Normal 
Abnormal, NCS 
Abnormal, CS 
Missing 
Total 
 
 
 
 
 
 
Female (Na=xx) 
 
 
 
 
 
       Normal 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, NCS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, CS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Missing 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Total 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Male (Na=xx) 
 
 
 
 
 
       Normal 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, NCS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, CS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Missing 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Total 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
Total (Na=xx) 
 
 
 
 
 
       Normal 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, NCS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Abnormal, CS 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Missing 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
       Total 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
xx (xx.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
a The number of all patients in the safety population for the group.  
Note: The worst assessment post baseline includes any unscheduled assessment(s) as well the post-treatment assessment. 
 
 
          
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
39  
 
PROGRAMMING NOTE: The following tables use for the format of the vital sign mean and mean change tables. Timepoints Baseline, V5 – Week 8, 
V7 – Week 16, V9 – Week 24, V10 – Week 26, V13 – Week 39, V14 – Week 44, V16 – Week 52, FU/Early Termination. 
 
 
Table 14.3.4.1 
 Descriptive Statistics for Chemistry Parameters Safety Population 
Table 14.3.4.2 
 Descriptive Statistics for Hematology Parameters Safety Population 
Table 14.3.4.3 
 Descriptive Statistics for Hormone Parameters Safety Population 
 
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
40  
Table 14.3.5 
 
Number and Percent of Patients Meeting Criteria for Hepatobiliary Laboratory Abnormalities  
Safety Population 
 
 
Criteria 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
Total 
(N=xx)  
   n   (%) 
>3xULN ALT or ASTa 
x (x.x) 
x (x.x) 
x (x.x) 
         >3xULN ALT 
x (x.x) 
x (x.x) 
x (x.x) 
         >3xULN AST 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
>5xULN ALT or ASTa 
x (x.x) 
x (x.x) 
x (x.x) 
         >5xULN ALT 
x (x.x) 
x (x.x) 
x (x.x) 
         >5xULN AST 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
>10xULN ALT or ASTa 
x (x.x) 
x (x.x) 
x (x.x) 
         >10xULN ALT 
x (x.x) 
x (x.x) 
x (x.x) 
         >10xULN AST 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
>20xULN ALT or ASTa 
x (x.x) 
x (x.x) 
x (x.x) 
         >20xULN ALT 
x (x.x) 
x (x.x) 
x (x.x) 
         >20xULN AST 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Elevation of Bilirubin 
 
 
 
         >1.5 xULN 
x (x.x) 
x (x.x) 
x (x.x) 
         >2xULN 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
>1.5xULN Alk Phos 
x (x.x) 
x (x.x) 
x (x.x) 
 
 
 
 
Elevation of ATb and Bilirubin 
 
 
 
         >3xULN AT and >1.5xULN Bilirubin 
x (x.x) 
x (x.x) 
x (x.x) 
         >3xULN AT and >2xULN Bilirubin 
x (x.x) 
x (x.x) 
x (x.x) 
         >3xULN AT and >=2xULN Bilirubin and Alkaline Phosphatase <2xULN or missing 
x (x.x) 
x (x.x) 
x (x.x) 
a Patients with both ALT and AST abnormalities for the same lab draw (same date and time) are counted as one event. 
b AT = aminotransferase. Either AST or ALT (or both) elevated. 
PROGRAMMING NOTE: It is intentional that the last criterion has ‘=’ added to the bilirubin threshold – to match exactly FDA Guidance doc 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
41  
 
Table 14.3.6 
 
Number and Percent of Patients Meeting Laboratory Criteria for Potential Renal Impairment 
Safety Population 
 
 
 
Criteria 
Female 
(N=xx)  
   n   (%) 
Male 
(N=xx)  
   n   (%) 
 
Total 
(N=xx)  
   n   (%) 
 
 
 
 
 
 
 
Change from Baseline ≥ 0.5 mg/dL 
x (x.x) 
x (x.x) 
 
x (x.x) 
 
>1.5xULN Creatinine 
x (x.x) 
x (x.x) 
 
x (x.x) 
 
EITHER Change from Baseline >=0.5 mg/dL or >1.5xULN Creatinine 
x (x.x) 
x (x.x) 
 
x (x.x) 
 
BOTH  Change from Baseline >=0.5 mg/dL and >1.5xULN Creatinine 
x (x.x) 
x (x.x) 
 
x (x.x) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
  
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
42  
Figure 14.1 
 
Association of Selected Efficacy Measures with Fit Linear Regression Line 
ITT Population: Visit = V5 Week 8 
 
   
               
               
 
               
            
 
 


Aclaris Therapeutics Inc. 
23 Apr 2019 
Protocol: ATI-50002-AGA-201 
Statistical Analysis Plan 
 
Data Source: XX 
 
Program: XYZ.SAS 
43  
 
 
 
Reperat for: 
 
Figure 14.2  
Association of Selected Efficacy Measures with Fit Linear Regression Line, ITT Population: Visit=V7 Week 16 
Figure 14.3  
Association of Selected Efficacy Measures with Fit Linear Regression Line, ITT Population: Visit=V10 Week 26 
Figure 14.4  
Association of Selected Efficacy Measures with Fit Linear Regression Line, ITT Population: Visit=V16 Week 52 
 
For the following plot change in averge hair wdth (vertical) vs Investigaor Global Assessment [4 figures – All timepoints on page]. Indicate timepoint at top of 
each of the visits. Separate color for males and females and linear regression line as per figure 14.1.Add Foonote: Note Lines represent fit linerar regression line. 
 
Figure 14.5  
Association of Change in Nonvellus Target Area Average Hair Width with Investigator Global Assessment, ITT Population 
 
For the following plot change in averge hair wdth (vertical) vs Subject Self Assessment [4 figures – All timepoints on page] Indicate timepoint at top of each of 
the visits. Separate color for males and females and linear regression line as per figure 14.1.Add Foonote: Note Lines represent fit linerar regression line. 
 
 
Figure 14.6  
Association of Change in Nonvellus Target Area Average Hair Width with Subject Self Assessment, ITT Population 
 
For the following plot change nonvellus target area hair count (vertical), change nonvellus target area hair width (vertical) and change nonvellus target area 
average hair width (vertical), vs time since diagnosis of androgenetic alopecia [4 figures on page – all timepoints on page] Indicate timepoint at top of each of the 
visits. Separate color for males and females and linear regression line as per figure 14.1.Add Foonote: Note Lines represent fit linerar regression line. 
 
Figure 14.7  
Association of Change in Nonvellus Target Area Hair Count with Time Since Diagnosis of Androgenetic Alopecia, ITT Population 
Figure 14.8  
Association of Change in Nonvellus Target Area Hair Width with Time Since Diagnosis of Androgenetic Alopecia, ITT Population 
Figure 14.9  
Association of Change in Nonvellus Target AreaAverage Hair Width with Time Since Diagnosis of Androgenetic Alopecia, ITT 
Population 
 
 
 
 
 
 


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Data Source: XX 
 
Program: XYZ.SAS 
44  
 Appendix 16.2.1.1 
 
Disposition 
 
Group: Female, Male 
 
 
 
Patient 
Date of 
Last 
Applicationa 
Date of 
DC/ 
Completion 
 
Days from Last 
Applicationb 
DC/  
Completion
Study Dayc 
 
 
Reason DC from Study 
Period of Study 
Discontinuation/ 
Completion 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
xx-xxx 
mmdddyy 
mmdddyy 
xx 
xx 
Adverse Event/SAE: list AE, link by AE number 
First 26 Weeks 
 
 
 
 
 
Protocol Violation 
6 Month Extension 
 
 
 
 
 
Lost to Follow-Up 
 
 
 
 
 
 
Withdrew Consent 
 
 
 
 
 
 
Pregnancy 
 
 
 
 
 
 
Other: specify 
 
 
 
 
 
 
Completed  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Date of last dose of application of study medication. 
b DC date/Completion Date– date last application +1. 
c DC date/ Completion Date – date of first application of study medication +1. 
 
NOTE: DC=Discontinued. 
 
  
 


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Program: XYZ.SAS 
45  
Appendix 16.2.1.2 
 
Demographics  
                                 
Group: Female, Male 
 
 
 
Patient 
Agea 
(Years) 
 
Gender 
 
Race 
 
Ethnicity 
Fitzpatrick 
Skin Type 
Heightb 
(inches) 
Weightb 
(lbs) 
 
 
 
 
 
 
 
 
xxx 
 
 
 
 
 
 
 
 
 
 
 
xx-xxx 
xx 
Male 
Caucasian/White 
Hispanic/Latino 
1,2,3,4,5,6 
xx.x 
xxx.x 
 
 
 
Female 
Black 
Not Hispanic/Latino 
 
 
 
 
 
 
 
Asian/Oriental 
Not Willing to Provide 
 
 
 
 
 
 
 
American Indian or Alaska Native 
 
 
 
 
 
 
 
 
Native Hawaiian or Pacific Islander  
 
 
 
 
 
 
 
Other - specify 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a At Date of signed informed consent. 
b FromV-2 Day 1 vitals page. 
  


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46  
Appendix 16.2.1.3 
 
History of Androgenetic Alopecia: Diagnosis and Prior Treatment and Therapies 
                                 
Group: Female, Male 
 
 
 
Patient 
 
 
Onset Date of Diagnosis 
Years Since 
Diagnosisa 
   Prior Treatment/ 
Therapy Type  
Treatment or Therapy 
Coded/Verbatim 
Start Date/ 
Stop Date 
Start Dayb/ 
Stop Dayb 
 
  
 
   
 
 
 
 
  
 
   
 
 
 
xx-xxx 
 
ddmmmyyyy  
xx.x 
  Pharmaceutical 
Minoxidil/ 
Rogaine/ 
01Jan2018 
03Jan2018 
55 
57 
 
 
 
 
  Pharmaceutical 
Finasteride/ 
Propecia 
01Jan2018 
03Jan2018 
55 
57 
 
 
 
 
  Device 
Coded term/ 
verbatim 
 
 
 
 
 
 
  Other 
Coded term/ 
verbatim 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
  
 
   
 
 
 
 
a (Date of Diagnosis  - Date of first application of study medication) / 365. 
b Start date – date of first application of study medication 
NOTE: Date of Diagnosis from medical history page of CRF 
 
PROGRAMMING NOTE: Will need to review prior treatment and therapies to assign “Type” i.e. “Pharmaceutical”, “Device” and “other”  
 


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47  
 Appendix 16.2.1.4 
 
Medical History 
 
Group: Female, Male 
 
 
Patient 
 
Body System 
 
Diagnosis 
Onset 
Date (Study Daya) 
Resolution 
Date (Study Daya) 
Ongoing at Time of 
Randomization? 
 
 
 
 
 
 
 
 
 
 
 
 
xx-xxx 
Skin 
Free Text 
ddmmmyyyy (xx) 
ddmmmyyyy (xx) 
Yes 
 
Central nervous system 
 
 
 
 
 
Ophthalmologic 
 
 
 
 
 
Ear, nose, throat 
 
 
 
 
 
Respiratory 
 
 
 
 
 
Gastrointestinal 
 
 
 
 
 
Hepatic 
 
 
 
 
 
Renal 
 
 
 
 
 
Cardiovascular 
 
 
 
 
 
Hematologic 
 
 
 
 
 
Genitourinary 
 
 
 
 
 
Endocrine/Lymphatic 
 
 
 
 
 
Musculoskeletal 
 
 
 
 
 
Systemic Infection 
 
 
 
 
 
Drug Allergy 
 
 
 
 
 
Substance Abuse 
 
 
 
 
 
Psychiatric 
 
 
 
 
 
Other 
 
 
 
 
 
a Study Day = Start/stop date – date of first application of study medication. 
 
PROGRAMMING NOTE:  List dates as entered - i.e., display partial dates.  Imputed dates in database are used to calculate study day.  
                                                If patient has no entries, put “None” under Body System.  


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48  
  
Appendix 16.2.1.5 
 
Prior and Concomitant Therapies 
 
Group: Female, Male 
 
 
 
Patient 
WHO ATC Level III/ 
WHO Preferred Term/ 
Verbatim Term 
 
Dose (unit)  
[Route] Frequency 
Study Period(s) 
in Which 
Receiveda 
 
Start Date/ 
Stop Date 
 
Start Dayb/ 
Stop Dayb 
 
 
Indication 
Associated 
with AE 
(AE#) 
 
 
 
 
 
 
 
 
 
 
xx (mg) [oral] QD 
 
P 
O 
F 
P,O 
O,F 
P,O,F 
01Jan2018 
03Jan2018 
55 
57 
Free Text 
No 
Yes (xx) 
 
 
 
 
Note:  if 
“ongoing”, 
put 
“ongoing” 
under Stop 
Date 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a P = Prior medication;  O = Treatment Period;  F=Post-Therapy 
b Start date – date of first application of study medication (+1 if ≥ first application of study medication). 
 
NOTE:  – = data not available 


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49  
 Appendix 16.2.1.6 
 
ATI-50002 Accountability 
 
Group: Female, Male 
 
 
 
 
Patient 
 
 
Date 
Dispensed 
 
Study 
Daya 
Dispensed 
Dispensed 
Bottle 
Weight  
(gm) 
 
 
Date 
Collected 
 
 
Study Daya 
Collected 
 
Collected 
Bottle Weight  
(gm) 
 
Amount 
Usedb 
(gm) 
Volume  
 
(mL)c 
Volume (mL) Per 
Applicationc 
 
 
 
 
 
 
 
 
 
 
xx-xxx ddmmmyyyy 
xx  
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
ddmmmyyyy 
xx 
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
ddmmmyyyy 
xx 
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
ddmmmyyyy 
xx 
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
ddmmmyyyy 
xx 
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
ddmmmyyyy 
xx 
xx 
ddmmmyyyy 
xx 
xx 
xx 
xx 
 
 
TOTAL: 
xx 
xxx 
xx.x 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Dispensed Date (or Collection Date) – Date first Application +1. 
b Weight dispensed – Weight collected (returned). 
c The density of study agent is 1 g/mL which is used to convert differences in bottle weights between dispensed and returned (ie weight of study agent 
used) to mLs. 
 
     


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50  
Appendix 16.2.1.7 
 
ATI-50002 Accountability Comments 
 
Group: Female, Male 
 
 
 
Patient 
 
Date 
Dispensed 
Study Daya 
Dispensed 
 
Date 
Collected 
Study 
Daya 
Collected 
 
 
Comment 
 
 
 
 
 
 
xx-xxx 
ddmmmyyyy 
xx  
ddmmmyyyy 
xx 
Free Text 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Dispensed Date (or Collection Date) – Date first Application +1. 


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Appendix 16.2.1.8 
 
ATI-50002 Application Changes and Missed Doses 
 
Group: Female, Male 
 
 
 
Patient 
 
Application Frequency 
Started 
 
Study Daya 
Dispensed 
 
Application Frequency 
Ended 
 
Study Daya 
Collected 
 
 
Applications/Day 
 
 
 
 
 
 
xx-xxx 
ddmmmyyyy 
xx  
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
ddmmmyyyy 
xx 
ddmmmyyyy 
xx 
xx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Dispensed Date (or Collection Date) – Date first Application +1. 


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52  
Appendix 16.2.1.9 
 
Eligibility Criteria 
 
Group: Female, Male 
 
 
Patient 
 
Inclusion Criteria Not Met 
 
Exclusion Criteria Not Met 
 
 
 
 
 
 
1,2 ….13 
None 
 
 
None 
1,2 ..16 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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Appendix 16.2.1.10 
 
Protocol Violations 
 
Group: Female, Male 
 
 
Patient 
 
Violation 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


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Appendix 16.2.2.1 
 
Target Area Hair Count and Width 
        
 
Group: Female, Male 
 
Patient  
Visit 
Assessment Date 
Study Daya 
TAHC 
 (hairs/cm2) 
TAHW 
 (cm) 
   
 
 
 
 
 
 
 
   
xx-xxx  
V2 – Day 1 
ddmmmyyyy 
xx 
xxx 
xxx 
   
 
 
V5 – Week 8 
ddmmmyyyy 
xx 
xxx 
xxx 
   
 
 
V7 – Week 16 
ddmmmyyyy 
xx 
xxx 
xxx 
   
 
 
V10 – Week 26 
ddmmmyyyy 
xx 
xxx 
xxx 
   
 
 
V16 – Week 52 
ddmmmyyyy 
xx 
xxx 
xxx 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a  Assessment date – date of first application of study medication (+1 if ≥ first application of study medication). 


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55  
Appendix 16.2.2.2 
 
Investigator and Subject Global Assessments 
        
 
Group: Female, Male 
 
Patient  
 
Visit 
Assessment 
Date 
Study 
Daya 
Investigator 
Global 
Assessmentb 
Subject 
 Global 
Assessmentb 
 
  
 
 
 
 
xx-xxx  V5 – Week 8 
ddmmmyyyy 
xx 
-3 
-3 
 
 V7 – Week 16 
ddmmmyyyy 
xx 
-2 
-2 
 
 V10 – Week 26 
ddmmmyyyy 
xx 
-1 
-1 
 
 V13 – Week 39 
ddmmmyyyy 
xx 
0 
0 
 
 V16 – Week 52 
ddmmmyyyy 
xx 
1 
1 
 
 Early Term 1st 26 Weeks 
ddmmmyyyy 
xx 
2 
2 
 
 Follow-Up 1st 26 Weeks 
 
 
3 
3 
 
 Early Term 6 Month Extension 
 
 
 
 
 
 Follow-Up 6 Month Extension 
 
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
a Assessment date – date of first application of study medication (+1 if ≥ first application of study medication). 
b  -3: Greatly decreased hair growth; -2: Moderately decreased hair growth; -1: Slightly decreased hair growth; 0: No Change; +1: Slightly increased 
hair growth;  +2: Moderately increased hair growth; +3: Greatly increased hair growth. 
 
Programming note:  Differentiation between Early term and Follow-up is based on the availability of V10 (1st 26 Weeks) or V16 (patients entering 
extension) data for ANY data (not just global assessments).  If a patient does not enter the extension and had no data collected for V10 and had 
discontinued the trial the follow-up is considered “Early Term 1st 26 Weeks”.  If they had V10 and did not enter the extension then it’s “Follow-Up 6 
Month Extension”.  Similarly for those entering extension, those without a V16 who discontinued the study are assigned “Early Term 6 Month 
Extension” and those with V16 are assigned to “Follow-Up 6 Month Extension”. 


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Program: XYZ.SAS 
56  
Appendix 16.2.2.3 
 
Norwood-Hamilton Scale in Males 
        
 
Group: Male 
 
Patient  
 
Visit 
Assessment 
Date 
Study 
Daya 
N-H Score 
Improvement 
from Baseline 
(Yes/No)c 
 
  
 
 
 
 
xx-xxx  V1 - Screening 
ddmmmyyyy 
xx 
I 
Yes/No 
 
 V2 – Day 1 
ddmmmyyyy 
xx 
IIb 
 
 
 V7 – Week 16 
ddmmmyyyy 
xx 
IIa 
 
 
 V10 – Week 26 
ddmmmyyyy 
xx 
III 
 
 
 V16 – Week 52 
ddmmmyyyy 
xx 
IIIa 
 
 
 Early Term 1st 26 Weeks 
ddmmmyyyy 
xx 
III Vertex 
 
 
 Follow-Up 1st 26 Weeks 
 
 
IV 
 
 
 Early Term 6 Month Extension 
 
 
IVa 
 
 
 Follow-Up 6 Month Extension 
 
 
V 
 
 
  
 
Va 
 
 
  
 
VI 
 
 
  
 
VII 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
a Assessment date – date of first application of study medication (+1 if ≥ first application of study medication). 
b  Used as baseline for analysis. 
c Yes – score improved by ≥1 level including shifts in “sub-level” (e.g. IIIa to III is considered a 1 level improvement).  
 
 
Programming note:  Differentiation between Early term and Follow-up is based on the availability of V10 (1st 26 Weeks) or V16 (patients entering 
extension) data for ANY data (not just Norwood-Hamilton).  If a patient does not enter the extension and had no data collected for V10 and had 
discontinued the trial the follow-up is considered “Early Term 1st 26 Weeks”.  If they had V10 and did not enter the extension then it’s “Follow-Up 1st 
26 Weeks”.  Similarly for those entering extension, those without a V16 who discontinued the study are assigned “Early Term 6 Month Extension” and 
those with V16 are assigned to “Follow-Up 6 Month Extension”. 
 


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Appendix 16.2.2.4 
 
Sinclair Scale in Females 
        
 
Group: Female 
 
Patient  
 
Visit 
Assessment 
Date 
Study 
Daya 
Sinclair Score 
Improvement 
from Baseline 
(Yes/No) 
 
  
 
 
 
 
xx-xxx  V1 - Screening 
ddmmmyyyy 
xx 
1 
Yes/No 
 
 V2 – Day 1 
ddmmmyyyy 
xx 
2b 
 
 
 V7 – Week 16 
ddmmmyyyy 
xx 
3 
 
 
 V10 – Week 26 
ddmmmyyyy 
xx 
4 
 
 
 V16 – Week 52 
ddmmmyyyy 
xx 
5 
 
 
 Early Term 1st 26 Weeks 
ddmmmyyyy 
xx 
 
 
 
 Follow-Up 1st 26 Weeks 
 
 
 
 
 
 Early Term 6 Month Extension 
 
 
 
 
 
 Follow-Up 6 Month Extension 
 
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
  
 
 
 
 
a Assessment date – date of first application of study medication (+1 if ≥ first application of study medication). 
b  Used as baseline for analysis. 
  
 
 
Programming note:  Differentiation between Early term and Follow-up is based on the availability of V10 (1st 26 Weeks) or V16 (patients entering 
extension) data for ANY data (not just Sinclair score).  If a patient does not enter the extension and had no data collected for V10 and had discontinued 
the trial the follow-up is considered “Early Term 1st 26 Weeks”.  If they had V10 and did not enter the extension then it’s “Follow-Up 1st 26 Weeks”.  
Similarly for those entering extension, those without a V16 who discontinued the study are assigned “Early Term 6 Month Extension” and those with 
V16 are assigned to “Follow-Up 6 Month Extension”. 


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58  
 
Appendix 16.2.3.1  
 
All Adverse Events 
 
Group: Female, Male 
 
 
Patient 
MedDRA SOC/ 
MedDRA Preferred Term/ 
CRF Verbatim Term (AE No.) 
Onset Study Daya 
Resolution Study Dayb  
Duration (Days)c 
Onset Date/ 
Stop Date 
Cutaneous AE? 
 
Relationship/ 
Action Taken/ 
Therapy Required 
Serious/ 
Severity/ 
Outcome 
 
501-01 
 
 
 
 
 
 
 
 
 
Cardiovascular System / 
   Bradycardia / 
       Slow Heart Rate (xx)d 
 
 
 
 
 
 
 
62  
64 
3 
 
 
 
 
01Sep12  
 
03Sep12 
Ongoing 
 
Yes (Scalp), 
Yes (not Scalp), 
No 
 
 
 
 
 
Related 
Not Related 
 
Dose not changed 
Dose Reduced 
Dose Interrupted 
Drug Withdrawn 
Unknown 
 
Yes/No 
 
Yes/No    
 
Mild/Moderate/Severe 
 
Recoverd/Resolved 
Not Recovered/Resolved 
Recovered/Resolved w/ Seq. 
Fatal 
Unknown 
 
a AE onset date – date of first application of study medication (+1 if ≥ first application). 
b AE resolution date – date of first application of study medication (+1 if ≥ first application). 
c Resolution Date – Onset Date +1 
d Not treatment emergent = Onset either prior to the first application or > 30 days after the last application of study medication.  
 
PROGRAMMING NOTES:  Sort by group, patient, onset date, SOC and PREF term.  Display partial dates as entered in raw data. Use derived dates 
for study day and duration 
Appendix 16.2.3.2 
Serious Adverse Events 
Appendix 16.2.3.3 
Adverse Events Resulting in Discontinuation of Study Treatment 
Appendix 16.2.3.4 
Adverse Events Resulting in Outcome of Death 


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Appendix 16.2.3.5 
 
Local Skin Reactions 
 Group: Female, Male 
 
 
Patient 
 
Visit 
Assessment 
Date 
Study 
Daya 
 
Assessor 
 
Signb 
 
Grade 
 
 
 
 
 
 
 
 
V2 – Day 1 
ddmmmyyyy 
1 
Investigator 
Erythema 
1 
 
 
 
 
 
Scaling 
2 
 
 
 
 
 
Dryness 
3 
 
 
 
 
Subject 
Stinging/burning 
 
 
 
 
 
 
Itching 
 
 
 
 
 
Investigator 
NO SIGN GRADED >0 
 
 
 
 
 
Subject/Legal Guardian 
NO SIGN GRADED >0 
 
 
V3 – Week 3 
ddmmmyyyy 
21 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Assessment Date – Date first Application +1. 
b Only signs with grade >   0 are displayed.  Within assessor, if all signs were grade 0 (or not done) appear as “NO SIGN GRADED > 0”.   
 


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60  
Appendix 16.2.3.6 
 
Physical Exam 
 
 
Group: Female, Male 
 
 
Patient 
Assessment 
Date 
 
Visit 
Study 
Daya 
Body 
System 
 
Findingb 
 
Description of Finding 
 
 
 
 
 
 
 
xx-xxx 
ddmmmyyyy 
V1 – Screening 
xx 
 
Normal, no findings 
 
 
 
ET/Follow-Up  
 
 
 
 
 
 
 
 
Body System a 
Abnormal, CS 
xxxxxxxxxxxxxxx 
 
 
 
 
Body System b 
Abnormal, NCS 
xxxxxxxxxxxxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Assessment Date – Date of First Application (+1 if ≥ first application of study medication). 
b CS = Clinically Significant; NCS = Not Clinically Significant 
 
 
NOTE:  Listing includes only lists body systems that were assessed with Abnormalities.  Patients with only “normal” findings  are listed as “Normal, no 
findings” (can have some body systems “not done” so long as those that were assessed are all “normal”. Not done are not listed.) 


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Appendix 16.2.3.7 
 
Vital Signs 
 
Group: Female, Male 
 
 
 
 
Patient 
 
 
Visit 
 
 
Study Daya 
 
Heart Rate 
(bpm) 
 
Respiratory Rate  
(bpm) 
 
Temperature 
(Degrees C) 
Blood Pressure  
Systolic / Diastolic  
(mmHg) 
     
 
 
 
 
 
 
  
     
xx-xxx 
V1 – Screening 
xx 
xxx 
xx 
xx 
xxx / xxx 
     
 
V2 – Day 1 
 
 
 
 
 
     
 
V3 – Week 3 
 
 
 
 
 
     
 
V4 – Week 4 
 
 
 
 
 
     
 
V5 – Week 8 
 
 
 
 
 
     
 
V6 – Week 12 
 
 
 
 
 
     
 
V7 – Week 16 
 
 
 
 
 
     
 
V8 – Week 20 
 
 
 
 
 
     
 
V9 – Week 24 
 
 
 
 
 
     
 
V10 – Week 26 
 
 
 
 
 
     
 
ET/Follow-Up 
 
 
 
 
 
     
 
V11 – Week 30 
 
 
 
 
 
     
 
V12 – Week 35 
 
 
 
 
 
     
 
V13 – Week 39 
 
 
 
 
 
     
 
V14 – Week 44 
 
 
 
 
 
     
 
V15 – Week 48 
 
 
 
 
 
     
 
V15 – Week 52 
 
 
 
 
 
     
 
Follow-Up/ Early Terminationb 
 
 
 
 
 
     
 
a Assessment Date – Date of First Application (+1 if ≥ first application of study medication). 
b Includes early terminations or follow-ups collected during the first 26 Weeks or during the additional six months for patients opting to continue 
treatment in the optional six month extension. 
 
 
 


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Appendix 16.2.3.8 
 
Electrocardiogram – Evaluator Interpretation 
 
 
Group: Female, Male 
 
Patient 
Visit 
Study Daya 
Interpretationb 
Specify – if abnormal 
       
 
 
 
 
 
       
xx-xxx 
  
xx 
Normal 
 
       
 
 
xx 
Abnormal, CS 
Sinus Bradycardia 
       
 
 
xx 
Abnormal, NCS 
Inverted T Waves; T Wave Abnormality: Biphasic T Waves        
 
 
xx 
Abnormal, NCS 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
 
 
 
 
       
 
a Assessment Date – Date of First Application (+1 if ≥ first application of study medication). 
b CS = Clinically Significant; NCS = Not Clinically Significant 
 
Programming Note: Suggestion is to list all abnormalities at a given collection be listed according to “interpretation” (ie CS and/or NCS). So if there are multiple 
reasons, as in the hypothetical example above for NCS, separate them by semi-colons. 
 
 
 


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 Appendix 16.2.3.9 
 
Clinical Laboratory Assessments: Hematology 
 
Group: Female, Male 
 
 
 
 
Patient 
 
 
Age 
(years) 
 
 
 
Gender 
 
 
 
Parameter (unit) 
 
 
 
Visit 
 
 
Study 
Daya 
 
 
 
Value 
 
 
Normal 
Range 
 
 
 
 
List Criteria Met, if 
applicable, for Hold of 
Study Medication 
 
 
 
 
 
 
 
 
 
 
 
 
xx-xxx 60 
Female 
Hemoglobin (g/dL)  
-10 
xxx 
xx.x-xx.x 
L 
 
 
< 8 g/dL or decrease > 2 g/dL 
 
 
 
 
 
  -1 b 
xxx 
  
H 
 
 
 
 
 
 
 
 
 28 
xxx 
 
 
 
 
 
 
 
 
 
 
 38  
xxx 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Hematocrit (%) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Assessment Date – Date of First Application (+1 if ≥ first application of study medication). 
b Value used for baseline. 
 
PROGRAMMING NOTE:   Sort by group, patient, parameter, and study day.  For criteria for holding study medication see protocol – Section 10.2.4.1 
 
                                                Repeat format for: 
Appendix 16.2.3.10 
 Clinical Laboratory Assessments: Biochemistry 
Appendix 16.2.3.11 
 Clinical Laboratory Assessments: Hormones 
 


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Appendix 16.2.3.12 
 
Clinical Laboratory Assessments: Urinalysis 
        
 
Group: Female, Male 
 
 
 
Patient 
 
Age 
(years) 
 
 
Gender 
 
 
Parameter 
 
 
Visit  
 
Study 
Daya 
 
 
Value 
 
 
 
 
 
 
 
 
 
 
60 
Female 
Glucose 
 
-10 
Positive 
 
 
 
 
 
 
  -1 b 
Negative   
 
 
 
 
 
 
 
Trace 
 
 
 
 
 
 
   
 
 
 
 
 
 
 
xx 
 
 
 
 
 
Protein 
 
 
 
 
 
 
 
RBC 
 
 
 
 
 
 
 
WBC 
 
 
 
 
 
 
 
Urobilinogen 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
a Assessment Date – Date of First Application (+1 if ≥ first application of study medication). 
b Value used for baseline. 
 
 
 
 
PROGRAMMING NOTES:  Display parameters in alphabetical order.   
                                                  Sort by group, patient, parameter, and study day. 


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65  
 
 
  
Appendix A 
 
MedDRA System Organ Class/Preferred Term and CRF Verbatim Terms 
 
 
MedDRA System Organ Class 
 
MedDRA Preferred Term 
 
CRF Verbatim Term 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
NOTE:  Events coded using MedDRA Version 21.1 
 

ICF_001.pdf: 
Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 1 of 7 
INFORMATION AND CONSENT FORM 
 
 
Study Title: 
An Open-Label Pilot Study of the Safety, Tolerability and Efficacy of ATI-50002 
Topical Solution Administered Twice-Daily in Adult Subjects with Non-Segmental 
Facial Vitiligo 
Study #: 
ATI-50002-VITI-201 
Sponsor: 
Aclaris Therapeutics, Inc. 
Study Doctor: 
<<investigator>> 
<<firm name>> 
<<street address>>, <<city>>, <<state>>  <<zip>> 
Telephone Number: <<000-000-0000>> 
After Office Hours: <<000-000-0000>> 
 
You are currently participating in Aclaris Therapeutics, Inc.’s ATI-50002-VITI-201 study, and the 
study doctor or study staff has asked you to read and sign this Addendum to the Consent Form.  
Everything in the main study consent form you signed before still applies to your participation in 
this study unless otherwise noted in this form.  
This form describes additional information about the study.  If you have any questions about or 
do not understand something in this form, you should ask the study doctor or study staff.  You 
should also discuss your participation with anyone you choose in order to better understand this 
study and your options. 
 
WHAT IS THE PURPOSE OF THIS ADDENDUM? 
Since the time you signed the last consent form for this study, new information related to the 
study has become available:  
The study sponsor has decided to increase the duration of the study to allow application of ATI-
50002 Topical Solution, 0.46% (study drug) to your face and if you have vitiligo on your neck to 
your neck also from 24 weeks up to 48 weeks with a 4-week after treatment follow-up visit.  You 
had previously indicated that you wanted to apply study drug for up to 24 weeks only.  If you 
would like to continue applying the study drug for the additional 24 weeks being offered, you will 
have to read and sign this consent addendum and then come into the clinic for four extra visits 
(Visits 12, 13, 14, & 15) scheduled 3 months apart which were not originally planned.  You will 
then come in for a final visit as originally planned 1 month after you last use the study drug (Visit 
16).  During these visits, the following tests may be performed by the study staff: 
 
 
 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 2 of 7 
• 
Physical Exam:  The study doctor or a member of the study staff will examine your 
overall general appearance, head, eyes, ears, nose, throat, stomach, nervous system, 
muscles and bones, lymphatics, and skin, and will listen to your heart and lungs. 
• 
Vital Signs:  Check your blood pressure by putting a band around your arm (this will 
squeeze your arm for about a minute), check your pulse, listen to you breathe in and out, 
and take your temperature. 
• 
Blood Testing:  Take some blood through a needle in your arm to do safety and 
laboratory tests.  If you continue with 4 additional visits, the maximum amount of blood 
you will have taken over the 52 weeks is slightly less than ¾ cup. 
• 
Urine Testing:  You will have your urine tested. 
• 
Pregnancy Testing:  Test your blood and/or urine to see if you are pregnant.  You will 
only have pregnancy testing if you are a woman and can have children. 
o The study doctor or study staff will tell you if the pregnancy test results are 
positive. 
o The results of the pregnancy testing must be negative in order for you to be 
enrolled in and continue in the study. 
• 
Electrocardiogram:  An electrocardiogram (ECG) measures the electrical activity of 
your heart. 
• 
Changes in Health or Medication Questions:  You will be asked about any changes in 
your health and the medications you are taking since the previous study visit. 
• 
Skin Examinations:  The study doctor or study staff will examine your face to determine 
the amount of white patches.  
• 
Questionnaires:  You will examine your face to determine and describe the appearance 
and severity of your facial vitiligo (white patches), and assess the impact of the facial 
vitiligo on your life.  At Visits 12, 13 and 14 (Day 225, 281 and 337) you will be asked to 
rate your satisfaction with the study drug results.   
• 
Photography:  The study doctor or study staff will take pictures of your face (your face 
will be included in these photographs) to document the white patches. These 
photographs may be used for research purposes related to the study and for 
presentation to government health authorities, such as the U.S. Food and Drug 
Administration (FDA), at scientific meetings, for scientific publications, for general 
corporate purposes, and may be used for marketing purposes. Your identity will not be 
revealed in these photographs.  Your identifying features, (eyes, mouth, tattoos) will be 
hidden in the photographs.  You do not have to let the study doctor or study staff 
take photographs if you don’t want to; however, if you decline to have these 
pictures taken, you will not be allowed to participate in this study. 
• 
Study Drug:  You will be given a supply of study drug and instructions for how to apply 
the study drug. 
• 
Subject Instruction Sheet:  You will be given a written instruction sheet. 
 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 3 of 7 
The following is a schedule of events and tests you will have at each additional visit: 
Procedures 
Open-Label Treatment 
Post-
Treatment 
 
Visit 12 
(Week 32) 
Visit 13 
(Week 40) 
Visit 14 
(Week 48) 
Visit 15 
(Week 52) 
Physical exam 
 
 
 
 
Vital signs 
 
 
 
 
Blood for safety and urine test 
 
 
 
 
Urine pregnancy test, if you are a woman who can 
have a baby 
 
 
 
 
ECG  
 
 
 
 
Examination of your face and calculation of the 
percentage of white patchy areas 
 
 
 
 
Examination of the percentage of your body with 
white patches 
 
 
 
 
Photographs of your face 
 
 
 
 
Questions about how noticeable is your facial vitiligo 
 
 
 
 
Questions about how satisfied you are with the response 
to study drug 
 
 
 
 
Questions about the impact of your facial vitiligo 
 
 
 
 
Questionnaire completion 
 
 
 
 
Receive Study Drug 
 
 
 
 
Health and medication questions 
 
 
 
 
 
DO I HAVE TO CONTINUE IN THIS STUDY? 
Your decision to still be in this study is voluntary.  You do not have to be in the study any longer 
if you don’t want to, and you can change your mind at any time.  There will be no penalty to you, 
and you won’t lose any benefits.  If you want to stop being in the study, tell the study doctor or 
study staff.  If you leave the study, the study doctor and study staff will still be able to use your 
information that they have already collected. 
 
WILL I RECEIVE PAYMENT? 
<<Quorum will add site-specific compensation language to the form based on information the 
site reports to Quorum.>> 
 
WHO CAN I TALK TO ABOUT THIS STUDY? 
In the event of an emergency, dial 911 immediately. 
If you require emergency care, be sure to tell the emergency care provider about your 
participation in this study.  Contact the study doctor or study staff as soon as possible. 
You can ask questions about the study at any time.  You can call the study doctor or study staff 
at any time if you have any concerns or complaints.  You should call the study doctor or study 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 4 of 7 
staff at the phone number listed on page 1 of this form if you have questions about the study 
procedures, study costs (if any), study payment (if any), or if you get hurt or sick during the 
study. 
Quorum Review reviewed this study.  Quorum Review is a group of people who review research 
studies to protect the rights and welfare of research participants.  Review by Quorum Review 
does not mean that the study is without risks.  If you have questions about your rights as a 
research participant, if you are not able to resolve your concerns with the study doctor or study 
staff, if you have a complaint, or if you have general questions about what it means to be in a 
research study, you can call Quorum Review or visit the Quorum Review website at 
www.QuorumReview.com. 
Quorum Review is located in Seattle, Washington. 
Office hours are 8:00 AM to 5:00 PM Pacific Time, Monday through Friday. 
Ask to speak with a Research Participant Liaison at 888-776-9115 (toll free). 
 
HOW WILL MY INFORMATION BE USED AND SHARED FOR THIS STUDY? 
This section explains who will use and share your health information if you agree to be in this 
study.  You must authorize this use and sharing of your information by signing this form or you 
cannot be in the study.  
The study doctor and study staff will collect, use, and share health information about you, 
including any information needed to do the study and other identifying information about you, 
such as your name, address, phone number, or social security number.  The information used 
and shared will include: 
• 
information from your medical records 
• 
information collected about you during the research, including study visits, notes, tests, 
procedures, photographs, etc. 
Your information may be used and shared with these people for the following purposes: 
• 
The study doctor and study staff to conduct this research. 
• 
The sponsor, Aclaris Therapeutics, Inc.; people who work with or for the sponsor; and 
other researchers involved in this study.  These people will use your information to 
review the study, to check the safety and results of the study drug, and to seek 
government approval of ATI-50002 Topical Solution. 
• 
Others required by law to review the quality and safety of research, including the FDA, 
Department of Health and Human Services, Office for Human Research Protections, 
other government agencies in the United States and other countries, and Quorum 
Review. 
After your information is shared with the people and companies listed above, the law may not 
require them to protect the privacy of your information.   
To maintain the integrity of this research, you might not have access to any health information 
developed as part of this study until it is completed.  At that point, you generally would have 
access to your health information. 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 5 of 7 
You can cancel your authorization to use and share your information at any time by writing a 
letter to the study doctor.  If you cancel your authorization, you will not be able to continue in the 
study.   
If you cancel your authorization, the study doctor and study staff will still be able to use and 
share your information that they have already collected. 
This authorization to use and share your information expires in 50 years. 
 
 
 
 
 
 
 
 
 
Signature of Participant 
 
 
 
 
 
Date 
 
<<Quorum staff: Include the following for Indiana sites: 
In Indiana, you must complete the following information: 
 
 
 
 
 
 
 
Participant’s Street Address 
 
 
 
 
 
 
 
Participant’s City, State, ZIP>> 
 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 6 of 7 
REGULAR DOCTOR OR SPECIALIST NOTIFICATION OPTION 
For your safety, you or the study doctor should tell your regular health care provider that you are 
in this study.  This is recommended so that your primary care doctor may contact the study 
doctor if they have any concerns or questions about your care. 
Please indicate below whether you want us to notify your regular doctor or your specialist of 
your participation in this study. 
 
 
Yes, I want the study doctor to inform my regular doctor/specialist of my participation in 
this study: 
 
 
 
 
 
Name of Doctor 
 
 
 
 
 
 
Phone 
 
 
No, I do not want the study doctor to inform my regular doctor/specialist of my 
participation in this study. 
 
I do not have a regular doctor/specialist. 
 
The study doctor is my regular doctor/specialist. 
 
CONSENT 
I have read this form, and I have been able to ask questions about this study. The study doctor 
or study staff has talked with me about this study. They have answered all my questions.  I 
voluntarily agree to be in this study. 
By signing this form, I do not give up any of my legal rights.  I will get a signed copy of this 
consent form. 
 
 
 
 
 
Printed Name of Participant 
 
 
 
 
 
Signature of Participant 
 
 
 
 
 
Date 
 


Information and Consent Form 
Aclaris Therapeutics, Inc. 
ATI-50002-VITI-201 
 
Initials __________    Date __________ 
Version 3, dated 10/15/18 
Page 7 of 7 
I attest that the individual providing consent had enough time to consider this information, had 
an opportunity to ask questions, and voluntarily agreed to participation in this study. 
 
 
 
Printed Name of Person Explaining Consent 
 
 
 
 
 
Signature of Person Explaining Consent 
 
 
 
Date 
 
 
I attest that I or my representative discussed this study with the individual providing consent. 
 
 
 
Signature of Principal Investigator or Sub-Investigator 
 
 

Prot_000.pdf: 
Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
1 
 
 
  
 
CLINICAL STUDY PROTOCOL 
PROTOCOL NUMBER:  ATI-50002-AGA-201 
An Open-Label Safety, Tolerability, and Efficacy Study in Male and Female Subjects with 
Androgenetic Alopecia Treated with ATI-50002 Topical Solution 
 
Original Protocol: V1.0 05 January 2018 
Amendment 1: V2.0 16 January 2018 
Amendment 2:  V3.0 01 February 2018 
Amendment 3:  V4.0 16 October 2018 
Amendment 4:  V5.0 24 January 2019 
 
Sponsor 
Aclaris Therapeutics, Inc. 
640 Lee Road 
Suite 200 
Wayne, PA 19087 
Telephone: 484-324-7933 
Facsimile: 484-320-2344 
 
Study Contact  
Judy Schnyder 
Executive Director, Clinical Operations 
Aclaris Therapeutics, Inc. 
640 Lee Road 
Suite 200 
Wayne, PA 19087 
Telephone: 484-329-2144 
Cell:  215-593-7119 
E-mail: jschnyder@aclaristx.com 
Medical Monitor 
David Gordon, MB, ChB 
 
Chief Medical Officer 
Aclaris Therapeutics, Inc. 
 
This document is a privileged and confidential communication of Aclaris Therapeutics, Inc. Acceptance of this 
document constitutes an agreement by the recipient that no unpublished information contained herein will be used, 
published or disclosed without prior written approval from Aclaris Therapeutics, Inc. 
 
 




Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
3 
 
 
  
 
INVESTIGATOR’S AGREEMENT 
Protocol Number:  ATI-50002-AGA-201  
Protocol Title:  An Open-Label Safety, Tolerability, and Efficacy Study in Male and Female Subjects 
with Androgenetic Alopecia Treated with ATI-50002 Topical Solution 
I have reviewed the above-titled protocol and agree that it contains all the information necessary to 
conduct the study as required.  I will conduct the trial in accordance with the principles of ICH Good 
Clinical Practice and the Declaration of Helsinki. 
I will maintain as confidential all written and verbal information provided to me by the Sponsor, 
including but not limited to, the protocol, case report forms, investigator’s brochure, material supplied at 
investigator meetings, minutes of teleconferences, etc.  Such materials will only be provided as necessary 
to site personnel involved in the conduct of the trial, involved IRBs or local regulatory authorities. 
I will obtain written informed consent from each prospective trial subject or each prospective trial 
subject’s legal representative prior to conducting any protocol-specified procedures.  The Informed 
Consent Document used will have the approval of the IRB appropriate for my institution. 
I will maintain adequate source documents and record all observations, treatments and procedures 
pertinent to trial subjects in their medical records.  I will accurately complete the case report forms 
supplied by the Sponsor in a timely manner.  I will ensure that my facilities and records will be available 
for inspection by representatives of the Sponsor, the IRB, and/or local regulatory authorities.  I will 
ensure that I and my staff are available to meet with Sponsor representatives during regularly scheduled 
monitoring visits. 
I will notify the Sponsor within 24 hours of any serious adverse events.  Following this notification, a 
written report describing the serious adverse event will be provided to the Sponsor as soon as possible, 
but no later than five days following the initial notification. 
Investigator Signature: 
 
 
 ______________________________________ 
 _________________________  
Investigator signature 
 
Date 
 
 
 
 ______________________________________ 
Investigator printed name 


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
4 
 
 
  
 
1. 
SYNOPSIS 
Name of Sponsor/Company:  
Aclaris Therapeutics, Inc. 
Name of Investigational Product:  
ATI-50002 Topical Solution 0.46% 
Title of Study:  
An Open-Label Safety, Tolerability, and Efficacy Study in Male and Female Subjects with 
Androgenetic Alopecia Treated with ATI-50002 Topical Solution 
Study center(s):  Approximately 3 investigational centers will participate in the study. 
 Phase of development:  
Phase 2a 
Objectives:  
Primary: 
• 
To assess safety, tolerability and efficacy of ATI-50002 Topical Solution, 0.46% in male and 
female subjects with androgenetic alopecia (AGA) 
• 
Secondary: 
• 
To assess the development of new hair growth/scalp coverage as assessed by the investigator and 
the subject using a seven-point rating scale and global scalp photographs. 
• 
To assess the change from baseline in cumulative target area hair width 
• 
To assess the change from baseline in androgenetic alopecia stage according to the Norwood-
Hamilton scale in males and the Sinclair Scale in females.   
Study Design:  
This is an open-label phase 2 study designed to evaluate the safety and efficacy of ATI-
50002 Topical Solution, 0.46% in male and female subjects with androgenetic alopecia.  
Subjects will be required to apply ATI-50002 study medication to their scalp twice a day for 
a total of 26 weeks.  Upon completion of the first 26 weeks of treatment, subjects will have 
the option to consent to continue on study medication for an additional 26 weeks of 
treatment.  All subjects will be required to complete a safety follow up visit4 weeks after last 
application of study medication.   
A total of 24 subjects (approximately 12 males and 12 females) ages 18-50 years with a clinical 
diagnosis of AGA will be enrolled in the study.  Male subjects must have a Norwood classification of 
Type III vertex, IV or V and woman must have a Sinclair Grade of 2, 3 or 4. 
Number of patients (planned):  
Approximately 12 male subjects and 12 female subjects will be enrolled to the study.  
Inclusion Criteria:  
Subjects must meet the following criteria to be eligible for participation in the study: 
1. Able to comprehend and willing to sign an Informed Consent Form (ICF). 


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
5 
 
 
  
 
2. Male subjects and non-pregnant, non-nursing female subjects18 to 50 years of age with a clinical 
diagnosis of AGA.     
3. Male subjects with a Norwood-Hamilton classified as III vertex, IV or V and woman subjects 
with a Sinclair Grade 2, 3 or 4. 
4.  Subjects willing to agree to have small 1.9 cm diameter circle of hair clipped to ~ 1 mm in length 
on their balding scalp.  
5. Subjects willing to agree to have a permanent dot tattoo applied to their scalp to mark the center 
of the identified target area 
6. Subjects must agree to maintain the same hair style and hair care regimen during the study.  
7. Subjects must agree to not use a medicated shampoo or conditioner containing biotin, saw 
palmetto or tea tree oil (melaleuca oil) throughout the course of protocol treatment and refrain 
from hair weaving, hair extensions, texturizers, relaxers, occlusive wigs and non-study hair 
growth products during the study and for a period of 30 days prior to Visit 2. 
8. Subjects must have an absolute neutrophil count (ANC) >1,000/mm3, and a platelet count 
>50,000/mL. 
9. Male subjects with a serum prostate-specific antigen (PSA) level of < 2.0 ng/mL. 
10. Subjects receiving oral or topical hormones must be on a stable dose for 6 months prior to V2.   
11. Subjects must be willing to refrain from washing hair, using hair products, and participating in 
strenuous exercise that would cause profuse sweating for 6 hours after each treatment application 
with ATI-50002.   
12. Woman of child bearing potential (WOCBP) must agree to use an approved method of highly 
effective birth control for the duration of the study and for 30 days after last study medication 
application.  
13. Sexually active  male subjects must agree to use a barrier form of birth control from the first 
application until 30 days post last application of ATI-50002.  
Exclusion Criteria: 
Subjects are excluded from this study if any 1 or more of the following criteria is met: 
1. Females who are nursing, pregnant, or planning to become pregnant for the duration of the study 
and up to 30 days after the last application of study medication. 
2. Clinical diagnosis of alopecia areata or other non-AGA forms of alopecia. 
3. Scalp hair loss on the treatment area, due to disease, injury or medical therapy.   
4. Active skin disease on the scalp (such as psoriasis or seborrheic dermatitis) or a history of skin 
disease on the scalp that in the opinion of the investigator would interfere with the study assessments 
of efficacy or safety. 
5. Evidence of hypogonadism defined as serum testosterone < 250ng/dl and/or LH>20mU/ml in 
males. 
6. Active unstable thyroid disease, including subjects on therapy for either hyperthyroidism or 
hypothyroidism unless their dose of thyroid medication has been stable for 3 months prior to Visit 2 
and serum TSH is normal.   
7. Subjects with non-pigmented hair. 


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
6 
 
 
  
 
8. Subjects unable to comply with the following required washout periods: 
• 
Scalp promoting hair growth products (e.g.minoxidil, finasteride, dutasteride, saw 
palmetto) for 6 months prior to Visit 2. 
• 
Anti-androgenetic therapies (e.g. spironolactone, ketoconazole, flutamide, cyproterone 
acetate) for 6 months prior to Visit 2.   
• 
Hair transplantation or hair weaving within 6 months of Visit 2 
• 
Anabolic steroids for 6 weeks prior to Visit 2.  
• 
Oral or topical JAK inhibitors 3 months prior to Visit 2 
• 
Platelet rich plasma treatment within 6 months of Visit 2 
• 
Hair Laser, light or energy treatments to the target hair area within 3 months of Visit 2 
• 
Systemic steroids for more than 14 consecutive days in the 2 months prior to Visit 2 
• 
Chemotherapy, cytotoxic agents within 12 months of Visit 2 
• 
No prior radiation to the scalp  
9. History of, current or suspected systemic or cutaneous malignancy and /or lymphoproliferative 
disease, other than subjects with: a history of adequately treated and well healed and completely cleared 
non-melanoma skin cancers (basal or squamous cell carcinoma) treated successfully with no evidence 
of disease.   
10. History of human immunodeficiency virus (HIV), hepatitis B or C.  
11. Current herpes zoster infection. 
12. Evidence of active, latent or inadequately treated Mycobacterium tuberculosis infection, or history 
of incompletely treated or untreated tuberculosis. Subjects who have successfully completed therapy 
for latent tuberculosis may participate.  
13. History of serious local infection (e.g., cellulitis, abscess) or systemic infection including but not 
limited to a history of treated infection (e.g., pneumonia, septicemia) within 3 months prior to Visit 1. 
Subjects on an antibiotic for a non-serious, acute local infection must complete the course prior to 
enrollment into the study. 
14. Participation in an investigational drug, device, or biologic trial in which administration of an 
investigational drug, device, or biologic occurred within 30 days or 5 half-lives (whichever is 
longer) of Visit 2.  Subjects who have participated in a study of an investigational drug, device or 
biologic agent for androgenetic alopecia within 1 year of screening will be eligible to participate 
only with individual permission from the Medical Monitor. 
15. Sensitivity to any of the ingredients in the study medication. 
16. Screening ECG findings of: 
• 
QTcF >450msec for males or >470msec for females (use of the ECG algorithm is acceptable 
for this purpose) 
• 
Resting heart rate < 45 or > 100 beats/minutes (inclusive) 
• 
Rhythm disturbance other than sinus arrhythmia or ectopic supraventricular rhythm (ectopic 
atrial rhythm) 
• 
Conduction disturbance including PR >240msec, pre-excitation (delta wave and PR 
<120msec), second degree or higher AV block 
• 
Acute or chronic signs of ischemia  


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
7 
 
 
  
 
• 
Left Bundle Branch Block 
• 
Prior myocardial infarction 
Investigational product, dosage and mode of administration:  
Open label ATI-50002 Topical Solution 0.46% will be applied to the balding area of male and female 
subjects with androgenetic alopecia.  Up to 4mls of ATI-50002 Topical Solution 0.46% will be 
applied to the scalps of eligible study subjects twice a day using a 1ml disposable dropper.   
Duration of treatment:  
All subjects will apply ATI-50002 Topical Solution 0.46% twice a day for 26 weeks.  Subjects will 
have the option to consent to remain on study for an additional 26 weeks.  All subjects will be 
required to have a safety follow up visit 4 weeks after their last application of study medication.   
Study Endpoints:   
Efficacy: 
The primary efficacy endpoint is the change from baseline in non-vellus target area hair count 
(TAHC)using a microphotographic technique. 
 
The secondary efficacy endpoints are defined as: 
• development of new hair growth/scalp coverage as assessed by the investigator and 
the subject using a seven-point rating scale and subject global scalp photographs.  
• Change from baseline in cumulative target area hair width (TAHW)  
• Change from baseline in AGA stage according to the Norwood Hamilton scale in 
males and the Sinclair Scale in females 
Safety: 
Subjects will be assessed for local skin reactions and adverse events throughout the duration of the 
trial.  In addition, vital signs, ECGs and clinical laboratory assessments will be performed on all 
subjects at the protocol defined time-points.  
Statistical methods: 
Summary descriptive statistics (N, mean, median, SD) by visit will be provided for all safety and 
efficacy parameters. The Intent to Treat population will be used for all efficacy assessments and the 
Safety population will be used for all safety assessments. No data imputation will be used.  Primary 
and secondary efficacy parameters are described below: 
 
Primary Efficacy Endpoints: 
Mean change from baseline in non-vellus target area hair count (TAHC)will be quantified using a 
microphotographic technique and calculated along with the median, standard deviation, standard 
error, and 95% confidence limits around each mean change to allow tests of the null hypothesis that 
the population mean changes are zero. 
 
Secondary Efficacy Endpoints: 
Mean change from baseline in cumulative target area hair width (TAHW) will be quantified using a 
microphotographic technique and calculated along with the median, standard deviation, standard 
error, and 95% confidence limits around each mean change to allow tests of the null hypothesis that 
the population mean changes are zero.  


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
8 
 
 
  
 
The seven-point rating scale to measure the investigator and subject assessment of the development of 
new hair growth/scalp coverage will be analyzed with summary statistics (N, mean, median, SD) for 
each visit where the assessment is made.  Similar summary statistics will be used to analyze change 
from baseline in AGA stage separately for males (Horwood Hamilton Scale) and for females (Sinclair 
Scale) at each applicable visit.  
 
Safety Assessments: 
Data from all enrolled and treated subjects will be presented and summarized. Safety analyses will 
include descriptive statistics calculated on the safety parameters using the safety population. The 
proportion of subjects with treatment-emergent adverse events will be tabulated and presented by 
Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class. Vital signs and 
clinically significant abnormal laboratory results will also be tabulated and presented.  Clinical 
laboratory and ECG results will be tabulated, highlighting abnormal results.  Local skin reaction 
results will be tabulated and summarized by visit. 
 
 


Protocol: ATI-50002-AGA-201 
Version 5.0:   24 January 2019 
Amendment 4 
 
 
 
 
9 
 
 
  
 
2. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF 
FIGURES  
TABLE OF CONTENTS 
INVESTIGATOR’S AGREEMENT ...............................................................................................3 
1. 
SYNOPSIS ...................................................................................................................4 
2. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF FIGURES .................9 
TABLE OF CONTENTS .................................................................................................................9 
LIST OF TABLES .........................................................................................................................12 
LIST OF FIGURES .......................................................................................................................13 
3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS.............................14 
4. 
AMENDMENT HISTORY ........................................................................................16 
4.1. 
Amendment Rationale ................................................................................................16 
4.2. 
Protocol Changes ........................................................................................................17 
5. 
INTRODUCTION ......................................................................................................19 
5.1. 
Overview of Androgenetic Alopecia ..........................................................................19 
5.2. 
Rationale for Using ATI-50002 to Treat AGA ...........................................................19 
6. 
TRIAL OBJECTIVES AND PURPOSE ....................................................................21 
6.1. 
Primary Objective .......................................................................................................21 
6.2. 
Secondary Objectives .................................................................................................21 
7. 
INVESTIGATIONAL PLAN .....................................................................................22 
7.1. 
Overall Study Design ..................................................................................................22 
7.2. 
Number of Subjects ....................................................................................................23 
7.3. 
Treatment Assignment ................................................................................................23 
7.4. 
Criteria for Study Termination ...................................................................................23 
7.5. 
Schedule of Assessments ............................................................................................24 
Table 2: 
Schedule of Assessments-Initial 26 weeks of Treatment ...........................................24 
8. 
SELECTION AND WITHDRAWAL OF SUBJECTS ..............................................31 
8.1. 
Subject Inclusion Criteria ...........................................................................................31 
8.2. 
Subject Exclusion Criteria ..........................................................................................31 
8.3. 
Subject Withdrawal Criteria .......................................................................................33 
9. 
TREATMENT OF SUBJECTS ..................................................................................35 
9.1. 
Hair Care During Study ..............................................................................................35 


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9.2. 
Target Area Hair Identification ..................................................................................35 
9.3. 
Preparation of Subjects Scalp for Global and Macro Photographs ............................36 
9.4. 
Scalp Tattoo ................................................................................................................37 
9.5. 
Application of Study Medication ................................................................................37 
9.6. 
Concomitant Medications ...........................................................................................38 
9.7. 
Prohibited Medications ...............................................................................................38 
9.8. 
Treatment Compliance ................................................................................................38 
9.9. 
Study Medication Interruption ....................................................................................38 
9.10. 
Randomization and Blinding ......................................................................................39 
10. 
STUDY DRUG MATERIALS AND MANAGEMENT ...........................................40 
10.1. 
Study Drug ..................................................................................................................40 
10.2. 
Study Drug Packaging, Labeling and Storage ............................................................40 
10.3. 
Study Drug Accountability and Disposal ...................................................................41 
10.4. 
Additional Clinical Supplies .......................................................................................41 
11. 
ASSESSMENT OF EFFICACY ................................................................................42 
11.1. 
Target Area Hair Count (TAHC) and Target Area Hair Width (TAHW) ..................42 
11.2. 
Norwood-Hamilton and Sinclair Scales .....................................................................42 
11.3. 
Investigator Global Assessment and Subject Self-Assessment ..................................42 
12. 
ASSESSMENT OF SAFETY .....................................................................................44 
12.1. 
Safety Parameters .......................................................................................................44 
12.1.1. 
Demographic/Medical History ...................................................................................44 
12.1.2. 
Vital Signs ..................................................................................................................44 
12.1.3. 
Physical Examination .................................................................................................44 
12.1.4. 
Electrocardiogram (ECG) ...........................................................................................44 
12.1.5. 
Pregnancy Tests ..........................................................................................................45 
12.1.6. 
Local Skin Reactions ..................................................................................................45 
12.1.7. 
Laboratory Assessments .............................................................................................46 
12.1.7.1. 
Clinical Chemistry/Hematology .................................................................................46 
12.1.7.2. 
Virus Serology ............................................................................................................47 
12.2. 
Adverse and Serious Adverse Events .........................................................................47 
12.2.1. 
Definition of Adverse Events .....................................................................................47 
12.2.1.1. 
Adverse Event (AE) ....................................................................................................47 


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12.2.1.2. 
Serious Adverse Event (SAE) ....................................................................................48 
12.2.1.3. 
Unexpected adverse event ..........................................................................................48 
12.3. 
Recording Adverse Events .........................................................................................48 
12.3.1. 
Adverse event reporting period ..................................................................................48 
12.3.2. 
Severity .......................................................................................................................49 
12.3.3. 
Relationship to study medication ................................................................................49 
12.3.4. 
Procedures for reporting adverse events .....................................................................49 
12.3.5. 
Procedure for reporting a serious adverse event .........................................................50 
12.4. 
Pregnancy ...................................................................................................................50 
12.4.1. 
Definition of Women of Child Bearing Potential (WOCBP) .....................................50 
12.4.2. 
Highly Effective Methods of Birth Control ................................................................50 
12.5. 
Protocol Deviation and Protocol Violations ...............................................................52 
13. 
STATISTICS ..............................................................................................................53 
13.1. 
Statistical Methods ......................................................................................................53 
13.2. 
Primary Efficacy Endpoint .........................................................................................53 
13.3. 
Secondary Efficacy Endpoints ....................................................................................53 
13.4. 
Safety Assessments .....................................................................................................53 
14. 
QUALITY CONTROL AND QUALITY ASSURANCE .........................................54 
15. 
ETHICS AND GENERAL STUDY CONDUCT CONSIDERATIONS ...................55 
15.1. 
Institutional Review Board (IRB)/Ethics Committee (EC) ........................................55 
15.2. 
Ethical Conduct of the Study ......................................................................................55 
15.3. 
Written Informed Consent ..........................................................................................55 
15.4. 
Study Conduct and Protocol Amendments .................................................................55 
15.5. 
Regulatory Documents ...............................................................................................56 
15.6. 
Contractual Requirements ..........................................................................................56 
16. 
DATA HANDLING AND RECORDKEEPING .......................................................57 
16.1. 
Data Collection ...........................................................................................................57 
16.2. 
Study Monitoring ........................................................................................................57 
16.3. 
Source Documentation ................................................................................................58 
16.4. 
Inspection of Records .................................................................................................58 
16.5. 
Retention of Records ..................................................................................................58 
17. 
REFERENCES ...........................................................................................................59 


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18. 
APPENDICES ............................................................................................................60 
APPENDIX 1. SUBJECT INSTRUCTIONS FOR STUDY MEDICATION 
APPLICATION TO THE SCALP ..............................................................................61 
APPENDIX 2. NORWOOD HAMILTON SCALE ...................................................................63 
APPENDIX 3. SINCLAIR SCALE ............................................................................................65 
APPENDIX 4. GLOBAL PHOTOGRAPHY PROCEDURE ....................................................66 
APPENDIX 5. MACRO HAIRCOUNT PHOTOGRAPHY AND IMAGE ANALYSIS 
PROCEDURE.............................................................................................................70 
LIST OF TABLES 
Table 1: 
Abbreviations and Specialist Terms ...........................................................................14 
Table 2: 
Schedule of Assessments-Initial 26 weeks of Treatment ...........................................24 
Table 3: 
Schedule of Assessments -6 month Extension Study .................................................28 
Table 4: 
Study Medication Interruption Criteria ......................................................................39 
Table 5: 
Investigational Product ...............................................................................................40 
Table 6: 
7-Point Rating Scale of Subjects Scalp Hair Based on Evaluation of Global 
Photographs ................................................................................................................42 
 


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LIST OF FIGURES 
Figure 1: 
Target Area Identification ...........................................................................................36 
 
  


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3. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
The following abbreviations and specialist terms are used in this study protocol. 
Table 1: 
Abbreviations and Specialist Terms  
Abbreviation or Specialist Term 
Explanation 
AA 
Alopecia areata 
AE 
Adverse event 
AGA 
Androgenetic alopecia 
ALT 
Alanine aminotransferase 
ANC 
Absolute neutrophil count 
AST 
Aspartate aminotransferase 
AT 
Alopecia totalis 
AU 
Alopecia universalis 
AV 
Atrioventricular 
BCC 
Basil cell carcinoma 
BUN 
Blood urea nitrogen 
CPK 
Creatine Phosphokinase 
DHT 
Dihydrotestosterone 
DMSO 
Dimethylsulfoxide 
EC 
Ethics committee 
ECG 
Electrocardiogram 
eCRF 
Electronic case report form 
FSH 
Follicle stimulating hormone 
GCP 
Good Clinical Practice 
HF 
Hair follicle 
HFSC 
Hair follicle stem cell 
HDL 
High-density lipoprotein 
HIV 
Human immunodeficiency virus 
ICH 
International Conference on Harmonization 
IRB 
Institutional Review Board 
JAK1 
Janus kinase 1 
JAK2 
Janus kinase 2 
JAK3 
Janus kinase 3 
LDH 
Lactate dehydrogenase 


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Abbreviation or Specialist Term 
Explanation 
LDL 
Low-density lipoproteins 
LH 
Lutenizing hormone 
OAE 
Other significant adverse event 
OSM 
Oncostatin M  
OSMR 
Oncostatin M receptor 
PI 
Principal Investigator 
SAE 
Serious adverse event 
SCC 
Squamous cell carcinoma 
TIBC 
Total iron binding capacity 
TAHC 
Target Area Hair Count 
TAHW 
Target Area Hair Width 
TyK2 
Tyrosine kinase 2 
WOCBP 
Woman of child bearing potential 
WBC 
White blood cell  
 


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4. 
AMENDMENT HISTORY 
4.1. 
Amendment Rationale 
Version 2.0 amendment 1 dated 16 January 2018, clarified the size of the area of the subject’s 
scalp that would be required to be clipped at each protocol defined timepoint.  The original 
protocol identified the area on the subject’s as a 1cm2 target area.  The accurate clipping size to 
perform the TAHC and TAHD is a 1.9 cm diameter circle clipped to ~ 1mm in length.  The 
subject inclusion criteria were also amended to require that subjects refrain from strenuous 
exercise that would cause profuse sweating for a period of 6 hours after application of study 
medication.  This requirement was added to ensure that the study medication was absorbed prior 
to performing any strenuous exercise.  The subject exclusion criteria were amended to allow 
subjects to enroll to the study if they have previously received treatment with a topical JAK 
inhibitor.  The use of systemic steroids for more than 14-day s in the 2 months prior to Visit 2 
was added to the prohibited medication section and the use of anabolic steroids prior to Visit 2 
was changed from 3 months to 6 weeks.   Section 11.3.6 was added to the protocol to provide 
definitions for protocol violations and protocol deviations.   
 
Version 3.0 amendment 2 dated 01 February 2018, added in the secondary objective to assess the 
change from baseline in target area hair width.  This objective should have been included in the 
original protocol.  The protocol was also amended to allow pre-menopausal women to enroll to 
the study.   
 
Version 4.0 amendment 3 dated 16 October 2018, allows subjects enrolled to the study to receive 
an additional 6 months of study medication.  Creatine Phosphokinase (CPK) was also added to 
the chemistry panel as an additional safety assessment.  The amount of study medication 
dispensed at each visit was also amended.  The previous versions of the protocol required that 3 
bottles of study medication be dispensed at each visit due to the short stability of the product 
once the bottles were opened.  New stability data has become available to allow the product to be 
used for 60 days after the bottle is opened.  This allows for less study medication to be dispensed 
between study visits.   
 
Version 5.0 amendment 4 dated 22 January 2019, has been amended to allow the investigator to 
perform the Investigator Global Assessment by comparing the current subject photos to the  to 
the baseline (Visit 2) photos.  The protocol was also amended to allow the subject to perform the 
Subject Self-Assessment by comparing the current subject photos to the  to the baseline (Visit 2) 
photographs.  This revision will enable both the investigator and subject to assess changes in hair 
growth over the duration of the study.   
 


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4.2. 
Protocol Changes 
Protocol 
Version 
Date 
Section 
Revisions 
Version 1.0 
5 January 2018 
NA 
Original Protocol  
Version 2.0 
Amendment 
1: 
16 January 2018 
Synopsis 
Inclusion and Exclusion criteria updated to reflect all 
changes in main body of protocol 
 
 
Section 6.1; 
Section 7.1; 
section 10.1 
Clarify the diameter size of the area on the subject’s 
scalp that is required to be clipped for the TAHC and 
TAHD assessments. 
 
 
Section 7.1; 
Appendix 1 
Subject 
Instructions 
Added language to Inclusion number 11 to require that 
subjects refrain from participating in strenuous 
exercise that would cause profuse sweating for a period 
of 6 hours after study medication application.   
 
 
Section 7.2 
Removed exclusion criteria of the use of topical JAK 
inhibitors and removed exclusion number 8 due to a 
duplication of the exclusion criteria.   
 
 
Section 8.7 
Changed the restricted use of anabolic steroids from 3 
months to 6 weeks prior to Visit 2 and added the 
restriction of using systemic steroids for more than 14 
consecutive days in the 2 months prior to Visit 2.   
 
 
Section 
11.3.6  
Added section to provide definition for protocol 
violations and deviations.   
Version 3.0 
Amendment 
2: 
01 February 2018 
Front Cover 
Front Cover of protocol updated with new address for 
Aclaris Therapeutics 
 
 
Synopsis 
Synopsis was updated to reflect all changes in the main 
body of the protocol.   
 
 
Section 5.2 
Added secondary objective to assess the change from 
baseline in cumulative target area hair width 
 
 
Section 6.5 
Added urine pregnancy and serum pregnancy testing fo 
woman and added Target Area Hair Width.   
 
 
Section 7.1 
Revised the inclusion criteria for woman to allow pre-
menopausal women to enroll to the study and to add in 
the required birth control procedures for woman being 
treated on the study.   


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Protocol 
Version 
Date 
Section 
Revisions 
 
 
Section 7.2 
Added an exclusion criteria to exclude female subjects 
who plan to get pregnant 30 days after last application 
of study medication.   
Version 4.0  
Amendment 
3 
16 October 2018 
Synopsis; 
Section 7.1, 
Section 7.5; 
Table 3 
Updated the study design section to state that subjects 
will be allowed to remain on study for an additional 6 
months.  New Table of assessments added for those 
subjects that consent to remain on study beyond the 
initial 26 weeks of treatment.   
 
 
Section 7.5; 
Section 
12.1.7 
Addition of Creatine Phosphokinase (CPK) to the 
chemistry panel as an additional safety assessment.   
 
 
Section 8.5 
and Section 
9.2 
Removed the requirement of having to dispense 3 
bottles of study medication at each visit due to the 
longer stability data of the opened bottles.   
Version 5 
Amendment 
4 
24 January 2019 
Front Cover 
Removed Stuart Shanler, MD as the medical monitor 
and replaced David Gordon MB, ChB.  
 
 
Section 11.3 
Added language to have the investigator perform the 
Investigator Global Assessment by comparing the 
current subject photos back to the baseline visit (Visit 
2).  Added language to have the subject perform the 
Subject Self-Assessment by comparing the current 
subject photo back to the baseline visit (Visit 2).   
 


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5. 
INTRODUCTION 
Aclaris Therapeutics, Inc. is developing ATI-50002 Topical Solution as topical treatment for alopecia 
areata (AA), alopecia totalis (AT), alopecia universalis (AU), androgenetic alopecia (AGA), and vitiligo.  
ATI-50002, the pharmacologically active metabolite of the orally administered ATI-50001, is a potent 
and highly selective inhibitor of Janus kinase 1 (JAK1) and Janus kinase 3 (JAK3)-dependent cytokine 
signaling, with minimal activity on JAK2- and Tyrosine kinase-2 (Tyk2)-dependent signaling. 
5.1. 
Overview of Androgenetic Alopecia 
Androgenetic alopecia is a hair loss disorder that affects both men and woman.  It is an 
extremely common disorder with relatively few treatment options.  There are only two FDA 
approved treatment options for men [Rogaine® (minoxidil) and Propecia® (finasteride)] and one 
FDA approved treatment for women (Rogaine®).   The disorder affects roughly 50% of men and 
perhaps as many women and the frequency increases with age in both men and women (Olsen E, 
et al., 2005) .  The pattern of hair loss is gradual in both men and women but the way in which 
the hair is lost differs.  Onset in men may begin any time after the onset of puberty and occurs in 
varying degrees in the bitemporal, frontal, mid scalp and vertex regions of the head (Olsen E, et 
al., 2005).  Woman with androgenetic alopecia experience diffuse thinning of the hair.   
Androgenetic alopecia can be an emotionally debilitating disease for both men and woman.    
5.2. 
Rationale for Using ATI-50002 to Treat AGA 
Recent studies in the laboratory of Dr. Angela Christiano (Columbia University; submitted) 
found that inhibition of JAK-STAT signaling initiates the hair cycle in normal mice and 
promotes hair growth in humans.  Particularly, in normal telogen phase, JAK signaling is 
elevated and is involved in maintaining quiescence of hair follicle stem cells.  Administering a 
JAK inhibitor results in initiating entry into anagen causing hair growth by inhibiting JAK-STAT 
signaling so that the hair follicle is no longer quiescent.  
Dr. Christiano’s laboratory further identified upstream factors that signal via the JAK-STAT 
pathway to promote hair growth.  Oncostatin M (OSM) is one such upstream factor.  OSM 
receptor (OSMR) is expressed on hair follicle stem cells, and all JAKs (JAK1, JAK2, JAK3, 
Tyk2) are activated by OSM.  Signaling from the JAKs is transduced by STATs, primarily via 
STAT5, in hair follicle keratinocytes.   
Unique anti-inflammatory macrophages, that are resident in the dermis near hair follicles, were 
identified as the source of OSM that promotes hair growth in the follicle.  These unique 
macrophages have been termed “trichophages” due to their having three distinct differences from 
typical M2-type macrophages.  Trichophages produce OSM during early and mid-telogen, which 
signals via JAK-STAT5 in hair follicle stem cells (HFSC) to inhibit proliferation and maintain 
hair follicle quiescence, particularly during second telogen.  These trichophages predominate 
during early and mid-telogen, and lose their OSM producing ability as telogen progresses, which 
allows the hair follicles (HFs) to enter the next anagen stage.   
Based on the above findings, as well as prior findings that administering recombinant OSM 
potently inhibits anagen (Yu, M.et al, 2008) Dr. Christiano anticipated that administering a 


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JAK/STAT inhibitor should inhibit OSM signaling, initiate anagen and associated hair growth.  
Christiano’s laboratory has demonstrated this activity in a mouse mode of AGA. 
ATI-50002 is a JAK1/3 inhibitor with enzyme IC50 activities of 2nM and 36nM against JAK1 
and JAK3, respectively (data on file).  At a cellular/physiologic level, ATI-50002 inhibits 
interferon-γ-induced pSTAT1 activation with IC50 of 38nM (data on file).  The anticipated IC90 
concentration for pSTAT1 activation is approximately 0.4μM.  To expect ATI-50002 to inhibit 
JAK1/3-mediated pSTAT signaling, a significant excess of the inhibitor must be present in the 
target tissue.  The ability of the 0.46% ATI-50002 formulation to deliver ATI-50002 into the 
dermis was studied using a human skin permeation model. (Franz chamber with human skin).  
Resulting ATI-50002 concentrations were found to be in the range of 10-20μM in the dermis and 
epidermis.  A 10μM concentration represents a > 10X excess to the estimated IC90 for pSTAT 
inhibition.  The 0.46% ATI-50002 formulation is, therefore, anticipated to deliver sufficient drug 
to the epidermis and the dermis to inhibit JAK1/3 signaling and diminish immune-mediated 
inflammation.   
 


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6. 
TRIAL OBJECTIVES AND PURPOSE 
6.1. 
Primary Objective 
 
The primary objective is to assess the safety, tolerability and efficacy of ATI-50002 Topical Solution, 
0.46% in male and female subjects with AGA. 
6.2. 
Secondary Objectives 
The secondary objectives will assess the following: 
• development of new hair growth/scalp coverage as assessed by the investigator and the 
subject using a seven-point rating scale and global scalp photographs.   
• to assess the change from baseline in cumulative target area hair width (TAHW) 
• assess the change in AGA stage according to the Norwood Hamilton scale in males and 
the Sinclair Scale in females. 
 


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7. 
INVESTIGATIONAL PLAN 
7.1. 
Overall Study Design 
This is an open-label phase 2 study designed to evaluate the safety and efficacy of ATI-50002 
Topical Solution, 0.46% in male and female subjects with androgenetic alopecia.  Subjects will 
be required to apply ATI-50002 study medication to their scalp twice a day for a total of 26 
weeks.  Subjects will have the option to consent to remain on study for an additional 26 weeks 
after Visit 10 is completed.  All subjects will be required to complete a safety follow up visit 4 
weeks after their last application of study medication.   
A total of 24 subjects (12 males and 12 females) ages 18-50 years of age with a clinical diagnosis 
of AGA will be enrolled in the study.  Male subjects must have a Norwood Hamilton 
classification of Type III vertex, IV or V and woman must have a Sinclair Grade of 2, 3 or 4.   
Subjects will be required to have a small 1.9 cm diameter circle clipped to ~ 1 mm in length on 
their balding scalp.  Subjects must also agree to have a dot tattoo applied in the center of the 
clipped area during Visit 2.     
Subjects will be required to complete the following treatment visits: 
Visit 1:  Screening (Day -14-0) 
Visit 2:  Day 1 
Visit 3:  Day 21 
Visit 4:  Day 28 
Visit 5:  Day 56 
Visit 6:  Day 84 
Visit 7:  Day 112 
Visit 8:  Day 140 
Visit 9:  Day 168 
Visit 10:  Day 182 
Subjects that consent to remain on study for an additional 6 months of treatment will have the 
following study visits: 
 
 
Visit 11:  Week 30 
 
 
Visit 12:  Week 35 
 
 
Visit 13:  Week 39 
 
 
Visit 14:  Week 44 
 
 
Visit 15:  Week 48 
 
 
Visit 16:  Week 52 
 


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All subjects will be required to have a Follow up Safety Visit 4 weeks post last application of 
study medication.  
All protocol defined treatment assessments are outlined in Table 2 .   
7.2. 
Number of Subjects 
A total of 24 evaluable subjects will be enrolled to the study (12 male and 12 female) at 
approximately 3 investigational centers.   
7.3. 
Treatment Assignment 
This is an open label study.  All subjects will apply ATI-50002 Topical Solution 0.46% twice a 
day for 26 weeks followed by a 4 week follow up period.   
7.4. 
Criteria for Study Termination 
This study may be terminated prematurely in whole or in part due to a change in the benefit/risk 
profile for ATI-50002 Topical Solution such that continuation of the study would not be justified 
on medical or ethical grounds.  This determination may be made by the Study Investigators in 
conjunction with the Sponsor, or by IRB or the U.S. Food and Drug Administration (FDA).  The 
Sponsor may also elect to terminate the study if enrollment is sufficiently slow to prevent the 
completion of the study in an acceptable timeframe, or if ATI-50002 development is 
discontinued. 
If the study is terminated prematurely, the Sponsor will notify the Study Investigators and the 
FDA. The Investigator must promptly notify all enrolled subjects and the IRB of study 
termination. 
 


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7.5. 
Schedule of Assessments 
Table 2: Schedule of Assessments-Initial 26 weeks of Treatment 
 
Screening 
Baseline 
Treatment 
Follow up 
Safety/Early 
Termination 
Visit15 
 
Visit 1 
Visit 2 
Visit 3 
Visit 4 
Visit 5 
Visit 6 
Visit 7 
Visit 8 
Visit 9 
Visit 10 
 
 
 
 
Week 3 
Week 4 
Week 8 
Week 
12 
Week 
16 
Week 
20 
Week 
24 
Week 
26 
4 weeks post last 
application of 
ATI-50002 
Treatment Day  
-14 to 0 
1 
21 
28 
56 
84 
112 
140 
168 
182 
 
Visit Windows 
N/A 
N/A 
+/- 3 
days 
+/-4 
days 
+7 days 
+/-7 
days 
+7 days 
+/-7 
days 
+/-7 
days 
+7 days 
+7 days 
Informed Consent1 
X 
 
 
 
 
 
 
 
 
 
 
Inclusion/Exclusion 
Criteria 
X 
X 
 
 
 
 
 
 
 
 
 
Demographics and 
Medical History 
X 
 
 
 
 
 
 
 
 
 
 
Physical Exam2 
X 
 
 
 
 
 
 
 
 
 
X 
Vital Signs3 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Fitzpatrick Skin 
Type 
X 
 
 
 
 
 
 
 
 
 
 
Prior Hair 
Treatment 
Assessment4 
X 
 
 
 
 
 
 
 
 
 
 


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Screening 
Baseline 
Treatment 
Follow up 
Safety/Early 
Termination 
Visit15 
 
Visit 1 
Visit 2 
Visit 3 
Visit 4 
Visit 5 
Visit 6 
Visit 7 
Visit 8 
Visit 9 
Visit 10 
 
 
 
 
Week 3 
Week 4 
Week 8 
Week 
12 
Week 
16 
Week 
20 
Week 
24 
Week 
26 
4 weeks post last 
application of 
ATI-50002 
Treatment Day  
-14 to 0 
1 
21 
28 
56 
84 
112 
140 
168 
182 
 
Visit Windows 
N/A 
N/A 
+/- 3 
days 
+/-4 
days 
+7 days 
+/-7 
days 
+7 days 
+/-7 
days 
+/-7 
days 
+7 days 
+7 days 
Clinical Chemistry 
and CBC5 
X 
 
 
 
X 
 
X 
 
X 
X 
X 
Quantiferon Gold6 
X 
 
 
 
 
 
 
 
 
 
 
Total Iron Binding 
Capacity (TBIC), 
Serum Iron, Serum 
Ferritin 
X 
 
 
 
 
 
 
 
 
 
 
Dihydrotestosterone 
(DHT), 
Testosterone, 
Estrogen, DHEA 
X 
 
 
 
X 
 
X 
 
X 
X 
X 
Urine Pregnancy 
Test 
 
X 
 
X 
X 
X 
X 
X 
X 
X 
X 
Serum Pregnancy  
X 
 
 
 
 
 
 
 
 
 
 
Serum PSA  
X 
 
 
 
 
 
 
 
 
 
 
Thyroid Panel 
X 
 
 
 
 
 
 
 
 
 
 
ECG 
X 
 
 
X 
 
 
 
 
 
X 
X 


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Amendment 3 
 
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Screening 
Baseline 
Treatment 
Follow up 
Safety/Early 
Termination 
Visit15 
 
Visit 1 
Visit 2 
Visit 3 
Visit 4 
Visit 5 
Visit 6 
Visit 7 
Visit 8 
Visit 9 
Visit 10 
 
 
 
 
Week 3 
Week 4 
Week 8 
Week 
12 
Week 
16 
Week 
20 
Week 
24 
Week 
26 
4 weeks post last 
application of 
ATI-50002 
Treatment Day  
-14 to 0 
1 
21 
28 
56 
84 
112 
140 
168 
182 
 
Visit Windows 
N/A 
N/A 
+/- 3 
days 
+/-4 
days 
+7 days 
+/-7 
days 
+7 days 
+/-7 
days 
+/-7 
days 
+7 days 
+7 days 
Norwood-Hamilton 
classification(males); 
Sinclair Scale 
(females)Assessment 
X 
X 
 
 
 
 
X 
 
 
X 
X 
Target Area Hair 
Identification and 
Tattoo application7 
 
X 
 
 
 
 
 
 
 
 
 
Global Scalp 
Photograph 8 
X 
X8 
 
 
X9 
 
X9 
 
 
X9 
X 
Macro Scalp 
Photographs10 
 
X 
 
 
X 
 
X 
 
 
X 
 
Target Area Hair 
Count (TAHC) and 
Target Area Hair 
Width (TAHW)11 
 
X 
 
 
X 
 
X 
 
 
X 
 
Demonstration of 
study drug 
application12  
 
X 
 
X 
X 
X 
X 
X 
X 
X14 
 
Dispensing of study 
medication13 
 
X 
 
X 
X 
X 
X 
X 
X 
X14 
 


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Amendment 3 
 
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Screening 
Baseline 
Treatment 
Follow up 
Safety/Early 
Termination 
Visit15 
 
Visit 1 
Visit 2 
Visit 3 
Visit 4 
Visit 5 
Visit 6 
Visit 7 
Visit 8 
Visit 9 
Visit 10 
 
 
 
 
Week 3 
Week 4 
Week 8 
Week 
12 
Week 
16 
Week 
20 
Week 
24 
Week 
26 
4 weeks post last 
application of 
ATI-50002 
Treatment Day  
-14 to 0 
1 
21 
28 
56 
84 
112 
140 
168 
182 
 
Visit Windows 
N/A 
N/A 
+/- 3 
days 
+/-4 
days 
+7 days 
+/-7 
days 
+7 days 
+/-7 
days 
+/-7 
days 
+7 days 
+7 days 
Investigator 
Assessment of Hair 
Growth 
 
 
 
 
X 
 
X 
 
 
X 
 
Subject Self -
Assessment of Hair 
Growth 
 
 
 
 
X 
 
X 
 
 
X 
 
Local Skin 
Reaction 
Assessment 
 
X 
X 
X 
X 
X 
X 
X 
X 
X 
X 
Adverse Events14 
 
X 
Concomitant 
Therapies 
 
X 
1A written, signed ICF must be obtained from each subject prior to performing any study related procedure 
2 A physical exam includes: general appearance, examination of head, eyes, ears, nose and throat, respiratory, cardiovascular, abdominal, neurological, musculoskeletal, lymphatic 
and skin assessment. 
3 Vital signs include: oral or ear temperature, blood pressure, heart rate, respiration rate (height and weight at baseline only). 
4 Prior therapies or treatments a subject received to treat his/her AGA must be documented for each subject.   
5 Serum chemistry panel to include:  albumin, alk phos, ALT, AST, BUN, bicarbonate, calcium, chloride, creatinine, glucose, lactate dehydrogenase (LDH), phosphorus, 
potassium, sodium, total bilirubin, total protein, uric acid, total cholesterol, LDL, HDL triglycerides, CPK, and complete blood count including differential.    
6 All subjects must have a blood sample drawn to test for the tuberculosis virus.  The blood sample will be sent to a central laboratory for analysis and results must be received 
prior to enrolling the subject.   


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7 The investigational study staff will be required to identify target area of the scalp that is located in an actively balding area.  Directions for proper hair clipping are outlined in 
Section 9.2 .  Sites will be required to apply a permanent dot tattoo to the subject’s scalp to identify the target hair area.   
8Global scalp photographs are to be performed at the screening visit.  If the photos are not of the appropriate quality, the site staff will perform a re-shoot at Visit 2.   
9 Canfield staff will review all global photographs within 2 days of the photographs being submitted.  If a re-take is required, Canfield will notify the site and the re-take 
photographs will need to be performed within 7 days of the visit.    
10 Canfield staff will review all macro photographs within 2 days of the photographs being submitted.  If a re-take is required, Canfield will notify the site and the re-take 
photographs will need to be performed within 7 days of the visit. 
11Sites will take the macro photographs and transmit these to Canfield in order for the Canfield scientists to perform the Target Area Hair Counts and the Target Area Hair Widths.  
The TAHC and TAHW will be entered into the Canfield database.   
12Sites will be required to review with each subject the proper application of the study medication and remind the subjects that they are only to wash their hair with the same 
shampoo/conditioner and maintain the same hair style throughout the study.    
13Subjects will be dispensed bottles of study medication at each monthly visit.  Study medication will be weighed prior to dispensing the medication to the subject and upon return 
of both used and unused bottles.  
14 Subjects that are not remaining on protocol therapy for an additional 6 months of treatment will not have study medication dispensed at Visit 10.  
All non-serious AEs will be collected from the time of first study medication application until the final study visit.  Serious AEs will be collected from the time of consent through 
the final study visit.   
15 Subjects that consent to remain on study for an additional 6 months of treatment will continue on study with Visit 11 (week 30) and then will have a final follow up Visit 4 
weeks after the last application of study medication in the 6-month extension part of the study.  Those subjects that do not consent to continuing on study medication for an 
additional 6 months will be required to have a final safety follow up visit conducted at week 30.  For those subjects that discontinue the study early, they must return to the clinic 4 
weeks following the last study medication application for a final safety visit.   
Table 3: 
Schedule of Assessments – 6-month Extension Study 
 
 
 
Follow up 
Safety/Early 
Termination 
Visit10 
 
Visit 11 
Visit 12 
Visit 13 
Visit 14 
Visit 15 
Visit 16 
Visit 17 
Treatment week 
Week 30 
Week 35 
Week 39 
Week 44 
Week 48 
Week 52 
Week 56 
Visit Windows 
+7 days 
+7 days 
+7 days 
+7 days 
+/-7 days 
+7 days 
+7 days 
Physical Exam1 
 
 
 
 
 
 
X 
Vital Signs2 
X 
X 
X 
X 
X 
X 
X 
Clinical Chemistry 
and CBC3 
 
 
X 
X 
 
X 
X 


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Dihydrotestosterone 
(DHT), 
Testosterone, 
Estrogen, DHEA 
 
 
X 
 
 
X 
X 
Urine Pregnancy 
Test 
X 
 
X 
X 
X 
X 
X 
ECG 
 
 
X 
 
 
 
X 
Norwood-Hamilton 
classification(males); 
Sinclair Scale 
(females)Assessment 
 
 
 
 
 
X 
X 
Global Scalp 
Photograph  
 
 
X4 
 
 
X4 
X 
Macro Scalp 
Photographs5 
 
 
 
 
 
X5 
 
Target Area Hair 
Count (TAHC) and 
Target Area Hair 
Width (TAHW)6 
 
 
 
 
 
X 
 
Demonstration of 
study drug 
application7  
X 
X 
X 
X 
X 
X 
 
Dispensing of study 
medication8 
X 
X 
X 
X 
X 
X 
 
Investigator 
Assessment of Hair 
Growth 
 
 
X 
 
 
X 
X 
Subject Self -
Assessment of Hair 
Growth 
 
 
X 
 
 
X 
X 


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1 A physical exam includes: general appearance, examination of head, eyes, ears, nose and throat, respiratory, cardiovascular, abdominal, neurological, musculoskeletal, lymphatic 
and skin assessment. 
2 Vital signs include: oral or ear temperature, blood pressure, heart rate, respiration rate. 
3 Serum chemistry panel to include:  albumin, alk phos, ALT, AST, BUN, bicarbonate, calcium, chloride, creatinine, glucose, lactate dehydrogenase (LDH), phosphorus, 
potassium, sodium, total bilirubin, total protein, uric acid, total cholesterol, LDL, HDL triglycerides, CPK, and complete blood count including differential.    
4 Canfield staff will review all global photographs within 2 days of the photographs being submitted.  If a re-take is required, Canfield will notify the site and the 
re-take photographs will need to be performed within 7 days of the visit.    
5 Canfield staff will review all macro photographs within 2 days of the photographs being submitted.  If a re-take is required, Canfield will notify the site and the 
re-take photographs will need to be performed within 7 days of the visit 
6Sites will take the macro photographs and transmit these to Canfield in order for the Canfield scientists to perform the Target Area Hair Counts and the Target 
Area Hair Widths.  The TAHC and TAHW will be entered into the Canfield database.   
7Sites will be required to review with each subject the proper application of the study medication and remind the subjects that they are only to wash their hair 
with the same shampoo/conditioner and maintain the same hair style throughout the study.    
8Subjects will be dispensed bottles of study medication at each monthly visit.  Study medication will be weighed prior to dispensing the medication to the subject 
and upon return of both used and unused bottles.  
9 All non-serious AEs will be collected from the time of first study medication application until the final study visit.  Serious AEs will be collected from the time 
of consent through the final study visit.   
10A final safety follow up visit will be conducted at week 56 (Visit 17).  For those subjects that discontinue the study early, they must return to the clinic 4 weeks 
following the last study medication application for a final safety visit.   
Local Skin 
Reaction 
Assessment 
X 
X 
X 
X 
X 
X 
X 
Adverse Events9 
X 
Concomitant 
Therapies 
X 


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8. 
SELECTION AND WITHDRAWAL OF SUBJECTS 
8.1. 
Subject Inclusion Criteria 
Subjects must meet the following criteria to be eligible for participation in the study: 
1. Able to comprehend and willing to sign an Informed Consent Form (ICF). 
2. Male subjects and non-pregnant, non-nursing female subjects 18 to 50 years of age with a clinical 
diagnosis of AGA.    
3. Male subjects with a Norwood-Hamilton classified as III vertex, IV or V and woman subjects with a 
Sinclair Grade 2, 3 or 4. 
4. Subjects willing to agree to have small 1.9 cm diameter circle of hair clipped to ~ 1 mm in length on 
their balding scalp. 
5. Subjects willing to agree to have a permanent dot tattoo applied to their scalp to mark the center of 
the identified target area.   
6. Subjects must agree to maintain the same hair style and hair care regimen during the study.  
7. Subjects must agree to not use a medicated shampoo or conditioner containing biotin, saw palmetto or 
tea tree oil (melaleuca oil) throughout the course of protocol treatment and refrain from hair weaving, 
hair extensions, texturizers, relaxers, occlusive wigs and non-study hair growth products during the 
study and for a period of 30 days prior to Visit 2. 
8. Subjects must have an absolute neutrophil count (ANC) >1,000/mm3, and a platelet count >50,000/mL. 
9. Male subjects with a serum prostate-specific antigen (PSA) level of < 2.0 ng/mL. 
10. Subjects receiving oral or topical hormones must be on a stable dose for 6 months prior to Visit 2.   
11. Subjects must be willing to refrain from washing hair, using hair products, and participating in 
strenuous exercise that would cause profuse sweating for 6 hours after each treatment application 
with ATI-50002.   
12. Woman of child bearing potential (WOCBP) must agree to use an approved method of highly 
effective birth control for the duration of the study and for 30 days after last study medication 
application. 
13. Sexually active male subjects must agree to use a barrier form of birth control from the first 
application until 30 days post last application of ATI-50002. 
8.2. 
Subject Exclusion Criteria 
Subjects are excluded from this study if any 1 or more of the following criteria is met: 
1. Females who are nursing, pregnant, or planning to become pregnant for the duration of the study and 
up to 30 days after the last application of study medication 
2. Clinical diagnosis of alopecia areata or other non-AGA forms of alopecia. 
3. Scalp hair loss on the treatment area, due to disease, injury or medical therapy.   


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4. Active skin disease on the scalp (such as psoriasis or seborrheic dermatitis) or a history of skin disease 
on the scalp that in the opinion of the investigator would interfere with the study assessments of efficacy or 
safety. 
5. Evidence of hypogonadism defined as serum testosterone < 250ng/dl and/or LH>20mU/ml in males. 
6. Active unstable thyroid disease, including subjects on therapy for either hyperthyroidism or 
hypothyroidism unless their dose of thyroid medication has been stable for 3 months prior to V2 and serum 
TSH is normal.   
7. Subjects with non-pigmented hair. 
8. Subjects unable to comply with the following required washout periods: 
• 
Scalp promoting hair growth products (e.g. minoxidil, finasteride, dutasteride, saw 
palmetto) for 6 months prior to Visit 2. 
• 
Anti-androgenetic therapies (e.g. spironolactone, ketoconazole, flutamide, cyproterone 
acetate) for 6 months prior to Visit 2.   
• 
Hair transplantation or hair weaving within 6 months of Visit 2 
• 
Anabolic steroids for 6 weeks prior to Visit 2.  
• 
Oral or topical JAK inhibitors 3 months prior to Visit 2 
• 
Platelet rich plasma treatment within 6 months of Visit 2 
• 
Hair Laser, light or energy treatments to the target hair area within 3 months of Visit 2. 
• 
Systemic steroids for more than 14 consecutive days in the 2 months prior to Visit 2. 
• 
Chemotherapy or cytotoxic agents within 12 months of Visit 2. 
• 
No prior radiation to the scalp. 
9. History of, current or suspected systemic or cutaneous malignancy and /or lymphoproliferative disease, 
other than subjects with: a history of adequately treated and well healed and completely cleared non-
melanoma skin cancers (basal or squamous cell carcinoma) treated successfully with no evidence of disease.   
10. History of human immunodeficiency virus (HIV), hepatitis B or C.  
11. Current herpes zoster infection. 
12. Evidence of active, latent or inadequately treated Mycobacterium tuberculosis infection, or history of 
incompletely treated or untreated tuberculosis. Subjects who have successfully completed therapy for latent 
tuberculosis may participate.  
13. History of serious local infection (e.g., cellulitis, abscess) or systemic infection including but not limited 
to a history of treated infection (e.g., pneumonia, septicemia) within 3 months prior to Visit 1. Subjects on 
an antibiotic for a non-serious, acute local infection must complete the course prior to enrollment into the 
study. 
14. Participation in an investigational drug, device, or biologic trial in which administration of an 
investigational drug, device, or biologic within 30 days or 5 half-lives (whichever is longer) of Visit 2.  
Subjects who have participated in a study of an investigational drug, device or biologic agent for 
androgenetic alopecia within 1 year of screening will be eligible to participate only with individual 
permission from the Medical Monitor. 
15. Sensitivity to any of the ingredients in the study medications. 
16. Screening ECG findings of: 
• 
QTcF >450msec for males or >470msec for females (use of the ECG algorithm is acceptable for 
this purpose) 
• 
Heart rate < 45 or > 100 beats/minutes (inclusive) 


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• 
Rhythm disturbance other than sinus arrhythmia or ectopic supraventricular rhythm (ectopic atrial 
rhythm) 
• 
Conduction disturbance including PR >240msec, pre-excitation (delta wave and PR <120msec), 
second degree or higher AV block 
• 
Acute or chronic signs of ischemia  
• 
Left Bundle Branch Block 
• 
Prior myocardial infarction 
8.3. 
Subject Withdrawal Criteria 
Subjects will be informed that they are free to withdraw from the study at any time and for any reason. 
The investigator may remove a subject from the study if, in the investigator's opinion, it is not in the best 
interest of the subject to continue the study. Examples of other reasons subjects may be discontinued from 
the study are: a change in compliance with an inclusion or exclusion criteria, occurrence of AEs, 
occurrence of pregnancy, use of a prohibited therapy or subject is unwilling or refuses to continue with 
the protocol defined procedures, treatments and/or study visits and/or subject withdraws consent. 
Subjects will be permanently discontinued from the study in the event of any of the following: 
• 
Severe infection requiring parental antimicrobial therapy or hospitalization 
• 
Symptomatic herpes zoster 
• 
Malignancy – except for non-melanoma skin cancer (SCC or BCC) not in or near the treatment 
area 
• 
Anaphylactic or severe allergic reaction 
• 
WBC Count: < 1 x 109/L or second occurrence of < 2 x 109/L 
• 
ANC:< 0.5 x 109/L or second occurrence of < 1 x 109/L  
• 
Lymphocyte count: < 0.3 x 109/L or second occurrence of < 0.5 x 109/L  
  
• 
Platelet count:   
< 50 x 109/L or second event of < 75 x 109/L - in each case, value should be 
confirmed by retesting before treatment discontinuation 
• 
Hemoglobin:   
< 6.5 g/dL or second occurrence of < 8 g/dL - in each case, value should be 
confirmed by retesting before treatment discontinuation 
• 
AST or ALT: 
 
o > 5 x ULN persisting for 2 weeks of study medication interruption or second event of > 5 
x ULN 
o 3 x ULN with total bilirubin >2 x ULN or symptoms of hepatocellular  
injury (fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, 
rash and/ or eosinophilia (>5%).  
 
• 
Any subject who develops any of the following ECG findings during the active treatment phase 
will be instructed to stop study medication and will be withdrawn from the study: 
 
o Clinically significant rhythm disturbance other than sinus rhythm or ectopic 
supraventricular rhythm (ectopic atrial rhythm) 
o Clinically significant conduction disturbance including PR >240msec, pre-excitation (delta 
wave and PR <120msec), second degree or higher AV block  


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o New finding of QRS>120ms (if not present at screen.  For example, subjects with Right 
Bundle Branch Block at screening would not need to be withdrawn from the study if their 
subsequent ECGs remained unchanged). 
o Evidence of QT-interval prolongation, defined as an increase in the QTcF interval>60ms 
from Visit 1 
o Acute signs of ischemia or infarction 
o Any ECG abnormality which may, in the opinion of the investigator, represent a new 
medical issue of concern 
A subject that experiences any of the above events must be discontinued from study medication treatment, 
the site staff must inform the Aclaris medical monitor of the event, and the site staff must perform protocol-
required procedures for trial discontinuation and follow-up.    


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9. 
TREATMENT OF SUBJECTS 
9.1. 
Hair Care During Study 
Subjects enrolled to the study must agree to use the same shampoo/conditioner through the 
course of the protocol therapy.  This information will be documented in the source notes and 
captured in the subject’s CRF.  Shampoo/Conditioner must not contain biotin, saw palmetto or 
tea tree oil (melaleuca oil).  Study medication must be applied to a dry scalp.  Subjects should 
not wash their hair for 6 hours after application of study medication.   Subjects are required to 
maintain the same hair style while on study.   
9.2. 
Target Area Hair Identification  
In order to assess the efficacy of the study medication, a 1 cm2 area of the subject’s scalp will be 
identified and be used to count the number of hair follicles that appear in the area throughout the 
duration of the study treatment period.   
Using the Canfield supplied clear plastic template, a 1 cm2 area will be identified in an actively 
balding area.  The hair will be pulled through the circular area of the plastic template and then 
the hair will be clipped short using the supplied electric hair clippers.  After the hair is clipped, 
the template is to be removed and the hairs are to be further trimmed to approximately 1mm in 
length using 4- inch curved scissors.  Once the target area is identified and the hair is clipped 
then a small tattoo will be applied to mark the target area.  Refer to Section 9.4  regarding the 
procedure for the scalp tattoo.   This target area will be used for each TAHC/TAHW assessment 
throughout the study.   
 


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Figure 1: 
Target Area Identification  
9.3. 
Preparation of Subjects Scalp for Global and Macro Photographs 
Investigational sites are to perform the global photographs prior to performing the macro 
photographs.   
For all vertex global scalp photographs (male subjects only), the subject’s hair is to be combed 
outwards from the vertex like the spokes of a wheel.  For all frontal global scalp photographs 


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(male and female subjects), the subject’s hair is to be parted in the center and the hair is to be 
combed away from the center part.   
The global photographs will be used by the investigator to perform the Investigative Global 
Assessment (IGA) and by subject to perform the Subject Self-Assessment (SSA).  Refer to 
Section 11.3 . 
9.4. 
Scalp Tattoo 
At Visit 2 all subjects will be required to have a permanent dot tattoo applied to their scalp in an 
actively balding area.  The tattoo will mark the center of the identified target area from which the 
macro photographs and TAHC and TAHW will be performed.   
All sites will be supplied with a tattoo machine and the necessary ink to apply the tattoo.  The 
tattoo machines will be supplied by Canfield and sites will be provided with instructions on how 
to prepare the machine and the subject’s scalp to ensure that the tattoo is applied appropriately.   
9.5. 
Application of Study Medication  
 
Subjects will be instructed to apply a thin film of ATI-50002 Topical Solution 0.46% (up to 4-
mL), twice-daily; once in the morning and approximately 12 hours later to the balding area of the 
clean dry scalp.  Refer to Appendix 1  for the complete application instructions.   The subject 
must wash her/his hands thoroughly before and after each study drug application. Investigational 
site staff will dispense a month’s supply of study medication along with disposable droppers to 
the subject at each study visit.  Study medication bottles will be weighed by the site staff at the 
time of dispensing and when the bottles are returned.  Subjects are to return used and unused 
study medication at each study visit.  The disposable droppers should be disposed of at the 
subject’s home. 
At each study visit, the study staff will review the study medication instructions and proper 
application technique. (Appendix 1 ) with the subject.  During Visit 2, the subject will apply the 
first dose of study medication in the office under the instruction and supervision of the study 
staff.   
During each site visit, the study staff member will: 
• Dispense study medication bottles to the subject. 
• Instruct the subject on the appropriate application technique following instructions in 
Appendix 1. 
• Weigh the bottles prior to dispensing the study medication 
• Observe the subject’s first study medication application to ensure proper coverage and 
monitor the subject for at least 20 minutes (Visit 2 only).   
   
All used study medication bottles will be weighed by the investigational site at each visit.   


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9.6. 
Concomitant Medications 
Concomitant therapies are any new or existing therapy received from Visit 1 until discharge from 
the study. 
Concomitant therapies include drug (e.g., prescription, over-the-counter [OTC]) and non-drug 
(e.g., chiropractic, physical therapy, energy-based treatments) therapies. Subjects will refrain 
from receipt of any therapy in compliance with the inclusion/exclusion criteria. Subjects should 
refrain from changing the use of any concomitant therapies during the study. 
All new or modified concomitant therapies used during the study must be recorded in the 
subject’s eCRF. 
9.7. 
Prohibited Medications 
The following medications will be prohibited from being used while the subject is on study and 
for the identified time-frame prior to Visit 2: 
• 
Scalp hair growth promoting products (e.g.minoxidil, finasteride, dutasteride, saw palmetto) for 6 
months prior to Visit 2. 
• 
Anti-androgenetic therapies (e.g. spironolactone, flutamide, cyproterone acetate) for 6 months prior 
to Visit 2.   
• 
Hair transplantation or hair weaving within 6 months of Visit 2. 
• 
Semi-permanent hair products (e.g.  texturizers, relaxers) for 30 days prior to Visit 2.   
• 
Anabolic steroids for 6 weeks prior to Visit 2.  
• 
Oral and topical JAK inhibitors for 3 months prior to Visit 2. 
• 
Medicated shampoo or conditioner containing biotin, saw palmetto or tea tree oil (melaleuca oil) 
for 30 days prior to Visit 2. 
• 
Platelet rich plasma treatment within 6 months of Visit 2. 
• 
Hair Laser, light or energy treatments to the target hair area within 3 months of Visit 2. 
• 
Systemic steroids for more than 14 consecutive days in the 2 months prior to Visit 2 
• 
Chemotherapy, cytotoxic agents or within 12 months of Visit 2. 
• 
 Prior radiation to the scalp.  
9.8. 
Treatment Compliance 
The investigator or designee will be responsible for monitoring subject compliance through 
questioning the subject, documenting missed doses, if any, weighing the bottle before dispensing 
and after return and visual inspection of the quantity in the study medication bottles (used and 
unused).  Study staff will counsel the subjects, as required to make sure subjects are compliant 
with study medication applications.  
9.9. 
Study Medication Interruption 
Treatment with ATI-50002 Topical Solution should be temporarily interrupted in the event of 
severe adverse events considered related to ATI-50002, or in the event of one or more of the 
abnormal laboratory values in Table 4 . 


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Table 4: 
Study Medication Interruption Criteria 
Laboratory Test 
Hold Study Medication if: 
Resume Study Medication if: 
WBC count 
< 2 x 109/L 
≥ 2.5 x 109/L 
ANC 
< 1 x 109/L 
≥ 1.5 x 109/L 
Lymphocyte count < 0.5 x 109/L 
≥ 0.75 x 109/L  
Platelet count 
< 75 x 109/L 
≥ 100 x 109/L 
Hemoglobin 
< 8 g/dL or a decrease > 2g/dL 
≥ 10 g/dL 
AST or ALT 
> 3 x ULN 
< 2 x ULN or within 20% of Baseline values 
Serum creatinine 
>2 x ULN 
<1.5 x ULN or within 10% of Baseline value 
If a subject has one or more of the abnormal laboratory values noted in Table 4 , the investigator 
or designee upon receipt and review of the central laboratory report should instruct the subject to 
hold study medication applications. The investigator or designee should ask the subject about 
symptoms, concomitant illnesses and medications and repeat the test(s) as soon as possible.  The 
Medical Monitor must be notified of dose interruptions due to SAEs considered related to study 
medication or laboratory abnormalities noted in Table 4 . 
If the retest confirms the abnormal laboratory value, then the study medication should continue 
to be held followed by repeat testing once a week or sooner at the discretion of the investigator.  
The subject should be followed until the laboratory abnormality(s) return to normal or to baseline 
values. 
9.10. 
Randomization and Blinding 
This study is an open label study so therefore subject randomization does not apply for this 
clinical trial.   


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10. 
STUDY DRUG MATERIALS AND MANAGEMENT 
10.1. 
Study Drug 
The study medication for this study is ATI-50002 Topical Solution 0.46%.  It is a clear, colorless 
to light pink solution.  The inactive ingredients include: purified water, transcutol P, propylene 
glycol, PEG400, dimethyl sulfoxide (DMSO), kolliphor CS 20, benzyl alcohol, poloxamer 188, 
and povidone K30 
Table 5: 
Investigational Product 
STUDY MEDICATION INFORMATION 
Study medication name 
ATI-50002 
Dosage Strength 
0.46% 
Manufacturer 
PMRS, Inc., Horsham, PA 
Pharmaceutical Form 
Topical Solution 
Container 
Amber Glass Bottle, 120 mL with screw cap  
Storage Conditions 
59°F to 77°F (15°C to 25°C)  
Dose regimen 
Route 
Topical 
Frequency 
Twice-daily 
Duration of administration 
52 weeks 
Other supplies 
Disposable, single-use droppers will be provided. 
 
10.2. 
Study Drug Packaging, Labeling and Storage 
The study medication must be used by the study subjects only.  Investigational site staff will 
explain the application of the study medication to subjects. 
Study medication will be provided by Aclaris Therapeutics, Inc. and labeled according to 
regulations.  Study medications must be stored in a secure area with limited access under 
appropriately controlled and monitored storage conditions.  Study medication should be stored at 
controlled room temperature 59°F - 77°F (15°C – 25°C).  Subjects will be instructed to store the 
study medication in the original glass bottle (in the carton provided) at room temperature, away 
from heat, moisture, direct light, and to keep it from freezing and out of the reach of children. 
The study medication will be supplied in amber glass 120 mL bottles.  Disposable droppers with 
1 mL calibration mark will be provided to the investigational sites for dispensing to enrolled 
subjects.   


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Study drug for this study is AT50002 Topical Solution 0.46%.  The study medication will be 
supplied to subjects enrolled to the study on a monthly basis.  
10.3. 
Study Drug Accountability and Disposal 
The Principal Investigator or designee is responsible for ensuring accountability for the 
investigational agent, including reconciliation of medications and maintenance of medication 
records.  Upon receipt of study medication, the clinical site will check for accurate delivery and 
acknowledge receipt by signing (or initialing) and dating the documentation provided.  One copy 
of this document will be returned to Aclaris Therapeutics, Inc. (or designee) and one copy will be 
maintained in the study file at the site. In addition, an accurate study medication disposition 
record will be kept, specifying the amount dispensed for each subject and the date of dispensing. 
This inventory record will be available for inspection at any time. At the completion of the study, 
the original inventory record will be available for review by Aclaris Therapeutics, Inc. upon 
request.  Final medication accountability will be performed by the study monitor at the 
completion of the study and all used and unused study medication bottles will be returned to 
Aclaris Therapeutics, Inc. or designee for disposal. 
10.4. 
Additional Clinical Supplies 
In addition to the study medication supplies, each site will be supplied with the following clinical 
supplies to conduct the study: 
• Disposable dispensing droppers 
• Canon Rebel T6i 18MP D-SLR 
• Canfield IntelliFlash 
• Stereotactic Head Device 
• Dedicated laptop computer 
• Canfield Capture Software 
• Blue Drape Cloth 
• Canfield VEOS SLR (18 MP) 
• Canfield LED 
• Secure Digital (SD) Memory Card 
• Tattoo Machine and Ink 
• Hair Clippers 
• 4-inch curved scissors 


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11. 
ASSESSMENT OF EFFICACY 
11.1. 
Target Area Hair Count (TAHC) and Target Area Hair Width 
(TAHW) 
The primary efficacy endpoint of the study is the change from baseline in non-vellus (TAHC 
(hairs/cm2) and one of the secondary efficacy endpoints is the change from baseline in the 
cumulative TAHW.  The subject’s identified target area will be assessed for TAHC and TAHW 
by the Canfield trained and validated image analysis technicians who are blinded to the 
center/subject/visit/treatment information.  Canfield Scientific, Inc. will supply all investigational 
sites with photography equipment, tattoo equipment, computer based scanners imaging software, 
and printers required to capture all global and macro photographs.  
As described in Section 8.4, all subjects will be required to have a small permanent tattoo applied 
to their scalp to mark the center of the identified target area.  Global and macrophotographs will 
be taken by the investigational study staff in accordance with the schedule of assessments.  Refer 
to Table 2 and Table 3 .     
The Canfield supplied VEOS SLR camera will be used to take the macrophotographs of the 
subject’s scalp.  These images will be transmitted to Canfield for analysis using a computer 
assisted technique to assess the TAHC and TAHW.   
Study staff will receive training from Canfield on the use of the photography equipment, 
scanners and imaging software in order to transmit all imaging data to Canfield.   
11.2. 
Norwood-Hamilton and Sinclair Scales  
Treating investigators will be required to rate the subject’s level of hair loss at baseline based on 
the Norwood Hamilton Scale for male subjects and the Sinclair scale for women.  In addition, at 
the protocol defined time-points, the Investigator will be required to use the appropriate scale to 
assess the change in hair growth in both male and female subjects.   
11.3. 
Investigator Global Assessment and Subject Self-Assessment 
Global photographs will be taken of the vertex scalp of male subjects and of the frontal scalp of 
both male and female subjects.  These global scalp photographs will be used by the investigator 
and the subject to rate the subject’s amount of hair growth at each protocol defined time-point.   
After the global photographs are taken the investigator and the subject will use the current photos 
to rate the change in hair growth from the baseline visit (Visit 2). Both the investigator and the 
subject will record their assessment into the Canfield computer system.  The following 7-point 
rating scale will be used: 
Table 6: 
7-Point Rating Scale of Subjects Scalp Hair Based on Evaluation of Global 
Photographs 
Grade 
Description 
-3 
Greatly decreased hair growth 
-2 
Moderately decreased hair growth 


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Grade 
Description 
-1 
Slightly decreased hair growth 
0 
No change 
+1 
Slightly increased hair growth 
+2 
Moderately increased hair growth 
+3 
Greatly increased hair growth 
 


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12. 
ASSESSMENT OF SAFETY 
12.1. 
Safety Parameters 
Safety will be assessed throughout the study by the investigator or a designated and appropriately 
trained staff member. 
12.1.1. 
Demographic/Medical History 
During the screening visit (Visit 1), the investigator or designee will interview each subject to 
obtain demographic information including date of birth, sex at birth, Fitzpatrick skin type, race 
and, if appropriate, ethnicity. The investigator or designee will interview each subject to obtain 
medical history information related to all medical conditions, surgeries and disease states that, at 
screening: are ongoing, require concomitant therapy or are, in the opinion of the investigator, 
relevant to the subject’s study participation.  In addition, the medical history of women who are 
not of childbearing potential should reflect the reason e.g. post-menopausal for 1 year or greater, 
total hysterectomy.  Information regarding the subject’s prior treatments or therapies related to 
AGA must be documented along with the type of shampoo/conditioner the subject will use 
during the study.   
12.1.2. 
Vital Signs 
Vital signs will be measured at each visit during the study. The following items will be 
measured: 
• Body temperature 
• Pulse rate 
• Respiration rate 
• Blood pressure (systolic and diastolic) after the subject sits quietly for at least 5 minutes 
• Height (at Visit 2 only) 
• Weight (at Visit 2 only) 
Any measure that is, in the opinion of the investigator, abnormal AND clinically significant (CS) 
must be recorded as history if found prior to the first study medication application, or as an AE if 
found after the first study medication application begins. 
12.1.3. 
Physical Examination 
The investigator or designee will perform a physical examination for all body systems (general 
appearance, examination of head, eyes, ears, nose and throat, respiratory, cardiovascular, 
abdominal, neurological, musculoskeletal, lymphatic and skin assessment) at Baseline (Visit 2) 
and end of study Week 30 (Visit 11) 
12.1.4. 
Electrocardiogram (ECG) 
Standard 12-lead ECGs will be performed by a qualified staff member at Screening (Visit 1), 
Visit 4 (Day 28) Visit 10 (Day 182) and Visit 11/Safety Follow up.  The ECGs must be obtained 


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using a standard 12-lead ECG with a 10mm/mV amplitude, at 25mm/sec and a 5-10-second 
duration. To ensure a steady heart rate the subject must rest quietly in the supine position for at 
least 5 minutes prior to performing the ECG. 
A central cardiology laboratory will provide ECG equipment, supplies and site training.  In 
addition, the central cardiology laboratory will process ECGs received by the sites and report 
results via a secure study portal.  The ECG results will be interpreted by a qualified health 
professional (evaluator) and the interpretation reported either directly on the tracing or in a 
separate report.   The evaluator will interpret the results of every ECG and define the ECG as 
“normal” or “abnormal”.  Variations such as minor ST changes (i.e., <0.5mm depression) and 
early re-polarization are considered normal. 
The investigator must review the evaluator’s interpretation of each subject’s screening ECG prior 
to Baseline (Visit 2). The investigator will review the evaluator’s interpretation of all ECG 
reports in a timely manner and comment on the clinical relevance of any result that is defined by 
the evaluator as abnormal. 
Any abnormalities that are, in the opinion of the investigator, clinically significant, must be 
reported as history if found prior to the start of the first study medication application or as an AE 
if found after the start of the first study medication application.   
If a subject’s ECG results meets the ECG withdrawal criteria that is outlined in section 7.3, the 
site staff must discontinue the subject from study medication and perform all protocol required 
assessments at the early termination/follow up visit.   
12.1.5. 
Pregnancy Tests 
Subjects who are WOCBP must have a negative serum pregnancy test result at Screening (Visit 
1) to continue in the study, and a negative UPT at Baseline (Visit 2) prior to study entry.  If the 
result of any post-treatment urine pregnancy test is positive, the subject will be withdrawn from 
the study and the subject’s pregnancy documented and followed.  In addition, the investigator or 
designee will perform a urine pregnancy test for subjects who are WOCBP at monthly visits 
during the study. 
12.1.6. 
Local Skin Reactions 
Investigators and subjects will be required to assess the treated scalp area for local skin reactions 
(LSRs) at visits 2-11.  The following signs and symptoms of contact dermatitis will be assessed: 
• Stinging/Burning 
• Itching 
• Dryness 
• Erythema 
• Scaling 
The Investigator and Subject should report the one integer that best describes the severity of each LSR 
sign or LSR symptom for the treated scalp area using the scale below. 
 


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Local Skin Reactions 
Grade 
Descriptor 
0 
None 
1 
Mild 
2 
Moderate 
3 
Severe 
If a local skin reaction requires treatment or causes an interruption or discontinuation of 
study medication then the LSR needs to be recorded and assessed as an adverse event.   
12.1.7. 
Laboratory Assessments 
12.1.7.1. Clinical Chemistry/Hematology  
All subjects are required to have a blood chemistry panel performed at Visit 1 (Screening), Visit 
5 (Day 56), Visit 7 (Day 112), Visit 9 (Day 168), Visit 10 (Day 182) and Visit 11 (Day 210) 
/Safety Follow up Visit.  A qualified staff member will collect non-fasting blood samples and 
ship the samples to a central laboratory for analysis.  The following tests will be performed: 
Chemistry Panel 
Complete Blood Count 
Albumin 
 
Hematocrit 
Alkaline phosphatase 
Hemoglobin 
Alanine aminotransferase (ALT) 
Platelet count 
Aspartate aminotransferase (AST) 
Red blood cell morphology 
Blood urea nitrogen (BUN) 
Red blood cell count 
Bicarbonate 
CPK 
White blood cell count 
Calcium 
White blood cell differential 
Chloride 
    % & absolute 
Creatinine 
    Basophils 
Glucose 
    Eosinophils 
Lactate dehydrogenase (LDH) 
    Lymphocytes 
Phosphorus 
    Monocytes 
Potassium 
    Neutrophils 
Sodium 
 
Total bilirubin 
Total protein 
 


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Uric Acid 
Total cholesterol, LDL, HDL 
 
Triglycerides 
 
Total Iron Binding Capacity (TIBC) (Visit 1 only) 
Serum iron (Visit 1 only) 
Serum Ferritin (Visit 1 only) 
Testosterone 
Estrogen 
DHEA 
Dihydrotesterone (DHT) 
Serum PSA (males only; Visit 1 only) 
Lutenizing Hormone (LH) (Visit 1 only) 
Thyroid Panel (Visit 1 only) (including TSH, Total T4, 
Total T3, Free T4, Free T3, Reverse T3 (rT3), T3U, 
Free T4 Index, and TPO and AntiTG antibodies) 
 
 
 
12.1.7.2. Virus Serology 
All subjects will be required to have a QuantiFERON TB blood test performed at screening to 
assess for exposure to the tuberculosis virus.  At Visit 1 (Screening) a blood sample will be 
drawn and sent to a central laboratory for analysis.  Subjects that test positive will not be eligible 
for enrollment to the study. 
12.2. 
Adverse and Serious Adverse Events 
12.2.1. 
Definition of Adverse Events 
12.2.1.1. Adverse Event (AE) 
An AE is the development of an undesirable medical condition or the deterioration of a pre-
existing medical condition following or during exposure to a pharmaceutical product, whether or 
not considered casually related to the product.  In clinical studies, an AE can include an 
undesirable medical condition occurring at any time, including baseline or washout periods, even 
if no study treatment has been administered. 
The investigator should, when certain, report a diagnosis rather than the signs, symptoms or 
clinically significant abnormal laboratory values associated with the AE. Otherwise, signs, 
symptoms or abnormal laboratory values may be used to describe the AE. 


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Any CS abnormality discovered prior to the first study medication treatment should be reported 
as medical history, not as an AE. 
All AEs that occur from the time of first study medication application (V2) until 30 days after 
last study medication application must be documented in the subject’s source documents and in 
the Aclaris’ electronic data capture system.   
12.2.1.2. Serious Adverse Event (SAE) 
A serious adverse event is an AE occurring during any study phase (ie, baseline, treatment, 
washout, or follow-up), and at any dose of the investigational product, comparator or placebo, 
that fulfils one or more of the following: 
• Results in death 
• It is immediately life-threatening 
• It requires in-patient hospitalization or prolongation of existing hospitalization 
• It results in persistent or significant disability or incapacity 
• Results in a congenital abnormality or birth defect 
• It is an important medical event that may jeopardize the patient or may require medical 
intervention to prevent one of the outcomes listed above. 
The term “life threatening” refers to an event in which the subject was at risk of death at the time 
of event; it does not refer to an event that hypothetically might have caused death if it were more 
severe. 
Important medical events are those that may not be immediately life threatening, result in death 
or hospitalization, but are clearly of major clinical significance and may jeopardize the subject or 
require intervention to prevent one of the outcomes listed in the SAE definition above. These 
should also usually be considered serious. 
SAEs will be reported from the time of informed consent until 30 days past last application of 
study medication, whether or not they are related to the study.  SAEs will be documented on the 
Aclaris SAE report form and in the eCRF.    
12.2.1.3. Unexpected adverse event 
An AE is considered unexpected if it is not listed in the Investigator Brochure or is not listed at 
the specificity or severity that has been observed. 
12.3. 
Recording Adverse Events 
12.3.1. 
Adverse event reporting period 
The investigator must start reporting non-serious AEs with the subject’s first study medication 
application and continue reporting until the end of the subject’s last study visit. Reporting for 
SAEs must start when the subject signs the ICF and continue until the end of the subject’s last 
visit. 


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12.3.2. 
Severity 
The investigator is to define the severity of each AE using the following definitions as a 
guideline. The investigator will consider the range of the possible severity of the event and 
identify the severity that is the most appropriate according to her/his medical judgment. 
Mild – Awareness of signs or symptom, but easily tolerated. 
Moderate – Discomfort, enough to cause interference with usual activity. 
Severe – Incapacitating with inability to perform usual activity. 
12.3.3. 
Relationship to study medication 
The investigator will determine if there is a reasonable causal relationship between the study 
medication and an AE or not. The investigator will use her/his best medical judgment and 
consider all relevant factors (e.g., temporal relationship, location of the event, the subject’s 
relevant medical history, concomitant therapies and concurrent conditions) to determine the 
relationship of the AE to the study medication. The investigator will define the relationship of an 
AE to the study medication by selecting one of the following categories: 
Related – There is a reasonable causal relationship between the study medication and the 
AE. 
Not Related – There is not a reasonable causal relationship between the study medication 
and the AE. 
The expression “reasonable causal relationship” is meant to convey in general that there are facts 
(evidence) or arguments to suggest a causal relationship (International Conference on 
Harmonization [ICH] E2A). 
12.3.4. 
Procedures for reporting adverse events 
At each post-enrollment visit, the investigator will question the subject to elicit AEs using a non-
directive question such as “Has there been any change in your health since the previous study 
visit?”  If appropriate, based on the subject’s response to non-directed questioning to elicit AEs, 
the investigator will follow-up with directed questions and appropriate evaluations. 
Any AE noted during the reporting period must be reported in the source documents and on the 
appropriate AE eCRF. AEs that are defined as “Not Related” to the study medications will be 
followed until they are resolved or until the subject’s last study visit. AEs that are defined as 
“Related” to the study medications will be followed until they are resolved or, if not resolved at 
the subject’s last study visit, until in the opinion of the investigator, the AE reaches a clinically 
stable outcome with or without sequelae. 
Should a pregnancy occur, it must be reported and recorded on Aclaris’s pregnancy form.  
Pregnancy in itself is not regarded as an AE unless there is a suspicion that an investigational 
product may have interfered with the effectiveness of a contraceptive medication. 
The outcome of all pregnancies (spontaneous miscarriage, elective termination, normal birth or 
congenital abnormality) must be followed up and documented even if the patient was 
discontinued from the study. 


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All reports of congenital abnormalities/birth defects are SAEs.  Spontaneous miscarriages should 
also be reported and handled as SAEs.  Elective abortions without complications should not be 
handled as AEs. 
12.3.5. 
Procedure for reporting a serious adverse event 
Upon becoming aware of a SAE occurring during the SAE reporting period (time of subject 
consent until the end of the subject’s last visit), whether or not related to the study medications, 
the investigator must: 
1. Take the appropriate medical action to ensure the subject’s safety. 
2. Immediately inform the Safety Monitor of the SAE by email, ensuring that the subject 
information is deidentified (only subject initials and subject number) to: ProPharma, 
Email: clinicalsafety@propharmagroup.com. 
3. Print a copy of the email confirmation from ProPharma and place in the study file. 
4. Within 24-hours complete, as fully as possible, an AE eCRF and an SAE form; e-mail the 
forms and any other relevant information (e.g., concomitant medication eCRF, medical 
history eCRF, laboratory test results) to ProPharma (Aclaris Therapeutics, Inc. Safety 
Monitor). 
5. Monitor and document the progress of the SAE until it resolves or, if not resolved after 
the subject’s last study visit, until in the opinion of the investigator the AE reaches a 
clinically stable outcome with or without sequelae AND the investigator and Aclaris 
Therapeutics, Inc.  Safety and Medical Monitor agree that the SAE is satisfactorily 
resolved. 
6. Inform the Aclaris Therapeutics, Inc. Safety Monitor of SAE updates, via telephone, 
followed by an SAE form update sent by e-mail. 
Comply with the appropriate regulatory requirements and Aclaris Therapeutics, Inc. instructions 
regarding reporting of the SAE to the responsible Institutional Review Board (IRB) or Ethics 
Committee (EC). 
12.4. 
Pregnancy 
12.4.1. 
Definition of Women of Child Bearing Potential (WOCBP) 
WOCBP includes any female who has experienced menarche and who has not undergone 
successful surgical sterilization (e.g., hysterectomy, bilateral tubal ligation, or bilateral 
oophorectomy) or is not postmenopausal. Postmenopausal is defined as ≥12 months with no 
menses without an alternative medical cause.  WOCBP must have a negative serum pregnancy 
test at screening and a negative UPT at baseline prior to randomization. 
12.4.2. 
Highly Effective Methods of Birth Control 
The Investigator or sub-investigator will discuss the potential risk factors associated with 
pregnancy and the importance of maintaining a highly effective method of contraception 
throughout the study with all WOCBP (for example, those which result in a low failure rate - i.e., 
less than 1% per year- when used consistently and correctly).  All WOCBP must use a highly 


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effective method of birth control during the study and for 30 days after the final application of 
study medication in a manner such that risk of failure is minimized.   
Highly effective methods include: 
• Combined (estrogen and progestogen containing) hormonal contraception associated with 
inhibition of ovulation: 
o oral 
o intravaginal 
o transdermal 
• Progestogen-only hormonal contraception associated with inhibition of ovulation: 
o oral 
o injectable 
o implantable 
• intrauterine device (IUD) 
• intrauterine hormone-releasing system (IUS) 
• vasectomized partner1 
• sexual abstinence2 
1Vasectomized partner is a highly effective birth control method provided that partner is the sole sexual partner 
of the WOCBP trial participant and that the vasectomized partner has received medical assessment of the 
surgical success.  
2 Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual 
intercourse during the entire period of risk associated with the study treatments. The reliability of sexual 
abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual 
lifestyle of the subject. 
 
WOCBP must be on a highly effective method of birth control for the following timeframes prior 
to study entry:  
• Implants (on a stable dose for ≥90 days) 
• Injectables (on a stable dose for ≥90 days) 
• Patches (on a stable dose for ≥90 days) 
• Combined oral contraceptives (on a stable dose for ≥90 days) 
• Intrauterine devices (inserted for ≥30 days). 
 
Prior to trial enrollment, WOCBP must be advised of the importance of avoiding pregnancy 
during trial participation and of the potential risk factors associated with pregnancy while in the 
study.  The subject must sign an informed consent form documenting this discussion.  During the 
trial, all WOCBP will be instructed to contact the investigator immediately if they suspect they 
might be pregnant (e.g., missed or late menstrual period). 
 


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If a subject or investigator suspects that the subject may be pregnant prior to study medication 
administration, the study medication must be withheld until the results of a pregnancy test are 
available.  If pregnancy is confirmed, the subject must not receive study medication and must be 
discharged from the study.  
 
If, following study medication administration, it is determined that the subject may have been or 
was pregnant at the time of study medication exposure (including at least 2 days after study 
medication administration) the investigator must immediately notify the Aclaris Therapeutics, 
Inc. Medical Monitor and record the event on a pregnancy surveillance form. While not an AE or 
SAE, the investigator must report every pregnancy using a pregnancy surveillance form and 
follow the reporting procedures described for SAE reporting. 
Protocol-required procedures for trial discontinuation and follow-up must be performed on the 
subject unless contraindicated by pregnancy (e.g., x-ray studies). Other appropriate pregnancy 
follow-up procedures should be considered if indicated. In addition, the investigator must report 
to Aclaris Therapeutics, Inc.’s Medical Monitor on the pregnancy surveillance form, follow-up 
information regarding the course of the pregnancy, including perinatal and neonatal outcome. 
Infants should be followed for a minimum of six weeks. 
 
12.5. 
Protocol Deviation and Protocol Violations 
A protocol deviation is defined as an accidental or unintentional change to, or non-compliance 
with the research protocol that does not increase risk, or decrease benefit or; does not have a 
significant effect on the subject’s right, safety, or welfare; and/or on the integrity of the data.  
Deviations may result from the action of the subject, researcher or research staff.  A protocol 
deviation does not involve inclusion/exclusion criteria. 
A protocol violation is defined as an accidental or unintentional change to, or non-compliance 
with the IRB approved protocol without prior sponsor approval.  Protocol violations generally 
increase risk or decrease benefit, affects the subject’s rights, safety, or welfare, or the integrity of 
the data.   
 


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13. 
STATISTICS 
13.1. 
Statistical Methods 
Summary descriptive statistics (N, mean, median, SD) by visit will be provided for all safety and 
efficacy parameters. The Intent to Treat population will be used for all efficacy assessments and 
the Safety population will be used for all safety assessments. No data imputation will be used.  
Primary and secondary efficacy parameters are described in the sections below. 
13.2. 
Primary Efficacy Endpoint 
Mean change from baseline in non-vellus TAHC will be quantified using a microphotographic 
technique and calculated along with the median, standard deviation, standard error, and 95% 
confidence limits around each mean change to allow tests of the null hypothesis that the 
population mean changes are zero. 
13.3. 
Secondary Efficacy Endpoints 
Mean change from baseline in cumulative TAHW will be quantified using a microphotographic 
technique and calculated along with the median, standard deviation, standard error, and 95% 
confidence limits around each mean change to allow tests of the null hypothesis that the 
population mean changes are zero. 
The seven-point rating scale to measure the investigator and subject assessment of the 
development of new hair growth/scalp coverage will be analyzed with summary statistics (N, 
mean, median, SD) for each visit where the assessment is made.  Similar summary statistics will 
be used to analyze change from baseline in AGA stage separately for males (Horwood Hamilton 
Scale) and for females (Sinclair Scale) at each applicable visit. 
13.4. 
Safety Assessments 
Data from all enrolled and treated subjects will be presented and summarized. Safety analyses 
will include descriptive statistics calculated on the safety parameters using the safety population. 
The proportion of subjects with treatment-emergent adverse events will be tabulated and 
presented by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class. 
Vital signs and clinically significant abnormal laboratory results will also be tabulated and 
presented.  Clinical laboratory and ECG results will be tabulated. 


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14. 
QUALITY CONTROL AND QUALITY ASSURANCE 
The study is conducted under the sponsorship of Aclaris Therapeutics, Inc. in compliance with 
the applicable regulatory requirements as well as applicable ICH guidelines, Declaration of 
Helsinki, and in respect of the Aclaris Therapeutics, Inc. and/or sub-contractor SOPs for study 
conduct and monitoring. 
Audits may be carried out by Aclaris Therapeutics, Inc. or Aclaris Therapeutics, Inc.’s 
representatives, and inspections may be performed by regulatory authorities or IRB/ECs before, 
during or after the study.  The investigator will provide the auditing/inspecting group direct 
access to all study records (e.g., eCRFs, subject medical records, study medication dispensing 
records) and the investigational center study facilities. The investigator and study staff will be 
available and will assist the auditing/inspecting groups as appropriate. 


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15. 
ETHICS AND GENERAL STUDY CONDUCT 
CONSIDERATIONS 
15.1. 
Institutional Review Board (IRB)/Ethics Committee (EC) 
The final study protocol, including the final version of the Informed Consent Form, must be 
approved or given a favorable opinion in writing by an IRB or IEC as appropriate.  The 
investigator must submit the written approval to Aclaris Therapeutics, Inc. before he or she can 
enroll any patient/subject into the study. 
The Principal Investigator is responsible for informing the IRB or EC of any amendment to the 
protocol in accordance with local requirements.  In addition, the IRB or EC must approve all 
advertising used to recruit patients for the study.  The protocol must be re-approved by the IRB 
or EC upon receipt of amendments and annually, as local regulations require. 
The Principal Investigator is also responsible for providing the IRB with reports of any 
reportable serious adverse drug reactions from any other study conducted with the investigational 
product.  Aclaris Therapeutics, Inc. will provide this information to the Principal Investigator. 
Progress reports and notifications of serious adverse drug reactions will be provided to the IRB 
or EC according to local regulations and guidelines. 
15.2. 
Ethical Conduct of the Study 
The rights, safety and well-being of the subjects are the most important considerations in this 
study and take priority over the interests of society and science. 
This study will be conducted in accordance with the ethical principles originating from the 
Declaration of Helsinki, the ICH E6 GCP guideline, local regulatory requirements and, at US 
investigational centers, in compliance with the HIPAA. The study will be conducted in 
compliance with the IRB/EC approved version of the protocol and any applicable amendments. 
15.3. 
Written Informed Consent 
The Principal Investigator(s) at each center will ensure that the patient is given full and adequate 
oral and written information about the nature, purpose, possible risk and benefit of the study.  
Patients must also be notified that they are free to discontinue from the study at any time.  The 
patient should be given the opportunity to ask questions and allowed time to consider the 
information provided. 
The patient’s signed and dated informed consent must be obtained before conducting any study 
procedures. 
The Principal Investigator(s) must maintain the original, signed Informed Consent Form.  A copy 
of the signed Informed Consent Form must be given to the patient. 
15.4. 
Study Conduct and Protocol Amendments 
With the exception of eliminating an immediate hazard to a subject, the investigator should not 
deviate from the protocol or implement any changes without prior written approval from the 


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Aclaris Therapeutics, Inc.’s representative or designee and prior review and documented 
approval from the IRB/EC. 
Changes that involve only logistical or administrative changes are allowed.  The investigator 
should document and explain any deviation from the protocol.  A protocol deviation is a non-
adherence to protocol-specific study procedures or schedules that does not increase the risk to a 
study subject and does not affect the scientific integrity of the study. 
A protocol violation is defined as any divergence from the protocol-specific inclusion/exclusion 
criteria or study procedures or schedules that may result in an increased risk to a study subject or 
that affect the scientific integrity of the study.  All protocol violations must be reported to the 
IRB by the Investigator, as directed by the IRB-specific procedures. 
15.5. 
Regulatory Documents 
The investigator must maintain a study file containing current and complete regulatory 
documentation in compliance with the current ICH E6 GCP guideline. This file will be reviewed 
as part of the routine monitoring for this study. 
15.6. 
Contractual Requirements 
A contractual agreement will be signed between Aclaris Therapeutics, Inc. and each investigator.  
This document will contain supplemental information, including financial terms, confidentiality, 
study schedule, third party responsibility, and publication rights. 


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16. 
DATA HANDLING AND RECORDKEEPING 
16.1. 
Data Collection 
The Investigator must maintain required records for all study subjects.  Data for this study will 
be recorded in the subject's source document and on the eCRFs.  All data on these eCRFs should 
be recorded completely and promptly.  A copy of the completed eCRFs for each subject will be 
retained by the investigational center. 
Records of the subject’s participation in this study will be held confidential except as disclosure 
is required by law. The study doctor, the sponsor, persons working on behalf of the sponsor, and 
under certain circumstances, the United States Food and Drug Administration and the 
Institutional Review Board will be able to inspect and copy confidential study-related records 
that identify subjects by name. Therefore, absolute subject confidentiality cannot be guaranteed. 
If the results of this study are published or presented at meetings, the subject’s identity will not 
be revealed. 
16.2. 
Study Monitoring 
Before an investigational site can enter a patient into the study, a representative of Aclaris 
Therapeutics will visit the investigational study site to: 
• Determine the adequacy of the facilities. 
• Discuss with the investigator(s) and other personnel their responsibilities with regard to 
protocol adherence, and the responsibilities of Aclaris Therapeutics, Inc. or its 
representatives.  This will be documented in a Clinical Study Agreement between Aclaris 
Therapeutics, Inc. and the investigator. 
During the study, a monitor from Aclaris Therapeutics, Inc. or representative will have regular 
contacts with the investigational site, for the following: 
• Provide information and support to the investigator(s). 
• Confirm that facilities remain acceptable. 
• Confirm that the investigational team is adhering to the protocol, that data are being 
accurately recorded in the case report forms, and that investigational product 
accountability checks are being performed. 
• Perform source data verification.  This includes a comparison of the data in the case 
report forms with the patient’s medical records at the hospital or practice, and other 
records relevant to the study.  This will require direct access to all original records for 
each patient (e.g. clinic charts). 
• Record and report any protocol deviations not previously sent to Aclaris Therapeutics, 
Inc. 
• Confirm AEs and SAEs have been properly documented on CRFs and confirm any SAEs 
have been forwarded to ProPharma and those SAEs that met criteria for reporting have 
been forwarded to the IRB. 


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The monitor will be available between visits if the investigator(s) or other staff needs 
information or advice. 
16.3. 
Source Documentation 
Investigators must keep accurate separate records (other than the eCRFs) of all subjects' visits 
that include all pertinent study related information.  A statement should be made indicating that 
the subjects have been enrolled in this clinical study and have provided written informed 
consent.  Any AEs must be completely documented. Source documentation includes results of 
any diagnostic tests conducted during the study. 
16.4. 
Inspection of Records 
Aclaris will be allowed to conduct site visits to the investigation facilities for the purpose of 
monitoring any aspect of the study.  The Investigator agrees to allow the monitor to inspect the 
drug storage area, study drug stocks, drug accountability records, subject charts and study source 
documents, and other records relative to study conduct. 
16.5. 
Retention of Records 
The Principal Investigator must maintain all documentation relating to the study for a period of 2 
years after the last marketing application approval, or if not approved 2 years following the 
discontinuance of the test article for investigation.  If it becomes necessary for Aclaris or the 
Regulatory Authority to review any documentation relating to the study, the Investigator must 
permit access to such records. 
 


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17. 
REFERENCES 
1. Dinh, Q and Sinclair R. "Female pattern hair loss:  Current treatment concepts".  Clinical 
Interventions in Aging 2007: 2(2) 189-199. 
2. Gupta Mrinal and Mysore Venkataram. " Classifications of Patterened Hair Loss:  A 
Review." Journal Cutaneous Aesthetic Surgery. 2016. Jan-Mar; 9(1): 3-12.  
3. Olsen E, et al. "Evaluation and treatment of male and female pattern hair loss." Journal 
American Academy Dermatology.  2005: 52: 301-311. 
4. Yu, M.et al. "Interluki-6 cytokine family member oncostatin M is a hair follicle 
expressed factor with hair growth inhibitory properties." Exp. Dermatology, 2008.  17 
(1): 12-90. 


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18. 
APPENDICES 


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APPENDIX 1. SUBJECT INSTRUCTIONS FOR STUDY MEDICATION 
APPLICATION TO THE SCALP 
Preparation and General Instructions:  
 
1. Gather a clean washcloth and towel, the study medication bottle, and disposable dropper. 
2. Scalp should be clean (free of hair styling products), and dry or at least towel-dried 
before applying study medication.  Ensure that your scalp is as dry as possible.  A clean 
scalp will allow the study medication to penetrate down into the scalp to ensure you are 
getting the best application.   
3. Subjects are to wash their hair using a non-medicated shampoo [not containing biotin, 
saw palmetto or tea tree oil (melaleuca oil)] during the treatment period (26 weeks).  
4. Subjects are not to wash their hair or participate in strenuous exercise that would cause 
profuse sweating for 6 hours after application of study medication.       
5. Wash your hands with soap and water before and after using this study medication.  
6. You will apply a thin layer of study medication to the identified area of the scalp as 
instructed by the study doctor or the study staff.  Keep applying study medication to the 
area of hair loss throughout the study, even if scalp hair is re-growing in these areas. 
7. The amount of study medication you will apply during one treatment is a total of up 
to 4ml (1 ml droppers supplied with study medication).   
8. Avoid study medication contact with the eye.  If the study medication gets on any part of 
your body other than your scalp, rinse the area well with water.  
9. You will apply study medication twice-a-day, approximately 12 hours apart.   
10. Remember to bring your study medication bottles both used and unused to each study 
visit. 
 
Study Medication Application: 
1. Draw up exactly 1 mL of study medication into the dropper. The medication level should 
be at the 1mL line.  
2. During study medication application, avoid any study medication running into your eyes.   
3. Squeeze a few drops of study medication onto the center of the identified area of your 
scalp and gently rub the study medication into your scalp. Keep applying a few drops and 
rubbing into your scalp until the entire area is covered with a thin film of study 
medication.    
4. Draw up additional medication - 1 mL at a time- to cover the entire area on your scalp 
with hair loss.   
5. Replace the screw top cap and make sure it is closed tightly.  Dispose of the used 
dropper(s). 
6. It is important to continue to apply study medication to the areas of hair loss throughout 
the study, even if there is hair growth in the area(s).   


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7. Wash your hands after using this product to prevent any residue being left on your hands. 
8. Allow the study medication to dry for at least 15 minutes before you apply any styling 
products to the hair.  All topical products applied to the hair and scalp must be reviewed 
and approved by your study doctor before use. 
9. If you missed a dose or doses, tell the study staff at your next visit.     
10. Do not wash your hair or scalp for at least 6 hours after applying study medication.  
Missed Doses: If you miss a dose of this study medicine, apply it as soon as possible. However, 
if it is almost time for your next dose, skip the missed dose, and go back to your regular dosing 
schedule. 
Storage: Store the medicine in the original glass bottle in the carton provided at room 
temperature, away from heat, moisture, and direct light. Keep from freezing.  Keep out of the 
reach of children. 
 
 


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APPENDIX 2. NORWOOD HAMILTON SCALE 
 
 
 
(Gupta M and Mysor V, 2016) 
Type I:  Minimal or no recession of the hairline 
Type II:  Triangular, usually symmetrical, areas of recession at the frontotemporal hairline 
Type III:  Deep symmetrical recession at the temples that are bare or only sparsely covered by 
hair.  In Type III vertex, the hair loss is primarily from the vertex with limited recession of the 
frontotemporal hairline that does not exceed the degree of recession seen in Type III. 
Type IV:  Frontotemporal recession is more severe than in Type III and there is sparse hair or no 
hair on the vertex.  The two areas of hair loss are separated by a band of moderately dense hair 
that extends across the top.  The is band connects with the fully haired fringe on the sides of the 
scalp. 


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Type V:  The vertex hair loss region is still separated from the frontotemporal region but it is less 
distinct.  The band of hair across the crown is narrower and sparser and the vertex and 
frontotemporal regions of hair loss are bigger. 
Type VI:  The bridge of hair that crosses the crown is gone with only sparse hair remaining.  The 
frontotemporal and vertex regions are joined together and the extent of hair loss is greater.   
Type VII:  The most severe form of hair loss and only a narrow band of hair in a horseshoe shape 
remains on the sides and back of the scalp.  This hair is usually not dense and may be quite fine.   
 
 


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APPENDIX 3. SINCLAIR SCALE 
 
 
(Dinh Q and Sinclair R, 2007) 
Grade 1: is normal. This pattern is found in all girls prior to puberty but in only forty-five percent of 
women aged eighty or over. 
Grade 2: shows a widening of the central part. 
Grade 3: shows a widening of the central part and thinning of the hair on either side of the central part. 
Grade 4: reveals the emergence of a diffuse hair loss over the top of the scalp. 
Grade 5: indicates advanced hair loss. 
 
  
 
 


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APPENDIX 4. GLOBAL PHOTOGRAPHY PROCEDURE 
I. 
Purpose: 
 
The purpose of these procedures is to ensure that reproducible, registered, serial global 
photographs are taken of each subject’s vertex scalp throughout the study period.  These 
photographs will document hair growth or loss of the vertex scalp and consistency of these 
photographs permits quality subjective evaluations. 
 
Subjects must not be prepared for macro haircount photography until global photography has 
been conducted. 
 
II. 
Photographic locations: 
 
Global photograph of the vertex scalp (male only) 
Global photograph of the frontal scalp (male and female) 
 
III. 
Equipment and Supplies: 
 
• 
Canon Rebel T6i 18MP D-SLR 
• 
Canon 60mm lens 
• 
Canfield IntelliFlash 
• 
Stereotactic Head Device 
• 
Dedicated laptop computer 
• 
Canfield Capture Software 
• 
Blue Drape cloth 
 
All supplied photographic equipment will remain the property of Canfield. 
 
IV. 
Global Photography Schedule Overview: 
 
• 
Visit 1 (Screening) 
• 
Baseline reshoot (Visit 2) (if applicable) 


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• 
Visit 5 (Day 56) 
• 
Visit 7 (Day 112) 
• 
Visit 10 (Day 182) 
 
V. 
Image Capture Procedures: 
 
1. The supplied equipment is to be used exclusively for this study. Modifications, 
adjustments or repairs of the camera equipment are not to be undertaken without the 
expressed instruction of Canfield Scientific, Inc. 
 
2. Prior to capturing the subject photographs, Site Photographer launches Canfield 
Capture software by selecting the icon from the laptop desktop.   
 
3. Site Photographer logs in using unique Username/Password and selects study 
protocol. 
 
4. Site Photographer either creates a New Subject for an initial visit or, for a return 
subject, highlights the appropriate existing Subject ID listed in the Canfield Capture 
database.  The visit name (as per Section IV above) is selected by Site Photographer 
and the photo date is captured by the software. 
 
5. Site Photographer follows the application software prompts to make the capture set. 
The capture set consists of the vertex view of the head/scalp. 
 
6. Vertex View Hair Preparation: The hair has to be clean, dry, and combed out radially 
from the vertex like the spokes of a wheel. The blue drape cloth has to be placed over 
the subject’s shoulders to mask the shirt and collar color.   
 
7. For each photograph captured in the Screening capture sequence, the photographer 
matches orientation to the reference diagrams in Canfield Capture while utilizing the 
live-preview available.  At the Follow-Up sessions, the live-preview with image 
overlay/ghosting helps maintain photo consistency i.e. magnification and head 
positioning relative to the camera.  
 
VI. 
Image Transfer Procedure: 
1. After each capture, Site Photographer is prompted to review the photograph for 
acceptability.  The Site Photographer either accepts the photograph and moves on to 


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the next capture or does not accept it and recaptures.  
2. Following the session, the Photographer submits the photographs to Canfield. Upon 
exiting, the software automatically reads, checksums, encrypts, packages, and 
duplicates the data to submit to Canfield via internet or removable media submission.   
3. Modes of image transfer: 
• 
Internet:  A secure, validated, compliant web server set up at Canfield is used for 
secure transfer of study photographs by study sites.  Photographs are to be 
transferred the day of capture.   
• 
Removable Media:  a dedicated flash media drive (connected to the PC’s USB 
port).  The Photographer removes the flash drive containing the study 
photographs from the PC, and ships it to Canfield via courier on the same day the 
photographs are acquired. 
 
Canfield Capture logs a record of this action to a local database and prompts the Site 
Photographer when completed.  
4. Trained Canfield staff review the photographs with the associated data for technical 
quality and acceptability and communicate comments and issues to the site. 
 
5. At the end of the study, a copy of site specific subject photographs with all associated 
data is provided to each Study Site.  This copy is in addition to the Photography 
Result Report forms made available on the Canfield Clinical Services website on a 
rolling basis for printing after each session.  Remote access to all photographs by the 
Sponsor and CRO (if applicable) is also provided.   
 
6. Canfield provides each Study Site with the necessary hardware as well as technical 
support as needed.  All supplied photographic equipment remains the property of 
Canfield.  Any questions regarding the photographic portion of this study are to be 
forwarded to the assigned Project Manager at Canfield Scientific. 
 
VII. 
Electronic Assessments  
1. An Investigator Global Assessment (IGA) and Subject Self-Assessment (SSA) web based 
application is made available for viewing on the supplied laptop.  Approved Screening 
photographs are presented alongside Follow-Up photographs for real-time assessments 
immediately following Follow-Up photographic capture.  
2. Canfield is responsible for programming and installing the application on the laptop. The 
study specific work instruction outlines the randomization and layout for the assessment 
application.  
3. Canfield electronically captures the assessment scores. The decode listing specifying 
photograph position is provided and details described in the Data Transfer Specification 
document. 
 
 


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Questions, issues and comments regarding the photographic portion of this protocol are directed to the 
assigned Project Management Team at: 
 
Canfield Scientific, Inc. 
4 Wood Hollow Road 
Parsippany, NJ  07054 
Telephone: 973-434-1200 
 


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APPENDIX 5. MACRO HAIRCOUNT PHOTOGRAPHY AND IMAGE 
ANALYSIS PROCEDURE 
Purpose: 
 
The purpose of these procedures is to ensure that reproducible, registered, serial macro 
haircount photographs are taken of each subject throughout the study period. These 
photographs document hair growth or loss through quantitative image analysis (Target Area 
Haircount [TAHC] and, Target Area Hair Width. The quality of these photographs is essential in 
order to allow for accurate image analysis. 
 
Subjects must not be prepared for macro haircount photography until global hair photography 
has been conducted. 
 
I. 
Photographic locations: 
 
• 
Subject ID 
• 
Macro haircount photograph of the clipped target area 
 
II. Equipment and Supplies: 
 
• 
Canfield VEOS SLR (18 MP) 
• 
Canfield LED 
• 
Secure Digital (SD) Memory Card 
 
 
III. 
Macro Photography Schedule Overview 
• 
Visit 2 (Baseline)  
• 
Visit 5 (Day 56) 
• 
Visit 7 (Day 112) 
• 
Visit 10 (Day 182) 
 
IV. 
Target Area Preparation/Image Capture Procedures: 
 
(Full details of hair clipping and macrophotography procedures are provided to the Study 
Sites in a Canfield Photography Manual prior to study site initiation) 


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1. The supplied equipment is to be used exclusively for this study. Modifications, 
adjustments or repairs of the camera equipment are not to be undertaken without 
the expressed instruction of Canfield Scientific, Inc. 
 
2. Magnification/Aperture: The lens and camera is fixed at a predetermined 
reproduction ratio and F-Stop.  
 
3. A circular haircount target area approximately 1.9 cm in diameter is identified in the 
actively balding area (detailed information and diagrams provided in the Canfield 
Photo Manual). Care is taken to ensure that the target area is a representative area 
of active hair loss. The target area is clipped short (approximately one [1] mm in 
length).  A small dot tattoo is placed in the center of the target area and is used as a 
permanent reference point. The same circular haircount target area is clipped and 
photographed at subsequent study visits. If the ink intensity of the tattoo has faded 
severely at any subsequent visit, the tattoo is to be reapplied prior to conducting of 
macrophotography. 
 
4. Only one subject session captured on each SD card.  Additional photographs are 
encouraged if there is any doubt as to the correctness or quality of the photographic 
technique.  
 
5. Two exposures of subject’s ID card include the following legible information in black 
indelible ink: 
 
• 
Protocol No. 
• 
Date 
• 
Investigator Number 
• 
Visit Day 
• 
Subject's Initials 
• 
Subject’s ID number 
• 
Photographer’s Initials 
 
6. A minimum of 2 exposures of the target haircount area is captured.  
 


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V.       Image Transfer Procedure: 
 
1. Upon completion of the photographic session, the contents of the SD memory card 
are uploaded to the Canfield Clinical Services Website. A secure, validated, 
compliant web server set up at Canfield is used for this secure transfer of study 
photographs by the study site. Photographs are transferred the day recorded. 
Remote access to all photographs by the sponsor and CRO (if applicable) is also 
provided.  Only approved individuals by Sponsor have access to the website. 
 
2. After the Site Photographer confirms that the photographs have been successfully 
uploaded to Canfield, all photographic files are deleted on the memory card by 
opening Windows Explorer and navigating to the card directory 
 
• 
Select all files by clicking on Edit /Select All 
• 
Delete the photographs by clicking on File / Delete 
3. After clearing the card of all files, the memory card is 
removed from the card slot and stored for re-use. 
 
 
VI.  Image Archiving and Monitoring: 
 
1. Once the photographs are received at Canfield from the study site, the photographs 
are copied to a dedicated, secure archival server. 
2. Trained Canfield staff review the photographs with the associated data for technical 
quality and acceptability and communicate comments and issues to the site. 
 
3. At the end of the study, a copy of site specific subject photographs with all 
associated data is provided to each Study Site.  This copy is in addition to the 
Photography Result Report forms made available on the Canfield Clinical Services 
website on a rolling basis for printing after each session.  Remote access to all 
photographs by the Sponsor and CRO (if applicable) is also provided.   
 
4. Canfield provides each Study Site with the necessary hardware as well as technical 
support as needed.  All supplied photographic equipment remains the property of 
Canfield.  Any questions regarding the photographic portion of this study are to be 
forwarded to the assigned Project Manager at Canfield Scientific. 
 
VII.  Image Analysis Procedures: 


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On the digital images, a circular 1-cm2 target area is identified using the landmark dot 
tattoo placed in the center of the circle of clipped hairs as a reference point.  From the 
identified 1- cm2 target area, the width of each hair is determined using an electronic 
caliber measurement.  All hairs >30 µm are classified as terminal hairs.  
 
Hair image analysis is randomized and blinded; subject identity and photographic 
sequence is unknown to the Canfield technicians conducting the image analysis. 
 
Parameters derived from hair measurement analysis include: 
 
• 
Target Area Haircount (TAHC) 
• 
Target Area Hair Width (TAHW) 
 
Questions, issues and comments regarding the photographic portion of this protocol are 
directed to the assigned Project Management Team at: 
 
Canfield Scientific, Inc. 
4 Wood Hollow Road 
Parsippany, NJ  07054 
Telephone: 973-434-1200