A Multicenter, Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata
Phase: PHASE3
Conditions: Alopecia Areata
Sponsor: Sun Pharmaceutical Industries, Inc.

Official title: A Multicenter, Open-Label, Extension Study to Assess the Long-Term Safety and 
Efficacy of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata   
Document: Study Protocol 
NCT number: NCT05041803 
Document date: 13 Nov 2023 

CTP-543 
 
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MEDICAL MONITOR / EMERGENCY CONTACT INFORMATION 
 

CTP-543 
 
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CONFIDENTIAL & PROPRIETARY 
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SUN PHARMACEUTICAL INDUSTRIES, INC. SIGNATURE PAGE 
PROTOCOL TITLE:  A Multicenter, Open-Label, Extension Study to Assess the Long-Term 
Safety and Efficacy of CTP-543 in Adult Patients with Moderate to Severe Alopecia Areata 
Study Number:  CP543.5002
5.2 
 

CTP-543
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CTP-543
13 November 2023
Protocol CP543.5002 
 
 Sun Pharmaceutical Industries, Inc. 
CONFIDENTIAL & PROPRIETARY

CTP-543
13 November 2023
Protocol CP543.5002 
 
 Sun Pharmaceutical Industries, Inc. 
CONFIDENTIAL & PROPRIETARY 
7 

CTP-543
13 November 2023
Protocol CP543.5002 
 
 Sun Pharmaceutical Industries, Inc. 
CONFIDENTIAL & PROPRIETARY

CTP-543
13 November 2023
Protocol CP543.5002 
 
 Sun Pharmaceutical Industries, Inc. 
CONFIDENTIAL & PROPRIETARY

CTP-543 
 
13 November 2023 
Protocol CP543.5002 
 
Sun Pharmaceutical Industries, Inc. 
CONFIDENTIAL & PROPRIETARY 
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Table 2: 
 Schedule of Events – Responders (Visit 10 – 16)  
Event
Visit 10 (Week 60)1 
Visit 11 (Week 68) 
Visit 12 (Week 76) 
Visit 13 (Week 84) 
Visit 14 (Week 92) 
Visit 15 (Week 100) 
 
Home Urine 
Pregnancy Testing
Week 56 
Week 64 
Week 72 
Week 80 
Week 88 
Week 96
Week 104
4-Week Post Final 
Study visit 
 
Visit 16 (Week 108; 
Final Study Visit) 
Early Termination 
Visit
 
Informed consent
 
 
 
Eligibility assessment
 
 
 
Complete physical examination 
X 
Brief physical examination 
X 
 
 
Pregnancy test2  
X 
X3 
X 
Clinical laboratory testing4 5 
X 
 
X 
12-lead electrocardiogram 
 
 
X 
Severity of Alopecia Tool assessment
X 
 
X 
Dispense study drug 
X 
 
 
Adverse events 
Continuous 
Concomitant medications 
Continuous 
1Visit window is ± 3 days for Visits 10-1 .
2Pregnancy testing will be performed every 4-weeks throughout study enrollment.  As in-clinic visits are 
performed bi-monthly, subjects will perform in-home urine pregnancy tests at Week 
104 and 4-weeks post the final study visit.  The test date and result will be documented. 
3In-home pregnancy testing window is ± 3 days 
4Collected pre-dose, when possible. 
5Includes fasted hematology, serum chemistry, SARS-CoV-2 testing, and lipids performed by the central lab. 

CTP-543 
 
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2. 
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF 
FIGURES  
MEDICAL MONITOR / EMERGENCY CONTACT INFORMATION ........................................ 2
SUN PHARMACEUTICAL INDUSTRIES, INC. SIGNATURE PAGE........................................ 3
SITE PRINCIPAL INVESTIGATOR’S AGREEMENT .................................................................. 4
1.
SYNOPSIS ...................................................................................................................... 10
2.
TABLE OF CONTENTS, LIST OF TABLES, AND LIST OF FIGURES ...............
3.
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ............................. 20
4.
INTRODUCTION .......................................................................................................... 21
4.1.
Overview of Alopecia Areata ........................................................................................ 21
4.2.
Rationale for the Study ................................................................................................... 21
4.3.
Nonclinical Information for CTP-543 ........................................................................... 22
4.4.
Clinical Information for CTP-543 ................................................................................. 22
5.
ETHICS ........................................................................................................................... 25
5.1.
Ethics Committee (EC) .................................................................................................. 25
5.2.
Written Informed Consent.............................................................................................. 25
STUDY OBJECTIVES .................................................................................................. 
Study Design and Method of Treatment Assignment .................................................. 
7.
SELECTION AND WITHDRAWAL OF PATIENTS................................................ 
7.1.
Patient Inclusion Criteria ................................................................................................ 
7.2.
Patient Exclusion Criteria............................................................................................... 
7.3.
Patient Withdrawal Criteria............................................................................................ 
7.3.1. 
Patient Withdrawal Procedures ...................................................................................... 30
7.4.
Criteria for Study Termination....................................................................................... 30
7.4.1. 
End of Trial ..................................................................................................................... 30
DESCRIPTION OF STUDY TREATMENTS............................................................. 32
Safety Criteria and Management for Stopping Doses .................................................. 32
 
Management of Risk with Janus Kinase (JAK) Inhibitors........................................... 33
 
SARS-CoV-2 Dose Interruption Guidance ................................................................... 33
Treatment Compliance ................................................................................................... 34
Study Drug Materials and Management........................................................................ 34
 
Physical Description of Study Drug .............................................................................. 34
 
Study Drug Packaging, Labeling, and Storage ............................................................. 34
 
Study Drug Preparation and Administration ................................................................. 34

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Study Drug Return and Disposal ................................................................................... 35
Study Drug Accountability ............................................................................................35
Concomitant Medications and Other Requirements for Continued Eligibility .......... 35
 
Continued Eligibility Beyond Initial 52-Weeks of Treatment ..................................... 
STUDY ASSESSMENTS AND PROCEDURES ....................................................... 37
Physical Examination ..................................................................................................... 37
2.
Clinical Laboratory Assessments ..................................................................................37
Electrocardiogram........................................................................................................... 
Severity of Alopecia Tool (SALT) ................................................................................ 
Unscheduled Visit ........................................................................................................... 
10.
ADVERSE EVENTS ..................................................................................................... 40
10.1.
Definition of Adverse Event .......................................................................................... 40
10.2.
Evaluation of Adverse Events ........................................................................................ 40
10.2.1.
Serious Adverse Event ................................................................................................... 40
10.2.2.
Severity/Intensity ............................................................................................................ 42
10.2.3.
Relationship to Study Drug ............................................................................................ 42
10.2.4.
Duration ........................................................................................................................... 43
10.2.5.
Action Taken ................................................................................................................... 43
Outcome .......................................................................................................................... 43
10.3.
Follow-Up ....................................................................................................................... 43
10.4.
Pregnancy ........................................................................................................................ 43
10.5.
Recording Adverse Events ............................................................................................. 44
Reporting Adverse Events.............................................................................................. 44
Reporting Serious Adverse Events ................................................................................ 44
Reporting Urgent Safety Issues ..................................................................................... 45
11.
STATISTICAL METHODS .......................................................................................... 
11.1.
Endpoints ......................................................................................................................... 
11.1.1.
Safety ............................................................................................................................... 
11.1.2.
Efficacy ........................................................................................................................... 
11.2.
Analyses .......................................................................................................................... 
11.2.1.
Disposition and Baseline Characteristics ...................................................................... 
11.2.2.
Study Drug Exposure ..................................................................................................... 47
11.2.3.
Safety ............................................................................................................................... 47
11.2.3.1.
Adverse Events ............................................................................................................... 47

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11.2.3.2.
Clinical Laboratory ......................................................................................................... 
11.2.3.3.
Vital Signs.......................................................................................................................
11.2.3.4.
Electrocardiogram........................................................................................................... 
12.
REGULATORY CONSIDERATIONS ........................................................................ 
12.1.
Good Clinical Practice.................................................................................................... 
12.2.
Sponsor’s Responsibilities ............................................................................................. 
12.3.
Investigator’s Responsibilities.......................................................................................50
12.4.
Protocol Amendments .................................................................................................... 51
12.5.
Audits and Inspections ................................................................................................... 51
Quality Control and Quality Assurance ........................................................................ 52
13.
DATA HANDLING AND RECORDKEEPING ......................................................... 53
13.1.
Confidentiality ................................................................................................................ 53
13.2.
Patient Data Protection ................................................................................................... 53
13.3.
Data Collection ............................................................................................................... 53
13.4.
Case Report Form Completion ...................................................................................... 54
13.5.
Database Management, Data Clarification, and Quality Assurance ........................... 54
Inspection of Records ..................................................................................................... 54
13.7.
Retention of Records ...................................................................................................... 55
14.
PUBLICATION POLICY .............................................................................................. 
15.
LIST OF REFERENCES ............................................................................................... 57
APPENDICES ................................................................................................................ 
Severity of Alopecia Tool (SALT) ................................................................................ 
 

CTP-543 
 
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LIST OF TABLES
Table 1: 
Schedule of Events – (All Enrolled Subjects (Visit 1 – 
 ........................................... 14
Table 2:
Schedule of Events – Responders (Visit 10 – 
.......................................................15
Table 3: 
Abbreviations and Specialist Terms .............................................................................. 20
Table 4: 
Clinical Trials with CTP-543 in Patients with Moderate to Severe Alopecia 
Areata .............................................................................................................................. 23
Table 5: 
Selected Hematologic Thresholds for Dose Interruption ............................................. 32
Table 
 
SARS-CoV-2 Dose Interruption Criteria ...................................................................... 34
Table 7:
Clinical Laboratory Assessments ..................................................................................

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3.
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS
Table 3: 
Abbreviations and Specialist Terms  
Abbreviation or Specialist Term 
Explanation 
AA 
Alopecia Areata 
BID 
Twice daily dosing 
CFR
Code of Federal Regulations
CTCAE 
Common terminology criteria for adverse events 
CYP3A4 
Cytochrome P450 3A4 
DMC
Data monitoring committee
DMP
Data Management Plan
EC 
Ethics Committee 
eCRF 
Electronic case report form 
FDA 
Food and Drug Administration 
GCP 
Good Clinical Practice 
HIV 
Human Immunodeficiency Virus 
ICF 
Informed consent form 
ICH 
International Council for Harmonisation 
IWRS 
Interactive Web Response System 
JAK 
Janus kinase 
LLN 
Lower Limit of Normal 
MDRD 
Modification of Diet in Renal Disease 
MedDRA 
Medical Dictionary for Regulatory Activities 
NDA 
New Drug Application 
PCS
Potentially clinically significant
QD 
Once daily dosing 
QTc 
QT interval corrected for heart rate 
QTcF 
QT interval corrected for heart rate (Fridericia’s method) 
SALT 
Severity of Alopecia Tool 
STAT 
Signal transducers and activators of transcription 
TB 
Tuberculosis 
TEAE 
Treatment-emergent adverse events 
ULN 
Upper Limit of Normal 
VAS 
Visual Analog Scale  

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4.
INTRODUCTION
4.1.
Overview of Alopecia Areata
Alopecia areata (AA) is an autoimmune disorder characterized by patches of non-scarring 
alopecia affecting scalp or scalp and body hair.  Alopecia areata is clinically heterogeneous, 
affects men and women, and has a prevalence rate of approximately 0.2% of the United States 
population (Safavi, 
Benigno, 2020).  There is no preventative therapy or cure.  Alopecia 
areata often presents as a cyclical disorder marked by unpredictable periods of hair loss and 
spontaneous regrowth, and variation in the degree or pattern of hair loss gives rise to different 
subtypes of alopecia areata, such as patchy, ophiasis, totalis, or universalis.  Approximately 7% 
of patients have severe disease with almost complete hair loss and little or no regrowth 
(Villasante Fricke, 2015).  Onset can occur at any age and affects both men and women over the 
course of their lifetime.  
episode of hair loss by 40 years of age and 40% by 20 years of age (Villasante Fricke, 2015).  
Alopecia areata can have a psychological impact with high rates of depression (Sellami, 2014) 
and anxiety reported, particularly in children and adolescents (Bilgic, 2013), and therefore, 
psychological counseling is often recommended as part of the standard of care (Al-
Mutairi, 2011). 
A cause for alopecia areata has not yet been identified, though as in other autoimmune disorders, 
genetic susceptibility and a wide array of environmental triggers are thought to be involved.  
Presently, treatments for alopecia areata include moderately effective intralesional corticosteroid 
injections or topicals, or aesthetic disguises such as makeup and wigs.  Baricitinib (Olumiant®), 
a JAK kinase inhibitor, was recently approved by the US Food and Drug Administration (FDA) 
and the European Commission for treatment of adults with severe AA, demonstrating the utility 
of this class of molecule for alopecia areata.  
4.2. 
Rationale for the Study 
Recent advances in the understanding of the pathogenesis of AA have shown Janus kinase (JAK) 
inhibitors to be a promising novel therapy.   
The JAKs are intracellular tyrosine kinases that play a central role in the signaling of cytokine 
and growth factor receptors (Ghoreschi, 
).  Cytokine-induced receptor conformation 
-member signal transducers and 
activators of transcription (STAT) protein transcription factor family.  Upon phosphorylation, 
STATs dimerize and translocate to the nucleus to regulate gene transcription.  Therapies that 
inhibit cytokine signaling or downstream JAK signaling have demonstrated efficacy in 
autoimmune disorders such as psoriasis, psoriatic arthritis, and rheumatoid arthritis, and multiple 
JAK inhibitors are in development for autoimmune disorders such as atopic dermatitis, systemic 
lupus erythematosus and others (Levy, 2015).  
shown to be central in AA, causing up-regulation of interleukin-15 in hair follicles and 
ultimately production of interferon-gamma, which targets the hair follicle for attack (Xing, 
2014).  As downstream regulators of interferon-gamma and interleukin-15, JAK inhibitors have 
been shown to eliminate the interferon signature and reverse disease.  
In in vitro kinase inhibition assays, the target potency and selectivity profile of CTP-543 for the 
JAK kinases JAK1, JAK2, JAK3 and Tyk2 was determined.  CTP-543 was found to have potent 
and selective inhibitory activity for intracellular JAK1 and JAK2 (IC50 (nM) = 4.7 and 20, 
respectively) signaling.   

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CTP-543 has been studied in patients with moderate to severe alopecia areata and at least 2 
different doses have demonstrated significant hair regrowth over a 24-week treatment period.  A 
Phase 2 dose-ranging clinical trial assessing the efficacy and safety of CTP-543 in moderate to 
severe AA patients was completed.  The overall objectives of the study were to assess the safety 
and efficacy of a 24-
-543 compared 
to matching placebo in adult patients with moderate to severe alopecia areata.  The primary 
efficacy endpoint was the proportion of responders, defined as patients achieving at least a 50% 
relative reduction in SALT score, from Baseline at Week 24.  The primary efficacy endpoint was 
BID group and the placebo group was not statistically significant.  
BID doses also produced significant results vs placebo when other analyses of SALT data were 
conducted as well as analyses of patient and clinician reported global impression.   
In the pivotal phase 3, randomized, double-blind, placebo-controlled study CP543.3001 
(THRIVE-AA1) evaluating the efficacy and safety of CTP-543 in adult subjects with moderate 
to severe AA, both CTP-
 mg BID and CTP-543 12 mg BID for 24 weeks resulted in a 
placebo) who achieved an 
be clinically meaningful to patients and physicians (Wyrwich et al., 2020).  CTP-543 was 
generally well tolerated at both doses, with the safety profile consistent with that of other drugs 
of the same class.  
Similarly, in the second pivotal phase 3, randomized, double-blind, placebo-controlled study 
CP543.3002 (THRIVE-AA2) evaluating the efficacy and safety of CTP-543 in adult subjects 
with moderate to severe AA, both CTP-
 mg BID and CTP-543 12 mg BID for 24 weeks 
 
Therefore, this current study will assess the long-term safety of CTP-543 in patients with 
alopecia areata while continuing to evaluate the long-term effects on hair-regrowth, in patients 
who complete 24 weeks of treatment in the preceding study.  In light of the known safety risks of 
the JAK class (see Section 
), patients who are non-responders after 52 weeks will be 
discontinued from the study.  However, those patients who are responding with hair re-growth 
can continue to receive treatment, while being monitored from a benefit-risk perspective.  Refer 
to the latest CTP-543 Investigator’s Brochure for full details of the efficacy and safety data with 
CTP-543. 
4.3. 
Nonclinical Information for CTP-543 
A series of nonclinical studies were performed with CTP-543, which evaluated the in vitro 
pharmacokinetic (PK) and metabolism properties, safety pharmacology, toxicity/toxicokinetics, 
and genotoxicity.  All safety pharmacology and toxicity studies considered necessary for human 
safety assessment were conducted in compliance with Good Laboratory Practice regulations.  
Further details of the nonclinical studies for CTP-543 are described in the respective 
Investigator’s Brochure. 
4.4. 
Clinical Information for CTP-543 
To-date, nine clinical studies with CTP-543 in healthy volunteers have been completed.   

CTP-543 
 
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Study No. and Title 
Phase 
Country 
No. of Subjects 
Dosing Regimen 
Status 
Germany, 
Hungary) 
CP543.5001: A Multicenter, 
Open-Label, Extension Study to 
Assess the Long-Term Safety 
and Efficacy of CTP-543 in 
Adult Patients with Moderate to 
Severe Alopecia Areata 
2/3 
United States 
Canada 
Approximately 
1,100  
CTP-543 dosed orally 
mg BID or 12 mg 
BID
 for up to 
weeks 
 
Ongoing 
eligible patients from Study CP543.2004 were eligible to enroll in this study following 24 weeks of treatment.

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5.
ETHICS
The procedures set out in this study protocol, pertaining to the conduct, evaluation and 
documentation of this study, are designed to ensure that the Sponsor and the Investigator abide 
by Good Clinical Practice (GCP), including but not limited to Title 21 Code of Federal 
Regulations (CFR) Par
the Declaration of Helsinki, International Council 
for Harmonisation Guidelines, and local regulations governing the conduct of clinical studies.   
The Investigator is responsible for protecting the rights, safety, and welfare of patients under 
his/her care, and for the control of the medications under investigation.  All ethical, regulatory, 
and legal requirements must be met before the first patient is enrolled in the study. 
5.1. 
Ethics Committee (EC) 
The Ethics Committee (EC) will meet all regulatory authority requirements governing ECs 
according to 
 
). The study protocol and any 
amendments, the Sponsor’s approved informed consent form(s) (ICF), patient information 
sheets, patient recruitment materials, and other appropriate documents will be submitted to the 
EC for review and approval. Following review of the submitted materials a copy of the written 
and dated approval/favorable opinion will be forwarded to the Sponsor (or designee).  
5.2. 
Written Informed Consent 
Prior to performing any study-related activities under this protocol, written informed consent 
with the approved ICF must be obtained from the patient.  
The ICF, as specified by the clinical site’s EC, must follow the Protection of Human Subjects 
regulations listed per local regulations. The background of the proposed study, the procedures, 
the benefits and risks of the study, and that study participation is voluntary must be explained to 
the patient.  The patient must be given sufficient time to consider whether to participate in the 
study.  
A copy of the ICF, signed and dated by the patient, must be given to the patient.  Confirmation of 
a patient’s informed consent must also be documented in the patient’s source documentation 
prior to any testing under this protocol.  The original signed consent form will be retained with 
the study records. 
All ICFs used in this study must be approved by the appropriate EC and by the Sponsor. The ICF 
must not be altered without the prior agreement of the relevant EC and the Sponsor. 

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-weeks post the final study visit.  For responders, additional home 
-weeks post the 
final study visit. The test date and result will be documented in a patient diary and brought to 
each in-clinic study visit by the patient.  SARS-CoV-2 testing will be performed at all visits.  
Significant cytopenias or other hematologic abnormalities will be managed by severity through 
dose interruption or discontinuation, and signs and symptoms of infection will be closely 
monitored and treated promptly (Section 
).  Investigators should also monitor patients for 
possible signs, symptoms, or exposure to tuberculosis, including any travel to high-risk 
countries.  In cases where TB infection or exposure is suspected, TB testing should be 
performed.  Patients who experience intolerable symptoms during treatment may discontinue the 
study at the discretion of the Investigator.  Patients may withdraw consent at any time.  At each 
study visit, an assessment should also be undertaken for any changes in the physical exam, or 
symptoms such as pain, swelling or tenderness in leg(s), persistent breathing difficulties or 
shortness of breath, or severe headache, that are suggestive of an increased risk of 
thromboembolism.  Patients must be withdrawn if they have a prior history of thrombotic 
event(s) or experience a thromboembolic event.   
Assessment of treatment response with SALT for efficacy will occur every 4 weeks for the first 
.       
 
 

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7. 
SELECTION AND WITHDRAWAL OF PATIENTS 
Patients eligible for enrollment in the study must meet all of the following inclusion criteria and 
none of the exclusion criteria. 
7.1. 
Patient Inclusion Criteria 
1. Written informed consent, and authorization for release and use of protected health 
information. 
2. Have completed a 24-week Treatment Period in a previous qualifying CTP-543 clinical 
trial. 
3. Female subjects are eligible to participate if at least one of the following conditions 
applies: 
a) Is a woman of childbearing potential (WOCBP) and using a medically highly 
effective form of birth control with a failure rate less than 1% per year from at least 4 
weeks prior to Baseline until at least 30 days following last dose of study drug.  
Examples of medically highly effective birth control methods include: 
i. Combined (estrogen and progestogen containing) hormonal contraception 
(oral, patch, vaginal ring)  
ii. Progestogen-only hormonal contraception associated with inhibition of 
ovulation (oral, injectable, implantable) 
iii. Intrauterine device or intrauterine hormone-releasing system 
iv. Bilateral tubal occlusion 
v. Vasectomized partner (partner is the sole sexual partner of the WOCBP 
trial participant and the vasectomized partner has received medical 
assessment of the surgical success) 
vi. Sexual abstinence (reliable as refraining from heterosexual intercourse 
during the above-mentioned period) 
b) Is not a WOCBP:  
i. 
Premenopausal with one of the following:  
a. Documented 
 
b. 
 
c. Documented bilateral oophorectomy.  
ii. 
Postmenopausal (cessation of menses for at least 12 months prior to 
screening) 
Postmenopausal is defined as no menses for 12 months without an 
alternative medical cause.  In addition, a high follicle stimulating 
hormone (FSH) level in the postmenopausal range may be used to 
hormonal contraception or hormone replacement therapy (HRT).  
However, in the absence of 12 months of amenorrhea, a single FSH 
measurement is insufficient.  Females on HRT and whose menopausal 
status is in doubt will be required to use one of the nonestrogen hormonal 
highly effective contraception methods if they wish to continue their 
HRT during the study.  Otherwise, they must discontinue HRT to allow 
confirmation of postmenopausal status before study enrollment 
4. Male participants must: 

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a. Agree to use, with their partners, male contraception (condom) and one of the 
highly effective contraceptive methods listed in Inclusion Criterion 5, from 
 
b. Refrain from donating sperm duri
end of the study.  
5. Willing to comply with the study visits and requirements of the study protocol. 
7.2. 
Patient Exclusion Criteria 
1. Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment 
to the scalp, significant trauma to the scalp, or other scalp condition that may interfere 
with the SALT assessment, or untreated actinic keratosis anywhere on the body. 
2. Females who are nursing, pregnant, or planning to become pregnant while in the study, 
and for 30 days after last dose of study medication. 
3. Donation of blood at any point throughout the study and for 30 days after last dose of 
study medication.  
4. 
withholding IP have been met and have not recovered to values required to resume 
dosing. 
5. Any medical (e.g. any prior history of thromboembolism), psychiatric, or social condition 
that is likely to unfavorably affect the risk-benefit of continued study participation, 
interfere with study compliance, or confound safety or efficacy assessments.  
7.3. 
Patient Withdrawal Criteria 
All patients are free to withdraw from participation in the study at any time, for any reason, and 
without prejudice.  The Investigator must withdraw any patient from the study if the patient 
requests to stop participating in the study.  The Investigator, Sponsor, or its designee may 
remove a patient from the study at any time and for any reason.  In addition, patients must be 
withdrawn if they: 
 Have a prior history of thrombotic event(s)  
 Experience a thromboembolic 
 
 Experience an intolerable adverse event, including but not limited to bone marrow 
considered possibly related to study drug.  Because laboratory abnormalities can 
occasionally occur that do not in themselves pose an unacceptable risk to subject 
safety (e.g., CK elevations with exercise, laboratory errors or artifacts), the 
 of 
concern to the Investigator, will be discussed with the Medical Monitor and a 
determination will be made, based on a reassessment of risk-benefit for the subject, 
whether the subject should discontinue randomized treatment.  Documentation on the 
determination will be maintained in the study files  
 Require a medication that is prohibited by the protocol.  However, under certain 
circumstances, and in consultation with the Medical Monitor, short-term use of a 

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prohibited medication may not necessitate withdrawal when its use is unlikely to 
unfavorably alter the risk-benefit of subject participation or confound the study 
endpoints  
 Experience an adverse event of progressive multifocal leukoencephalopathy (PML) 
or skin cancer which are rare but potential risks of the drug  
 Do not follow guidelines specified in the protocol (i.e., is noncompliant with protocol 
procedures or study treatment administration)  
 In the opinion of the Investigator, have any medically appropriate reason or 
significant protocol violation   
 
 
 Have a serious adverse event associated with a SARS-CoV-2 
 
 Are lost to follow up  
 Are, in the judgement of the Investigator, not exhibiting clear benefit following one 
full year of active treatment with CTP-543  
 In the opinion of the Investigator, feel that continued study participation is 
undesirable or the risk-benefit profile has become unfavorable.  
Patients who withdraw or are withdrawn from the study will not be replaced. 
7.3.1. 
Patient Withdrawal Procedures 
A patient who prematurely discontinues study treatment/study participation should have all Early 
Termination Visit assessments performed (See Table 1 and Table 2).   
If a patient terminates early from the study, the Investigator will record the reason(s) for early 
termination on the relevant electronic case report form (eCRF).  The specific reason for the 
withdrawal should be carefully documented on the eCRF.   
Adverse events resulting in patient early termination will be followed to the satisfactory 
resolution and determination of outcome, as ascertained by the Investigator (and/or Sponsor, or 
See Section 10 Adverse Events.  The data will be recorded on the appropriate 
eCRF. 
7.4. 
Criteria for Study Termination 
7.4.1. 
End of Trial 
The end of the trial is defined as the date of the last visit of the last subject undergoing this trial.  
ays after the end of the clinical trial, the regular termination of the trial will be 
reported to the responsible ethics committee and competent authority according to national laws 
and regulations.  If the trial has to be terminated early, this period shall be reduced to 15 days 
and the reasons clearly explained.  
There are no prospective stopping criteria for this study.  The Sponsor reserves the right to 
discontinue the study at any time for clinical or administrative reasons.   
In the case of a negative change in the risk-benefit ratio, the study will not be continued even if 
adaptations to the maximum insured amount are requested. 

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Any termination required by the Sponsor must be implemented by the Investigator, if instructed 
to do so, in a time frame that is compatible with the patient’s well-being. 

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104, and 4-weeks post the final study visit.  The test date and result will be documented in a 
patient diary and brought to each in-clinic study visit by the patient.  Less significant changes in 
laboratory values may warrant clinical intervention and the Investigator should use his/her best 
clinical judgement when considering a dose interruption, whether for singular or aggregate 
laboratory results outside of the normal range, or for other clinical signs, symptoms, or 
considerations that suggest dose interruption is in the best interest of the patient.  Prior to a dose 
interruption, the laboratory values of interest should be confirmed with repeat testing at an 
interval deemed appropriate by the Investigator (e.g., at the next protocol-defined visit or an 
Unscheduled Visit).  Again, the Medical Monitor should be notified whenever possible prior to 
decisions for dose interruption. 
8.1.1. 
Management of Risk with Janus Kinase (JAK) Inhibitors  
On 23 January 2023, EMA’s human medicines committee (CHMP) endorsed the measures 
recommended by the Pharmacovigilance Risk Assessment Committee (PRAC) to minimize the 
risk of serious side effects with approved Janus kinase (JAK) inhibitors used to treat several 
chronic inflammatory disorders.  These recommendations were based on a review of available 
data, including the final results from a clinical trial of the JAK inhibitor Xeljanz (tofacitinib) and 
preliminary findings from an observational study involving Olumiant.  These side effects include 
cardiovascular conditions, blood clots, cancer and serious infections.  Janus kinase (JAK) 
inhibitors used to treat chronic inflammatory disorders are linked to a higher risk of major 
adverse cardiovascular events (MACE), venous thromboembolism (VTE), malignancy, serious 
infections and all-cause mortality.  
As CTP-543 is an investigational product in the JAK inhibitor class of drugs, CTP-543 should be 
used with caution in patients with risk factors for blood clots in the lungs and in deep veins 
(venous thromboembolism, VTE), patients at increased risk of major cardiovascular problems 
(such as heart attack or stroke), those who smoke or have done so for a long time in the past and 
those at increased risk of cancer.  Any patients presenting with an increased risk should be 
withdrawn per Section 7.3.  
8.1.2. 
SARS-CoV-2 Dose Interruption Guidance   
Subjects with both a positive SARS-CoV-2 test (antigen or antibody) AND signs or symptoms 
consistent with SARS-CoV-2 infection should immediately interrupt dosing with study drug.  
Dose interruption should continue until symptoms subside (it is not necessary to have a negative 
SARS-CoV-2 test result).  If symptoms do not subside within a 21-day dose interruption period, 
a discussion between the PI and the Medical Monitor should occur to evaluate whether or not the 
subject should discontinue treatment permanently.  Signs and symptoms include, but are not 
limited to: fever or chills, cough, shortness of breath or difficulty breathing, fatigue, muscle or 
body aches, headache, new loss of taste or smell, sore throat, congestion or runny nose, nausea or 
vomiting, diarrhea, trouble breathing, persistent pain or pressure in the chest, new confusion, 
inability to wake or stay awake, bluish lips or face. 
Subjects who have signs or symptoms consistent with SARS-CoV-2 infection should be tested 
for SARS-CoV-2 and do not have to dose interrupt unless the test result is positive.  Subjects 
who have a positive SARS-CoV-2 test (antigen or antibody) but do not have any signs or 
symptoms consistent with SARS-CoV-2 infection are not required to dose interrupt.   

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Table 6: 
SARS-CoV-2 Dose Interruption Criteria 
SARS-CoV-2 Test Result 
SARS-CoV-2 Symptoms 
Dose Interruption 
Positive 
Yes 
Yes 
Positive 
No 
No 
Negative 
Yes 
No 
Medical Monitor should be notified in case of a subject is diagnosed with monkeypox. Study 
drug should be interrupted during acute infection. 
8.2. 
Treatment Compliance 
At each scheduled study visit after enrollment, the Investigator or designee will interview the 
patient regarding treatment compliance and compare the number of dispensed versus returned 
study drug tablets.      
8.3. 
Study Drug Materials and Management 
Please consult the Pharmacy Manual for a complete description of the study drug and 
requirements for storage, handling, dispensing, accountability, returns and destruction. 
8.3.1. 
Physical Description of Study Drug 
CTP-543 are white, capsule-shaped tablets prescribed for oral administration.   
8.3.2. 
Study Drug Packaging, Labeling, and Storage 
CTP-543 tablets will be packaged and labeled by an appropriately qualified vendor.  Details of 
the packaging, labeling and dispensing instructions can be found in the Pharmacy Manual. 
The label(s) for the investigational product will include, at a minimum, Sponsor name, address 
and telephone number, the protocol number, investigational product name, dosage form, amount 
of investigational product per container, lot number, unique dosing bottle number, storage 
conditions, and required caution statements and/or regulatory statements, as applicable per local 
regulations. 
Adequate supplies of study drug will be provided to each site.  Study drug should be stored in the 
original package between 15°C to 25°
°F to 77°F), as stated on the product label, in a 
secure, temperature-monitored, locked area, under the responsibility of the Investigator or other 
authorized individual until dispensed to the patients. 
Study drug dispensed to patients should be stored in the original package at room temperature as 
stated on the package label.  No special handling procedures are required.  Additional 
information can be found in the Pharmacy Manual.    
8.3.3. 
Study Drug Preparation and Administration 
No study drug preparation is required.  Patients will be instructed to take study drug according to 
the study drug label.  Patients should dose study drug in the clinic on all Study Visit Days after 
clinical laboratory blood draws are completed.       

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Study drug should be taken at approximately the same times each day, with water, and 
consistently with or without meals.  If a dose is missed, the patient should skip the missed dose 
and resume dosing at the next scheduled dose.  The patient should not take two doses at the same 
time.  Deviations from prescribed dosing should be discussed at each visit for assessment of 
compliance and retraining when necessary. 
8.3.4. 
Study Drug Return and Disposal 
The Sponsor (or designee) will review with the Investigator and relevant site personnel the 
process for study treatment return, disposal, and/or destruction, including responsibilities for the 
site versus the Sponsor (or designee).  Specific requirements for destruction or return are defined 
in the Pharmacy Manual. 
8.3.5. 
Study Drug Accountability 
To satisfy regulatory requirements regarding drug accountability, all study drug will be 
reconciled in full.  The Investigator or designee must maintain accurate records of the receipt of 
study drug, including date received, lot number, amount received, condition of the package, and 
the disposition of study drug. 
Current dispensing records will also be maintained, including the date and amount of medication 
dispensed to each individual patient.  Returned study drug records will be maintained and final 
study drug reconciliation will also be recorded for each patient. 
8.4. 
Concomitant Medications and Other Requirements for Continued 
Eligibility 
All medications, including over-the-counter therapies (e.g., vitamins, herbal, and nutritional 
supplements) and vaccinations (including the SARS-CoV-2 vaccination), will be recorded in the 
patient’s source documentation and documented in the eCRF.   
Any change in medical history (including newly acquired risk factors for thromboembolism such 
as but not limited to: prolonged immobilization or surgery), adverse events, concomitant 
medications or physical exam findings that in the opinion of the investigator may lead to an 
increased risk of thromboembolism, should be evaluated for continuation of the subject in the 
study. The Medical Monitor should be consulted if, in the opinion of the investigator, the subject 
is at an increased risk for thromboembolism.   
To date, there is no clear evidence of any safety concern for receipt of a SARS-CoV-2 vaccine 
during the treatment period.  Existing data on JAK inhibitors and vaccines, including the SARS-
CoV-2 vaccine, are insufficient to conclude whether JAK inhibitors, including CTP-543, have an 
effect on vaccine effectiveness, though data to date do not suggest that a major effect is likely.  
As subject and public health are paramount, SARS-CoV-2 vaccination should not be delayed or 
withheld for subjects already enrolled in this trial.   
Knowledge about the vaccines and virus is continually growing.  The Sponsor or the Medical 
Monitor will notify sites as new information is obtained that might further inform vaccine-related 
questions during the study.  Please also carefully read the product information (package insert) 
for the vaccine being used, noting any specific precautions or warnings for concomitant 
administration with JAK inhibitors or immunosuppressants. 
Any concomitant medication deemed necessary for the wellbeing of the subject may be given at 
the discretion of the Investigator.  Use of medications that are prohibited per protocol might 

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9.
STUDY ASSESSMENTS AND PROCEDURES
The schedule of assessments and procedures is presented in Table 1 and Table 2 and should be 
referenced for details regarding the collection of each assessment at each visit.  Any missed visits 
and associated assessments due to the COVID pandemic (e.g. site closures or travel restrictions) 
will be recorded on the e-CRF as COVID-related.   
As the Baseline Visit for this study will occur on the same day as the End of Study Visit for the 
preceding clinical trial, assessments will not be repeated and results from the End of Study Visit 
will be carried over for this study. 
9.1.
Physical Examination
Complete physical exams will include vital signs, weight, an examination of all major organ 
systems, with an emphasis on assessing for active signs and symptoms of infection, exposure to 
tuberculosis, and skin examinations for skin cancers.  Evaluation for progressive multifocal 
leukoencephalopathy (PML) symptoms such as facial droop, general weakness, clumsiness, 
trouble speaking, personality changes, memory problems, and vision changes will be assessed.  
Complete physical exams will be performed according to the Schedule of Events (Table 1 and 
Table 2).           
Brief physical examinations will be performed according to the Schedule of Events (Table 1 and 
Table 2).  Brief PE should focus on an evaluation of safety and include vital signs, weight, 
abdominal palpation, and head, eyes, ears, nose and throat assessment, assessing for active signs 
and symptoms of infection, exposure to TB, inquiry on changes to skin lesions and/or moles 
indicative of skin cancers, and an evaluation for progressive multifocal leukoencephalopathy 
(PML) as noted above.  
At each study visit, an assessment should also be undertaken for any changes in the physical 
exam, or symptoms such as pain, swelling or tenderness in leg(s), persistent breathing difficulties 
or shortness of breath, or severe headache, that are suggestive of an increased risk of 
thromboembolism.   
Vital signs will be measured after the patient has been in a supine or semi-supine position for at 
least 5 minutes and will include blood pressure, pulse rate, respiratory rate, and temperature.   
Weight will be measured per institution standard of care.  Patients should wear light clothing and 
remove his/her shoes before weight is measured.   
9.2. 
Clinical Laboratory Assessments 
Clinical laboratory assessments for hematology, serum chemistry, and lipids (Table 7), will be 
performed under fasted conditions 
 
thereafter as presented in the Schedule of Events (Table 1 and Table 2).  Serum pregnancy 
testing will be performed at all in-clinic visits.  To minimize patient burden of additional clinic 
visits, u
, 
, and 4-weeks post the final study visit.  For responders, additional home pregnancy testing 
-weeks post the final study visit.  
The test date and result will be documented in a patient diary and brought to each in-clinic study 
visit by the patient.   

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Clinical laboratory samples should be collected at the beginning of each clinic visit.  Every effort 
should be made to collect clinical laboratory samples prior to the first daily dose on all Study 
Visit Days.      
The Investigator should indicate review of the laboratory reports throughout the study by signing 
and dating each report.  
All clinical laboratory results that fall outside the reference range will be interpreted by the 
Investigator as Abnormal, not clinically significant, or Abnormal, clinically significant. 
Laboratory results deemed Abnormal, clinically significant will be recorded as an adverse event 
in the eCRF and should be fully investigated and repeated for verification.  Clinically significant 
laboratory abnormalities indicative of hematologic or other effects requiring intervention should 
be discussed with the Medical Monitor.  Additional tests and evaluations required to establish the 
significance or etiology of a clinically significant abnormal result or to monitor the course of an 
adverse event should be obtained when clinically indicated.  Whenever possible, the etiology of 
the clinically significant abnormal findings will be documented on the eCRF.   
Any significant laboratory abnormalities that are either serious or unexpected will be promptly 
reported to the Sponsor.  Any additional relevant laboratory results obtained by the Investigator 
during the course of this study will be reported to the Sponsor or designee. 
Table 7: 
Clinical Laboratory Assessments 
Hematology 
Chemistry 
Serum and Urine Pregnancy 
Complete blood count  
Absolute neutrophil count 
Absolute eosinophil count 
Platelet count 
White blood cell count with 
differential 
Absolute reticulocyte count  
Hemoglobin A1c 
Alanine aminotransaminase 
Albumin  
Alkaline phosphatase  
Amylase  
Aspartate aminotransaminase  
Total bilirubin 
Direct bilirubin 
Indirect bilirubin
Blood urea nitrogen  
Calcium 
Carbon dioxide  
Chloride  
Creatinine 
Creatine kinase  
Glucose 
Lipase 
Total protein 
Phosphorus 
Potassium 
Sodium 
Uric Acid 
Human chorionic gonadotropin
(females of childbearing 
potential only) 
Lipids 
Other
Total cholesterol 
Low-density lipoprotein 
High-density lipoprotein 
triglycerides 
SARS-CoV-2 testing 
9.3. 
Electrocardiogram 
Twelve-lead electrocardiograms will be performed at the timepoints specified in the Schedule of 
Events (Table 1 and Table 2).   

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The patient should rest in a supine or semi-supine position for at least 5 minutes prior to the 
assessment being performed.  Individual parameters including heart rate, PR, QT, QTcF, QRS, 
and RR intervals will be collected.  Repeat electrocardiograms (if deemed necessary) should be 
performed at least 5 minutes apart. The Investigator should indicate review of the 
electrocardiogram reports throughout the study by signing and dating each report.   
9.4. 
Severity of Alopecia Tool (SALT) 
The SALT score was introduced as part of investigative guidelines published by the National 
Alopecia Areata Foundation (Olsen, 2004).  The SALT is a measure of hair absence that 
quantifies the amount of scalp surface without hair in a pre-specified quadrant of the scalp (right 
side, top, left side, back), that is further summed after applying a quadrant-specific multiplier 
indicative of scalp surface area contribution, to provide an overall score of total hair loss.  The 
SALT assessment will occur via live examination of the patient every 4 weeks for the first 12 
weeks then 
 thereafter.      
To reduce variability, one rater should perform the SALT assessment for the patient for the 
duration of the study.  All investigators using the SALT should be trained prior to use.  Please 
consult the associated SALT Manual for additional details regarding SALT scoring and training 
requirements for this study.  An example of the SALT assessment tool is provided in 
Appendix 
.  
9.5. 
Unscheduled Visit 
In addition to regularly scheduled protocol visits, an Unscheduled Visit may be conducted to 
ensure appropriate safety monitoring or follow-up of the patient, at the discretion of the 
Investigator.  For example, an Unscheduled Visit may be scheduled to monitor potential or actual 
clinically meaningful safety laboratory results, for confirming hematology results to support dose 
interruption or resumption of dosing thereafter, or for other clinical signs, symptoms, or 
considerations that warrant additional safety follow-up.  Only those criteria requiring additional 
monitoring should be performed at an Unscheduled Visit.  An Unscheduled Visit will not replace 
regularly scheduled protocol visits.  

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10.
ADVERSE EVENTS
10.1.
Definition of Adverse Event
An adverse event is any untoward medical occurrence that may appear or worsen in a patient 
during the course of a study.  It may be a new intercurrent illness, a worsening concomitant 
illness, an injury, or any concomitant impairment of the patient’s health, including laboratory test 
values, regardless of etiology.  Any worsening (i.e., any clinically significant adverse change in 
the frequency or intensity of a pre-existing condition) should be considered an adverse event.  A 
diagnosis or syndrome should be recorded on the adverse event page of the eCRF rather than the 
individual signs or symptoms of the diagnosis or syndrome.  A worsening of the condition under 
study, alopecia areata, will not be reported as an adverse event.  
All patients will be monitored for adverse events during the study.  Assessments may include 
monitoring of the following parameters:  the patient’s clinical symptoms, laboratory, physical 
examination findings, or findings from other tests and/or procedures. 
An adverse event reported after informed consent, but before the first dose of study drug (i.e., 
Day 1), will be considered a pretreatment adverse event and will be captured on the eCRF.  
Adverse events will be considered treatment-emergent if the onset is after the first dose of study 
drug or if an ongoing Adverse Event worsens after dosing is initiated.   
An abnormal laboratory value is considered to be an adverse event if the abnormality: 
 
 
 is judged by the Investigator to be of significant clinical importance requiring 
treatment, modification/interruption of investigational product dose, or any other 
therapeutic intervention 
Regardless of severity grade, only laboratory abnormalities that fulfill a seriousness criterion 
need to be documented as a serious adverse event.   
If a laboratory abnormality is one component of a diagnosis or syndrome, then only the diagnosis 
or syndrome should be recorded on the adverse event eCRF.  If the abnormality was not a part of 
a diagnosis or syndrome, then the laboratory abnormality should be recorded as the adverse 
event.  If possible, the laboratory abnormality should be recorded as a medical term and not 
simply as an abnormal laboratory result (e.g., record thrombocytopenia rather than decreased 
platelets). 
Vaccine-related AEs (e.g. fever, fatigue, headache) resulting directly from the vaccination may 
occur.  The temporal relationship of the event(s) to the vaccination should be clearly described 
and the PI should consider whether these AEs are “unlikely” to be, or are “not related” to study 
medication.  
10.2.
Evaluation of Adverse Events 
A qualified Investigator will evaluate all adverse events as to seriousness, severity/intensity, 
relationship to study drug, duration, action taken, and outcome.   
10.2.1. 
Serious Adverse Event  
A serious adverse event is an adverse event that fulfills the following criteria: 
 
 

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 Is life-threatening (Note: the term "life-threatening" refers to an event in which the 
 
 Requires inpatient hospitalization or prolongation of existing hospitalization 
 
 Results in persistent or significant disability/incapacity (a substantial disruption of the 
patient's ability 
 
 
 
 Constitutes an important medical event that may jeopardize the patient or may require 
medical or surgical intervention to prevent one of the other outcomes listed above. 
Important medical events are defined as those occurrences that may not be immediately 
life-threatening or result in death, hospitalization, or disability, but may jeopardize the patient or 
require medical or surgical intervention to prevent one of the other outcomes listed above.  
Examples of such medical events include allergic bronchospasm requiring intensive treatment in 
an emergency room or at home, blood dyscrasias or convulsions that do not result in inpatient 
hospitalization.   
Events not considered to be serious adverse events are hospitalizations for: 
 A procedure for protocol/disease-related investigations (e.g., sampling for laboratory, 
pharmacokinetic, and pharmacodynamic tests).  However, hospitalization or 
prolonged hospitalization for a complication of such procedures remains a reportable 
serious adverse event. 
 Hospitalization or prolongation of hospitalization for technical, practical, or social 
reasons, in absence of an adverse event. 
 A procedure that is planned (i.e., planned prior to the starting of t
must be documented in the source document and the eCRF.  Hospitalization or 
prolonged hospitalization for a complication remains a reportable serious adverse 
event. 
 An elective treatment of or an elective procedure for a pre-existing medical condition 
that does not worsen.  
 Emergency outpatient treatment or observation that does not result in admission, 
unless fulfilling other seriousness criteria above. 
If an adverse event is considered serious, the adverse event and serious adverse event eCRF must 
be completed. 
For each serious adverse event, the Investigator will provide information on severity, start and 
stop dates, relationship to investigational product, action taken regarding investigational product, 
and outcome. 
Queries pertaining to serious adverse events will be handled through the electronic data capture 
system or other appropriate means.  Urgent queries (e.g., missing causality assessment) may be 
handled by telephone. 

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10.2.2. 
Severity/Intensity 
For both adverse events and serious adverse events, the Investigator must assess the 
severity/intensity of the event.  
The National Cancer Institute-Common Terminology Criteria for Adverse Events (CTCAE) 
Version 5.0 should be used to grade the severity/intensity of all events.  These criteria will be 
provided in the Site Operations Manual.  If a CTCAE criterion does not exist, the Investigator 
should grade the severity according to the following criteria:  
 Grade 1 (mild): does not interfere with the patient’s usual function 
 Grade 2 (moderate): interferes to some extent with patient's usual function 
 Grade 3 (severe): interferes significantly with patient's usual function 
 Grade 4 (life-threatening): results in a threat to life or in an incapacitating disability 
 Grade 5 (death): results in death 
The term “severe” is often used to describe the intensity of a specific event (as in mild, moderate 
significance (such as severe headache).  This criterion is not the same as “serious” which is 
based on patient/event outcome or action criteria associated with events that pose a threat to a 
patient’s life or functioning.   
Seriousness, not severity, serves as a guide for defining regulatory obligations. 
10.2.3. 
Relationship to Study Drug 
Relationship should be assessed and provided for every adverse event/serious adverse event 
based on currently available information.  Relationship is to be reassessed and provided as 
additional information becomes available.  Adverse events will be classified by the Investigator 
as follows: 
Definitely Related: There is clear evidence to suggest a causal relationship, and other possible 
contributing factors can be ruled out.  The clinical event, including an abnormal laboratory test 
result, occurs in a plausible time relationship to drug administration and cannot be explained by 
concurrent disease or other drugs or chemicals.  The event resolves or improves upon withdrawal 
of drug (de-challenge).  The event would be considered as definitely related to the study drug 
upon results of a positive re-challenge procedure. 
Probably Related: There is evidence to suggest a causal relationship (e.g., the event occurred 
within a reasonable time after administration of the trial medication).  However, the influence of 
other factors that may have contributed to the event (e.g., the patient’s clinical condition, other 
concurrent disease, concomitant medications or events) is unlikely, and the event follows a 
clinically reasonable response upon withdrawal of drug (de-challenge).   
Possibly Related: There is evidence to suggest a causal relationship (e.g., the event occurred 
within a reasonable time after administration of the study drug). However, the influence of other 
factors may have contributed to the event (e.g., the patient’s clinical condition, other concurrent 
disease, concomitant medications or events).  
Unlikely Related: A clinical event, including an abnormal laboratory test result, whose temporal 
relationship to drug administration makes a causal relationship improbable (e.g., the event did 
not occur within a reasonable time after administration of the trial medication) and in which 

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other drugs or concurrent or underlying disease provide plausible explanations (e.g., the patient’s 
clinical condition, other concomitant treatments). 
Not related: The adverse event is completely independent of study drug administration, and/or 
evidence exists that the event is definitely related to another etiology.  There must be an 
alternative, definitive etiology documented by the clinician. 
10.2.4. 
Duration 
For all adverse events whether or not considered serious, the Investigator will provide a record of 
the start and stop dates of the event. Every effort should be made to resolve all adverse events 
with continued follow-up with the patient until appropriate resolution can be achieved. If an 
event is unresolved at the end of the study it will be recorded as ongoing.  
10.2.5. 
Action Taken 
The Investigator will record the action taken with investigational product as a result of an 
adverse event or serious adverse event on the eCRF, as applicable (e.g., discontinuation, or 
interruption of investigational product, as appropriate) and record if concomitant and/or 
additional treatments were given for the event. 
10.2.6. 
Outcome 
The Investigator will record the outcome of adverse events on the eCRF, as applicable (e.g., 
recovered, recovered with sequelae, not recovered, or death (due to the adverse event).   
10.3.
Follow-Up 
Adverse events, severe adverse events and serious adverse events, including clinically significant 
laboratory tests, electrocardiograms, or physical examination findings, will be followed, 
regardless of causality, for as long as necessary to adequately evaluate the patient’s safety, or 
until the event stabilizes, is otherwise explained, death occurs, or the patient is lost to follow up.  
If resolved, a resolution date should be provided.  The Investigator is responsible for ensuring 
that follow-up includes any supplemental investigations indicated to elucidate the nature and/or 
causality of the adverse event.  This may include additional clinical laboratory testing or 
investigations, examinations, or consultation with other health care professionals as is practical.  
10.4.
Pregnancy 
The Sponsor must be informed within 24 hours upon learning that a patient, or male patient’s 
partner, has become pregnant any time after the first dose of study drug until 30 days after the 
last dose of study drug. Prior to any data collection for a pregnant participant or a pregnant 
partner of a participant in study CP543.5002, written informed consent on the pregnant 
partner/participant information and consent form must be provided by the study participant or the 
partner of the participant, in accordance with local practice, law or regulation.  Information about 
the follow-up of the pregnancy will be explained to the pregnant participant or the pregnant 
partner of a participant.  A copy of the pregnant partner/participant information and consent form 
must be given to the patient, if applicable. 
While pregnancy itself is not considered an AE or SAE, pregnancy reports will be processed in 
accordance with the same procedures set forth for non-expedited SAEs.  The Pregnancy 
Notification eCRF in EDC should be used to report the pregnancy to the Sponsor or its designee.  
Patient pregnancies (or pregnancy of a male patient’s partner) must be followed until termination 

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of pregnancy or the birth of the child via a pregnancy notification.  The Pregnancy Outcome 
eCRF should be used to report information regarding the status of the infant.  No mandatory 
visits, tests, or specific assessments are required for this study.  However, the Principal 
Investigator will liaise regularly with the health care provider of the participant or with the health 
care provider of the partner until termination of the pregnancy or until the birth of the child.  
Follow-up of the pregnancy will be scheduled by the study participant or the partner of the 
participant with their health care provider and conducted according to the standard of care.  
Standard of care is defined as a diagnostic and customary clinical treatment/practice process that 
a clinician chooses according to their clinical judgement for a certain type of illness, or clinical 
circumstance based on the clinical practice guidelines.  The Clinical Research Organization 
(CRO) responsible for pharmacovigilance will contact the investigative site, advise them to 
follow the pregnancy, and query the site to report any complications during the pregnancy, or 
any adverse pregnancy outcomes. 
The pregnancy eCRF pages are completed in EDC, and require the site report any clinical 
finding on prenatal testing.  The obstetrical history is also collected.  The Principal Investigator 
advises the participant to call the site to report any complications.  In addition, the CRO 
pharmacovigilance group will query the site after the determined due date to obtain final 
pregnancy and newborn outcome.  
Pregnancy cases are not considered closed until all data fields on the pregnancy report form are 
properly completed, including pregnancy outcome.  Information on the outcomes of pregnancies 
will be reported along with all other safety data from the study in the final study report.  In 
addition, a database of information relating to reported pregnancies across all CTP-543 studies 
will be maintained in order to include these data in any subsequent marketing application. 
If pregnancy occurs in a female patient, then study drug should be discontinued immediately.  If 
a female partner of a male patient taking investigational product becomes pregnant, the male 
patient taking the investigational product should notify the Investigator, and the pregnant female 
partner should be advised to call her healthcare provider immediately. 
10.5.
Recording Adverse Events
All adverse events (regardless of seriousness or relationship to study drug) including those from 
the time informed consent is obtained through to the final study visit are to be recorded in the 
eCRF.  Each individual adverse event is to be listed as a separate entry.  The Investigator will 
provide information about dates of onset and resolution, seriousness, severity, action(s) taken, 
outcome, and relationship to the study drug. All adverse events should be documented in the 
patient’s source documents.  
10.6.
Reporting Adverse Events 
The Investigator must report to Sponsor or its designee all adverse events that occur during the 
study from the time written informed consent is given until the final study visit or early 
termination, regardless of their relationship to the study drug. Serious adverse events and 
pregnancies will be reported from the time written informed consent is given through 30 days 
beyond the last dose of study drug. 
10.6.1. 
Reporting Serious Adverse Events 
The Investigator is required to notify the Sponsor, and the Sponsor’s designated Drug Safety 
Unit, immediately, without undue delay, and no later than 24 hours after becoming aware of the 

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occurrence of a serious adverse event.  All serious adverse events will be reported through 
completion of the adverse event eCRF.  The Investigator will be responsible for reporting serious 
adverse events to the EC. 
If an Investigator becomes aware of a serious adverse event within 30 days after the last dose of 
study drug and it is considered by him/her to be caused by the study drug with a reasonable 
possibility, the event must be documented and reported through completion of the adverse event 
eCRF. 
10.6.2. 
Reporting Urgent Safety Issues 
If the study site staff becomes aware of an actual or potential urgent safety issue, then the 
Sponsor and Sponsor’s designee (Medical Monitor) must be immediately contacted so that 
appropriate urgent safety measures can be agreed upon.  An urgent safety issue is defined as:  
 An immediate hazard to the health or safety of patients participating in a clinical 
study 
 A serious risk to human health or potentially a serious risk to human health 
An urgent safety issue may include: (1) issues with an investigationa
(2) 
-
conduct of the study or that pose a risk to study patients. 
In exceptional circumstances of imminent hazard and in order to safeguard the health or safety of 
individuals, the Investigators may take urgent safety measures before informing the Sponsor, but 
the Sponsor must be informed immediately after the hazard has resolved.   

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11.
STATISTICAL METHODS
This section describes the rules, conventions, statistical analysis, and presentation of data for this 
study. Full details will be provided in the statistical analysis plan (SAP) for this study. 
Revisions during the study may be made to accommodate protocol amendments and to make 
changes to adapt to unexpected issues in study execution that could affect planned analyses.  A 
formal SAP for the analysis and presentation of data from this study will be prepared and issued 
before database lock.  The SAP will provide a more technical and detailed description of the 
proposed data analysis methods and procedures.  Deviations from the statistical analyses outlined 
rther modifications will be noted in the 
clinical study report (CSR).  All statistical analyses will be performed under oversight of the 
Sponsor. 
All available data will be included in data listings and tabulations. 
All data collected in this study will be presented using summary tables and patient data listings.  
Summary statistics for raw and change from Baseline data of continuous variables will 
minimally include n, mean, standard deviation (SD), minimum, median, and maximum. For 
categorical variables, frequencies and percentages will be presented. Graphical displays will be 
provided as appropriate.   
Endpoints will be assessed using simple descriptive statistics.   
11.1.
Endpoints 
11.1.1. 
Safety 
Safety of CTP-543 will be assessed by evaluating adverse event, clinical laboratories, physical 
examinations, vital signs, concomitant medications, and electrocardiogram results.  
11.1.2. 
Efficacy 
Efficacy of CTP-543 will include all patients who receive study drug and have at least 1 post-
treatment SALT assessment in this study.  Relative change in SALT score over time will be 
summarized descriptively by visit, as appropriate.   
11.2.
Analyses 
Efficacy will include all patients who receive study drug and have at least 1 post-treatment 
SALT assessment in this study.  Safety will include all patients who receive study drug. Patients 
will be summarized according to study drug regimen received (i.e., as treated). 
Data will be summarized by dosing regimen.  All data for analysis will be listed by patient.  
Dosing regimens for summarization will be defined in SAP.   
Continuous measures will be summarized descriptively (mean, standard deviation, median, 
minimum value, and maximum value) and categorical measures will be presented as number and 
percentage.   
11.2.1. 
Disposition and Baseline Characteristics 
Disposition will be summarized by dosing regimen.  The number and percentage of patients, who 
are enrolled, treated, prematurely discontinued, and completers will be summarized.  

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Baseline characteristics will be summarized by dosing regimen for patients participating in the 
Treatment Period.  Dosing regimens for summarization will be defined in SAP.   
Medical history will be coded with the Medical Dictionary for Regulatory Activities (MedDRA) 
terminology.  Concomitant medications will be summarized by World Health Organization Drug 
Dictionary Anatomical-Therapeutic-Chemical classification and preferred term.   
11.2.2. 
Study Drug Exposure 
For each patient, the number of days of exposure to CTP-543 will be summed across the 
Treatment Period.  The total number of days on study drug will exclude dose interruptions.  The 
total number of days of exposure to CTP-543 by dose will be summarized with the mean, 
standard deviation, median, minimum, and maximum number of days.   
11.2.3. 
Safety 
All safety summaries will be descriptive with no statistical hypothesis testing and based on the 
Safety Population.  All safety endpoints will be listed in by-patient data listings. 
Adverse events will be coded using MedDRA and summarized by system organ class and 
preferred term.  Clinically significant deteriorations in physical examination findings will be 
reported and summarized as adverse events. 
Laboratory values will be converted to the project-defined unit of measurement, as applicable, 
before analysis.  Abnormal, clinically significant laboratory values will be reported and 
summarized as adverse events. 
11.2.3.1. Adverse Events 
Treatment-emergent adverse events (TEAEs) will be defined as any adverse event that occurs 
after administration of the first dose of study drug until completion of the study or the Early 
Termination Visit.  The number and percentage of patients who report TEAEs will be 
summarized by system organ class and preferred term.  
Treatment emergent adverse events will also be summarized by intensity as well as relationship 
to study drug.   
Patients who report the same preferred term on multiple occasions will be counted once for the 
preferred term: under the highest severity when summarized by severity and under the closest 
relationship to study drug when summarized by relationship.  If a patient reports multiple 
preferred terms for a system organ class, the patient will be counted only once for that system 
organ class. 
The number and percentage of patients who experience TEAEs will be summarized by dosing 
regimen for the following: 
 By system organ class and preferred term 
 By severity/intensity, system organ class, and preferred term 
 By relationship to study drug, system organ class, and preferred term 
 Serious adverse events by system organ class and preferred term 
 Serious adverse events by relationship to study drug, system organ class, and 
preferred term 

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 Adverse events resulting in discontinuation of study drug by system organ class and 
preferred term 
 Adverse events that result in study drug dose interruption by system organ class and 
preferred term 
By-patient listings will be provided for any deaths, serious adverse events, and adverse events 
leading to discontinuation of treatment.   
11.2.3.2. Clinical Laboratory 
Clinical laboratory variables will be presented 3 ways.  First, change from Baseline to each 
scheduled assessment will be summarized descriptively.  Baseline will be defined as the 
laboratory value obtained before the first dose of study drug on Day 1. 
Second, treatment-emergent potentially clinically significant (PCS) laboratory values will be 
identified.  Potentially clinically significant values are defined as those that meet Grade 3 or 
Grade 4 toxicity criteria from the CTCAE criteria.  Treatment-emergent PCS laboratory values 
are those in which the Baseline value is not PCS and the post-baseline value is PCS. 
Third, treatment-emergent adverse events that result in dose interruption will also be identified.  
The mean change from Baseline to each scheduled assessment will be summarized descriptively 
by dosing regimen for each clinical laboratory variable specified in this protocol. 
The number and percentage of patients with Abnormal, clinically significant laboratory values 
(per Investigator judgement) and the number and percentage of patients with treatment-emergent 
PCS laboratory values will be summarized by dosing regimen for each clinical laboratory 
variable. 
11.2.3.3. Vital Signs 
The mean change from Baseline to each scheduled assessment will be summarized descriptively 
by dosing regimen for each vital sign variable specified in this protocol. 
Baseline will be defined as the last vital sign value obtained before the first dose of study drug on 
Day 1.  
11.2.3.4. Electrocardiogram 
The change from Baseline in electrocardiogram intervals (PR, QT, QTcF, QRS, and RR) to each 
scheduled assessment will be summarized descriptively by dosing regimen. 
 

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12.
REGULATORY CONSIDERATIONS
It is the responsibility of the clinical site and staff to notify the Sponsor and Sponsor’s designee 
immediately upon becoming aware of a serious breach of GCP or of the study protocol.  It is the 
responsibility of the Sponsor or its designee to notify appropriate regulatory authorities of any 
serious breach which is likely to effect, to a significant degree, the safety or mental integrity of 
the patients of the study or the scientific value of the study.  
12.1.
Good Clinical Practice 
The procedures set out in this study protocol pertaining to the conduct, evaluation, and 
documentation of this study are designed to ensure that the Sponsor, its authorized 
(R2) and in 
accordance with the general ethical principles outlined in the Declaration of Helsinki.  The study 
will receive approval from the EC prior to commencement.  The Investigator will conduct all 
aspects of this study in accordance with applicable national, state, and local laws of the pertinent 
regulatory authorities. 
12.2.
Sponsor’s Responsibilities 
The Sponsor or its designee is responsible for the following: 
 Selecting qualified Investigators 
 Providing Investigators with the information they need to properly conduct an 
investigation 
 Ensuring proper monitoring of the investigation 
 Ensuring that the applicable regulatory authorities, and all participating Investigators 
are properly informed of significant new information regarding adverse events or 
risks associated with the medication being studied 
Before an investigational site can enter a patient into the study, a representative of the Sponsor 
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 the Sponsor or its representatives.  This 
will be documented in a Clinical Study Agreement between the Sponsor and the 
Institution/Investigator. 
During the study, a monitor from Sun Pharmaceutical or its 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 

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records relevant to the study.  This will require direct access to all original records for 
each patient (e.g., clinic charts). 
 Due to the COVID-
travel 
restrictions, source data verification may be performed remotely to the degree allowed 
per local regulations. 
NOTE: If allowed per local regulations, remote source data verification will be instituted 
only during the COVID-
mporary measures that will be 
rescinded as soon as the rules governing the management of clinical trials during the 
COVID-
. 
 Record and report any protocol deviations. 
 Confirm adverse events and serious adverse events have been properly documented on 
eCRFs and confirm any serious adverse events have been forwarded to the Sponsor and 
those serious adverse events that met criteria for reporting have been forwarded to the 
EC. 
The monitor will be available between visits if the Investigator(s) or other staff needs 
information or advice. 
As the Sponsor, Sun Pharmaceutical has delegated some responsibilities to a designee, or 
Contract Research Organization. 
12.3.
Investigator’s Responsibilities 
Investigator responsibilities are set out in the ICH Guideline for GCP and in the local 
regulations.  Each Investigator participating in this study is required to maintain complete and 
accurate study documentation in compliance with current GCP standards and all applicable local 
regulations related to the conduct of a clinical study. 
The Principal Investigator will ensure that all persons assisting with the study are adequately 
informed about the protocol, any amendments to the protocol, the study drug, and their 
study-related duties and functions.  The Principal Investigator will maintain a list of sub-
Investigators and other appropriately qualified persons to whom he or she has delegated 
significant study-related duties.   
The Investigator is responsible for keeping a record of all patients who sign an informed consent 
document and are screened for entry into the study.  Patients who fail eligibility requirements 
must have the reason(s) recorded in the patient’s source documents. 
The Sponsor, or designee, should inform the EC of any event likely to affect the safety of 
patients or the continued conduct of the study, in particular any change in safety.  Additionally, 
all updates to the Investigator’s Brochure will be sent to the EC.  A progress report will be sent 
to the EC and the protocol will be reviewed annually (e.g., re-approved) or more frequently, as 
required by the EC or local regulations.  
The Investigator will maintain a copy of all correspondence with the EC, including copies of 
approved documents submitted by the Sponsor or designee.  The Investigator will also maintain 
a copy of the EC membership list with occupation and qualification (or a statement confirming 
compliance with GCP requirements for committee composition).   
The Sponsor or designee is responsible for notifying the EC/RA of the conclusion of the clinical 

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terminated early, notification must be made within 15 days of the early termination. The final 
study report must be submitted within 1 year after study completion. 
The Investigator, or a designated member of the Investigator’s staff, must be available during 
monitoring visits to review data, resolve queries and allow direct access to patient records (e.g., 
medical records, office charts, hospital charts, and study-related charts) for source data 
verification.  The Investigator must ensure timely and accurate completion of eCRFs and queries. 
12.4.
Protocol Amendments
Any permanent change to the protocol, whether it is an overall change or a change for specific 
study center(s), must be handled as a protocol amendment.  All amendments to the protocol will 
be written by the Sponsor.  The Investigator will not make any changes to this protocol without 
prior written consent from the Sponsor and subsequent approval by the EC.  Except for 
administrative amendments, Investigators must await EC approval of protocol amendments 
before implementing the change(s).  The Sponsor will ensure submission of any protocol 
amendments to the appropriate regulatory agencies and EC. 
Trial conduct may be impacted in light of the COVID-
quarantines, site closures, travel limitations, interruptions to the supply chain for the investigational 
product, etc.) leading to difficulties in meeting protocol-specified procedures, including 
administering or using the investigational product or adhering to protocol-mandated visits and 
laboratory/diagnostic testing.  Taking into consideration recent Regulatory Agency guidance, 
amendments to protocol defined criteria may be implemented without EC approval or before filing 
an amendment to the IND/CTA in order to minimize or eliminate immediate hazards or to protect the 
life and well-being of research participants (e.g., to limit exposure to COVID-
.  
Administrative amendments are defined as having no effect on the safety of the research 
subjects, scope of the investigation, or quality of the study.  However, a protocol change 
intended to eliminate an apparent immediate hazard to patients should be implemented 
immediately, and the EC notified within 5 days or per local regulations.   
When, in the judgement of the chairman of the local EC, the Investigators, and/or the Sponsor, 
the amendment to the protocol substantially alters the study design and/or increases the potential 
risk to the patient, the currently approved written ICF will require similar modification.  In such 
cases, repeat informed consent will be obtained from patients enrolled in the study before 
continued participation under the new amendment. 
12.5.
Audits and Inspections 
The Sponsor’s Quality Assurance Unit (or representative) may conduct audits at the study site(s) 
or remotely.  Audits will include, but are not limited to:  drug supply, presence of required 
documents, the informed consent process, laboratory specimen processing, and comparison of 
eCRFs with source documents.  The Investigator agrees to cooperate with audits conducted at a 
reasonable time and in a reasonable manner. 
Regulatory authorities worldwide may also inspect the Investigator during or after the study.  
The Investigator should contact the Sponsor immediately if this occurs, and must fully and 
reasonably cooperate with Regulatory inspections. 
The Investigator is required to make all study documentation promptly available for inspection, 
review or audit upon request by Sponsor, its representatives, or any appropriate regulatory 
agencies. 

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12.6.
Quality Control and Quality Assurance
All aspects of the study will be carefully monitored by the Sponsor or its authorized 
representative for compliance with applicable government regulations with respect to current 
GCP and standard operating procedures.   
A quality control and quality assurance plan addressing aspects of the study that may impact data 
integrity or the protection of human subjects may be instituted for this study.  Audit findings and 
follow-up will be documented as appropriate based on Sponsor Standard Operating Procedures.   
In light of the COVID-
risk-based approaches to patient safety will be instituted, as 
applicable and per local regulations.  Compliance with Good Clinical Practice (GCP) will be 
maintained and risks to patient safety and data integrity will be maintained.   

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13.
DATA HANDLING AND RECORDKEEPING
13.1.
Confidentiality 
All information disclosed or provided by the Sponsor (or designee), or generated or produced 
during the study including, but not limited to, the protocol, the eCRFs, the Investigator’s 
Brochure, and the results obtained during the course of the study, are confidential.  The 
Investigator or any person under his/her authority agrees to keep confidential and not to disclose 
the information to any third party without the prior written approval of the Sponsor. 
Submission of this protocol and any other necessary documentation to the EC is expressly 
permitted, EC members having the same obligation of confidentiality.  Authorized regulatory 
officials and Sponsor personnel (or designee) will be allowed full access to inspect and copy the 
records.  The copied and inspected records will remain at the site and will not be transmitted or 
removed from the site.  Study drug, patient bodily fluids, and/or other materials collected shall be 
used solely in accordance with this protocol, unless otherwise agreed to in writing by the 
Sponsor and responsible ethics committee(s) or regulatory authorities. 
Patients’ names may, however, be made known to a regulatory agency or other authorized 
officials in the event of inspections.  Documents containing the full name or other personally 
identifiable information of the patient are to remain at the site.  This information will not be 
transferred to the Sponsor nor be contained in regulatory filings.   
13.2.
Patient Data Protection 
Prior to any testing under this protocol, candidates must provide all authorizations required by 
local law (e.g., protected health information authorization).  
Patients will be identified only by unique patient numbers in eCRFs and other datasets generated 
for this study.  The patient will not be identified by name in the eCRF, in any study samples or 
study reports.  All data generated in this study is for research purposes only.  The Sponsor, its 
partner(s) and designee(s), and various government health agencies may inspect the records of 
this study.  Every effort will be made to keep the patient’s personal medical data confidential.  
The Sponsor will protect individual patient information to the fullest extent possible during this 
study.  At no time will a patient become identified in any publication or presentation.  However, 
the patient may have to become identified in the event of a regulatory authority audit or 
inspection in order to verify the accuracy of the data.  Access to patient information is at the 
discretion of the Sponsor and cannot occur prior to database lock or other specified events as 
determined solely by the discretion of the Sponsor. 
13.3.
Data Collection 
All data obtained for analysis in the clinical study described in this protocol will use an 
electronic data capture system.  Data reported in the eCRFs should be consistent with and 
substantiated by the patient’s medical record and original source documents.  Any discrepancies 
must be explained.   
Prior to the start of the study, the Principal Investigator will complete a Delegation of Authority 
form (Site Signature and Delegation Log), showing the signatures and handwritten initials of all 
individuals and the delegation of responsibilities, such as identifying those individuals who are 
authorized to make or change entries on eCRFs. 

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13.4.
Case Report Form Completion
Data within the eCRF will be monitored by a Clinical Research Associate according to the 
Monitoring Plan.  Queries will be generated based on discrepancies found while monitoring.  
Site personnel will review and respond to these queries appropriately.  Additionally, the 
Sponsor’s designee and the Sponsor may periodically perform aggregate data reviews, which 
could result in queries being generated for site personnel resolution.  The completed eCRF for 
each patient must be signed and dated by the Principal Investigator to signify that the Principal 
Investigator has reviewed the eCRF and certifies it to be complete and accurate.  
13.5.
Database Management, Data Clarification, and Quality Assurance
The Sponsor’s designee (i.e., a designated Contract Research Organization) will be responsible 
for data management.  Data Management will develop a Data Management Plan (DMP) 
document, and provide it to the Sponsor for approval.  The DMP document will define all 
activities in the data collection and cleaning process.  The detailed DMP will be based on the 
protocol, work scope, contract, analysis plans, data-flows, eCRFs, data cleaning procedures, 
other supporting documents, and data management standards and practices. 
The programmed data validations will be run to check for database completeness and 
consistency, and queries will be generated upon data entry or via review by a Clinical Data 
Manager after entry.  The sites will respond to the data queries in a timely manner. 
Quality control procedures will be conducted prior to database lock according to the designated 
Contract Research Organization standard operating procedures. 
When the database has been declared to be complete and accurate, it will be locked.  Any 
changes to the database after that time will only be made by joint written agreement between the 
Sponsor, Statistician, Data Manager, and Quality Assurance Auditor according to designated 
standard operating procedures of the Contract Research Organization. 
13.6.
Inspection of Records 
According to the ICH guidelines for GCP, the Sponsor or designee must verify data entered in 
the eCRF entries against the source documents. The objective of source document verification is 
to comply with GCP and international regulatory requirements and to reduce the risks of fraud.  
Source document verification means ensuring that source documents are an accurate and 
confirmable reflection of the patient’s evaluations during participation in the study and that all 
relevant information recorded in the source document is accurately entered into the eCRF. All 
source documents should be correctly labeled and filed and associated with a single, verifiable 
patient.  
All data required for this study should be captured in source notes.  No data obtained by the 
Investigator or other study personnel should be captured directly in the eCRF.  All source 
documents pertaining to this study will be maintained by the Investigator and made available for 
inspection by authorized persons.  If electronic progress notes and other electronic source 
documents are not compliant with applicable regulatory guidance, they are not considered a valid 
source for this study.  All patient progress notes must be dated and signed at the time of the visit.  
The Sponsor reserves the right to terminate the study for refusal of the Investigator to supply 
original source documentation for this clinical study. 
The Investigator will note in a source independent from the eCRF the following information: 

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 Information to confirm that the patient exists, as applicable per local regulations (e.g., 
initials, year 
 
 
 
 Confirmation that the patient is taking part in the clinical study  
 
 
 
 
 
 
 Details of concomitant and investigational medications. 
Source document verification is not a substitute for clinical study monitoring, the purpose of 
which is to ensure that the protocol has been followed correctly, the eCRF has been fully and 
accurately completed, source document verification has been carried out, and the study timelines 
and enrollment goals and requirements have been met.  In light of the COVID-
, 
source document verification may occur remotely, as applicable, and in accordance with local 
regulations.  Additional detail on remote source document verification will be provided in the 
study plans.  
13.7.
Retention of Records 
For all trials in which the clinical trial data are used to support a marketing authorization, per 
y records will be retained for at 
least 15 years after completion or discontinuation of the trial or at least two years after the 
granting of the last marketing authorization in the European Union (when there are no pending or 
contemplated marketing applications in the EU) or for at least two years after formal 
discontinuation of clinical development of the investigational product, whatever is the longest. 
The Investigator must maintain all study documentation as confidential, and take measures to 
prevent accidental or premature destruction of these documents. 
The Investigator must notify the Sponsor prior to destroying any study essential documents. 
If the Investigator can no longer ensure archiving, they shall inform the Sponsor.  The relevant 
records shall be transferred to a mutually agreed upon designee. 

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14.
PUBLICATION POLICY
The results of this study may be published in a medical publication, journal, or may be presented 
at a medical conference or used for teaching purposes.  Additionally, this study and its results 
may be submitted for inclusion in all appropriate health authority study registries, as well as 
publication on health authority study registry websites, as required by local health authority 
regulations.  Selection of first authorship will be based on several considerations, including, but 
not limited to study participation, contribution to the protocol development, and analysis and 
input into the manuscript, related abstracts, and presentations in a study.