A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
Phase: PHASE1
Conditions: Muscular Dystrophy, Duchenne
Sponsor: Sarepta Therapeutics, Inc.

SRP-9001 
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CLINICAL STUDY PROTOCOL 
DRUG: 
SRP-9001 (formerly referred to as 
rAAVrh74.MHCK7.microdystrophin)  
STUDY NUMBER: 
SRP-9001-102  
STUDY TITLE: 
A Multicenter, Randomized, Double-Blind, Placebo-
Controlled Trial for Duchenne Muscular Dystrophy 
Using SRP-9001 
IND NUMBER: 
17763 
SPONSOR: 
Sarepta Therapeutics, Inc. 
215 First Street  
Cambridge, MA 02142 USA 
Phone: +1-617-274-4000 
CURRENT VERSION DATE: 
Version 9 (Amendment 8), 12 April 2023  
REPLACES VERSION DATE: 
Version 8 (Amendment 7), 27 August 2020 
Version 7 (Amendment 6), 18 May 2020 
Version 6 (Amendment 5), 25 September 2019 
Version 5 (Amendment 4), 02 July 2019 
Version 4 (Amendment 3), 04 June 2019 
Version 3 (Amendment 2), 26 February 2019 
Version 2 (Amendment 1), 09 November 2018 
Version 1 (Amendment 0), 13 April 2018 
CONFIDENTIALITY STATEMENT 
The information contained in this document, is the property of the Sponsor and is confidential. This information 
may not be disclosed, reproduced or distributed to anyone other than personnel directly involved in the conduct 
of the study and in response to a relevant Institutional Review Board/Independent Ethics Committee and Review 
by a Regulatory Authority as required by the applicable laws and regulations, without the written authorization of 
the Sponsor, except to the extent necessary to obtain written informed consent from those individuals to whom 
the drug may be administered. These restrictions will continue to apply after the study has closed. 

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INVESTIGATOR’S AGREEMENT 
 
I have received and read the Investigator’s Brochure for SRP-9001. I have read the 
SRP-9001-102 Study Protocol (Version 9, Amendment 8 dated 12 April 2023) and agree to 
conduct the study as outlined. I agree to maintain the confidentiality of all information received 
or developed about this protocol. 
 
 
 
 
Printed Name of Investigator 
 
Signature of Investigator 
 
Date 
 

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TABLE OF CONTENTS 
CLINICAL STUDY PROTOCOL ..................................................................................................1 
SIGNATURE PAGE .......................................................................................................................2 
INVESTIGATOR’S AGREEMENT ...............................................................................................3 
TABLE OF CONTENTS .................................................................................................................4 
LIST OF TABLES ...........................................................................................................................8 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS ..................................................9 
1. 
PROTOCOL SUMMARY ..........................................................................................11 
1.1. 
Synopsis ......................................................................................................................11 
1.2. 
Schema ........................................................................................................................15 
1.3. 
Schedule of Activities .................................................................................................16 
2. 
INTRODUCTION ......................................................................................................21 
2.1. 
Study Rationale ...........................................................................................................21 
2.2. 
Background .................................................................................................................21 
2.3. 
Benefit/Risk Assessment ............................................................................................22 
3. 
STUDY DESIGN .......................................................................................................23 
3.1. 
Overall Design ............................................................................................................23 
3.1.1. 
Screening/Baseline Period ..........................................................................................23 
3.1.2. 
Treatment Period ........................................................................................................23 
3.1.2.1. 
Part 1 ...........................................................................................................................23 
3.1.2.2. 
Part 2 ...........................................................................................................................24 
3.1.2.3. 
Part 3 ...........................................................................................................................25 
3.2. 
Scientific Rationale for Study Design ........................................................................26 
3.3. 
 ..................................................................................................26 
3.4. 
End of Study Definition ..............................................................................................27 
4. 
STUDY POPULATION .............................................................................................28 
4.1. 
Inclusion Criteria ........................................................................................................28 
4.2. 
Exclusion Criteria .......................................................................................................28 
4.3. 
Lifestyle Considerations .............................................................................................29 
4.4. 
Screen Failures ............................................................................................................29 
5. 
STUDY INTERVENTION ........................................................................................30 
5.1. 
Study Intervention(s) Administered ...........................................................................30 
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5.1.1. 
Pre-infusion Immunosuppressants ..............................................................................30 
5.1.2. 
Post-infusion Immunosuppressants ............................................................................30 
5.1.3. 
Intravenous Infusion (Day 1, Part 1 and Part 2) .........................................................31 
5.1.4. 
Post Infusion Monitoring ............................................................................................31 
5.1.4.1. 
Immediately Following Infusion ................................................................................31 
5.1.4.2. 
Extended Follow-up ....................................................................................................31 
5.2. 
Preparation/Handling/Storage/Accountability ............................................................31 
5.3. 
Measures to Minimize Bias: Randomization and Blinding ........................................31 
5.3.1. 
Establish Patient Identification Number .....................................................................31 
5.3.2. 
Assignment to a Treatment Group ..............................................................................31 
5.3.3. 
Randomization ............................................................................................................32 
5.3.4. 
Blinding/Unblinding ...................................................................................................32 
5.4. 
Concomitant Therapy .................................................................................................33 
5.4.1. 
Rescue Medicine .........................................................................................................33 
5.5. 
Dose Modification ......................................................................................................33 
5.5.1. 
Stopping Rules ............................................................................................................33 
5.5.2. 
Dosing Schedule .........................................................................................................33 
5.6. 
Study Discontinuation ................................................................................................34 
5.7. 
Intervention after the End of the Study .......................................................................34 
6. 
STUDY ASSESSMENTS AND PROCEDURES ......................................................35 
6.1. 
Efficacy Assessments .................................................................................................35 
6.1.1. 
Muscle Biopsy ............................................................................................................35 
6.1.2. 
Immunofluorescence Fiber Intensity / Immunofluorescence PDPF ...........................35 
6.1.3. 
 ...................................................................................35 
6.1.4. 
t .......................................................................................35 
6.1.5. 
Physical Functional Assessments ...............................................................................36 
6.1.5.1. 
Time to Rise from the Floor .......................................................................................36 
6.1.5.2. 
Ascend 4 Steps ............................................................................................................36 
6.1.5.3. 
North Star Ambulatory Assessment ...........................................................................36 
6.1.5.4. 
10-meter Timed Test ...................................................................................................36 
6.1.5.5. 
100-meter Timed Test .................................................................................................36 
6.2. 
Safety Assessments .....................................................................................................36 
6.2.1. 
Physical Examinations ................................................................................................36 
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6.2.2. 
Vital Signs ..................................................................................................................37 
6.2.3. 
Electrocardiograms and Echocardiograms .................................................................37 
6.2.4. 
Clinical Safety Laboratory Assessments ....................................................................37 
6.2.5. 
Safety Monitoring .......................................................................................................39 
6.2.5.1. 
Safety Monitoring for Liver Chemistry Tests ............................................................39 
6.2.5.2. 
Safety Monitoring for Hypersensitivity and Complement-Mediated 
Reactions .....................................................................................................................39 
6.2.5.3. 
Safety Monitoring for Platelet Count Results .............................................................40 
6.2.5.4. 
Safety Monitoring for Rhabdomyolysis .....................................................................41 
6.2.6. 
 ......................................................................................41 
6.2.7. 
 .........................................................................................................42 
6.2.8. 
 ................................................................................42 
6.3. 
Adverse Events and Serious Adverse Events .............................................................42 
6.3.1. 
Collection of Adverse Events .....................................................................................42 
6.3.2. 
Definition of Adverse Events .....................................................................................42 
6.3.2.1. 
Adverse Event .............................................................................................................42 
6.3.2.2. 
Serious Adverse Events ..............................................................................................43 
6.3.2.3. 
Adverse Events of Special Interest .............................................................................43 
6.3.3. 
Classification of Adverse Events ................................................................................43 
6.3.3.1. 
Relationship to Investigational Product ......................................................................43 
6.3.3.2. 
Relationship to Study Procedures ...............................................................................44 
6.3.3.3. 
Relationship to Underlying Disease ...........................................................................44 
6.3.3.4. 
Severity of Adverse Events ........................................................................................44 
6.3.3.5. 
Outcome ......................................................................................................................44 
6.3.3.6. 
Action Taken Regarding the Investigational Product .................................................45 
6.3.3.7. 
Expectedness of an Adverse Event .............................................................................45 
6.3.3.8. 
Suspected Unexpected Serious Adverse Reactions ....................................................45 
6.3.4. 
Recording of Adverse Events .....................................................................................45 
6.3.5. 
Reporting Serious Adverse Events .............................................................................45 
6.3.6. 
Special Situations ........................................................................................................46 
6.3.6.1. 
Overdose .....................................................................................................................46 
6.3.6.2. 
Adverse Events of Special Interest .............................................................................46 
6.3.6.3. 
Death ...........................................................................................................................46 
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6.3.6.4. 
Unblinding due to a Medical Emergency ...................................................................47 
6.3.7. 
Responsibilities of the Investigator ............................................................................47 
6.3.8. 
Responsibilities of the Sponsor ..................................................................................47 
6.4. 
Treatment Overdose ....................................................................................................48 
6.5. 
Pharmacokinetics ........................................................................................................48 
6.6. 
Pharmacodynamics .....................................................................................................48 
6.7. 
 ..................................................................................................48 
6.7.1. 
 ....................................................................................49 
6.7.2. 
 ............................................................................................49 
6.8. 
Biomarkers ..................................................................................................................49 
6.9. 
 ....................................................49 
6.9.1. 
 .....................................................................................................49 
6.9.2. 
 ..................................................50 
7. 
STATISTICAL CONSIDERATIONS .......................................................................51 
7.1. 
Statistical Hypotheses .................................................................................................51 
7.2. 
Sample Size Determination ........................................................................................51 
7.3. 
Analysis Populations ..................................................................................................52 
7.4. 
Statistical Analyses .....................................................................................................52 
7.4.1. 
Efficacy Analyses .......................................................................................................52 
7.4.2. 
Safety Analyses ..........................................................................................................53 
7.5. 
Interim Analyses .........................................................................................................54 
7.5.1. 
Interim Analysis at Week 12 in Part 1 ........................................................................54 
7.5.2. 
Interim Analysis at the Completion of Part 1 .............................................................54 
7.5.3. 
 ..........................................54 
7.5.4. 
Interim Analysis Based on Regulatory Interactions ...................................................54 
7.6. 
Data Monitoring Committee .......................................................................................54 
8. 
DISCONTINUATION OF STUDY INTERVENTION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL .......................................55 
8.1. 
Discontinuation of Study Intervention........................................................................55 
8.2. 
Participant Discontinuation/Withdrawal from the Study ...........................................55 
8.3. 
Lost to Follow-up .......................................................................................................55 
9. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS ..................................................................................................57 
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9.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight Considerations ....................57 
9.1.1. 
Clinical Monitoring of the Study ................................................................................57 
9.1.2. 
Regulatory and Ethical Considerations ......................................................................57 
9.1.3. 
Financial Disclosure ...................................................................................................58 
9.1.4. 
Informed Consent Process ..........................................................................................58 
9.1.5. 
Data Protection ...........................................................................................................59 
9.1.5.1. 
Data Safety Monitoring Plan ......................................................................................59 
9.2. 
Committees Structure .................................................................................................59 
9.2.1. 
Data Monitoring Committee .......................................................................................59 
9.2.2. 
Dissemination of Clinical Study Data ........................................................................59 
9.2.2.1. 
Final Study Report ......................................................................................................59 
9.2.3. 
Source Documents ......................................................................................................59 
9.2.4. 
Publication Policy .......................................................................................................59 
9.3. 
Appendix 2: 
 .................................................................................................60 
10.
REFERENCES ...........................................................................................................61 
LIST OF TABLES 
Table 1: 
Schedule of Activities – Part 1 ...................................................................................16 
Table 2: 
Schedule of Activities – Part 2 ...................................................................................18 
Table 3: 
Schedule of Activities – Part 3 Open-Label Follow-Up .............................................20 
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LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
Abbreviation  
Definition 
AAV 
adeno-associated virus 
AE 
adverse event 
AESI 
adverse event of special interest 
aHUS 
atypical hemolytic uremic syndrome 
ALT 
alanine aminotransferase 
AST 
aspartate aminotransferase 
CFR 
Code of Federal Regulations 
 
 
CRF 
case report form 
CRO 
Contract research organization 
DMC 
data monitoring committee 
DMD 
Duchenne muscular dystrophy 
 
 
ECG 
electrocardiogram 
ECHO 
echocardiogram 
GCP 
Good Clinical Practices 
GGT 
gamma-glutamyl transferase 
GLDH 
glutamate dehydrogenase 
HIV 
human immunodeficiency virus 
ICF 
informed consent form  
ICH 
International Council on Harmonisation 
IEC 
Independent Ethics Committee 
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Abbreviation  
Definition 
IF 
immunofluorescence 
IP 
investigational product 
IRB 
Institutional Review Board 
ITT 
intent-to-treat 
IV 
intravenous(ly) 
IXRS 
interactive voice/web response system 
 
 
NOAEL 
no observable adverse effect level 
NSAA 
North Star Ambulatory Assessment 
PCS 
potentially clinically significant 
PDPF 
percent dystrophin positive fibers 
PI 
Principal Investigator 
 
 
qPCR 
quantitative polymerase chain reaction 
 
 
SAE 
serious adverse event 
SAP 
Statistical Analysis Plan 
SoA 
Schedule of Activities 
SUSARs 
suspected unexpected serious adverse reactions 
TEAE 
treatment-emergent adverse event 
ULN 
upper limit of normal 
WBC 
white blood cell  
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1. 
PROTOCOL SUMMARY 
1.1. 
Synopsis 
Protocol Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for 
Duchenne Muscular Dystrophy Using SRP-9001 
Short Title: A Randomized, Double-Blind, Placebo-Controlled Study of SRP-9001 for 
Duchenne Muscular Dystrophy 
Rationale: Based on nonclinical studies in mice and canine species deficient in dystrophin and 
from the 2006 Duchenne muscular dystrophy (DMD) trial (Mendell 2010) and the ongoing 
microDys-IV-001 clinical trial, data supports the use of gene transfer for micro-dystrophin 
expression in skeletal and cardiac muscle.  
Objectives and Endpoints: This study will assess the safety and efficacy of exogenous gene 
transfer in DMD patients by measuring biological and clinical endpoints in 3 parts: two 48-week 
randomized, double-blinded, placebo-controlled periods (Part 1 and 2), and an open-label 
follow-up period (Part 3). The primary objectives of this study are the assessments of the safety 
and efficacy of intravenous (IV) administration of SRP-9001 in DMD patients. 
Objectives 
Endpoints 
Safety 
• To evaluate the safety of SRP-9001  
• Treatment-emergent adverse events (TEAEs) 
and serious adverse events (SAEs) 
• Select clinical laboratory assessments 
Primary  
• To evaluate micro-dystrophin expression from 
SRP-9001 at 12 weeks post dosing (Part 1) as 
measured by Western blot of biopsied muscle 
tissue 
• Change in quantity of micro-dystrophin protein 
expression from Baseline to Week 12 (Part 1) 
as measured by Western blot  
• To evaluate the effect of SRP-9001 on physical 
functional assessments as assessed by the North 
Star Ambulatory Assessment (NSAA) over 
48 weeks (Part 1) 
• Change in NSAA total score from Baseline to 
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Objectives 
Endpoints 
Secondary  
• To evaluate the effect of SRP-9001 on physical 
functional assessments over 48 weeks (Part 1) 
• Change in time to rise from the floor from 
Baseline to Week 48 (Part 1) 
• Change in time to ascend 4 steps from 
Baseline to Week 48 (Part 1) 
• Change in time of 10-meter timed test from 
Baseline to Week 48 (Part 1) 
• Change in time of 100-meter timed test from 
Baseline to Week 48 (Part 1) 
• To evaluate micro-dystrophin expression from 
SRP-9001 at 12 weeks (Part 1) as measured by 
immunofluorescence (IF) fiber intensity of 
biopsied muscle tissue 
• Change in micro-dystrophin expression from 
Baseline to Week 12 (Part 1) as measured by 
IF fiber intensity  
• To evaluate micro-dystrophin expression from 
SRP-9001 at 12 weeks (Part 1) as measured by 
IF percent dystrophin positive fibers (PDPF) of 
biopsied muscle tissue 
• Change in micro-dystrophin expression from 
Baseline to Week 12 (Part 1) as measured by 
IF PDPF 
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2. 
INTRODUCTION 
SRP-9001 is a nonreplicating, recombinant adeno-associated virus (AAV) that is being 
developed for treatment in patients with Duchenne muscular dystrophy (DMD). 
2.1. 
Study Rationale 
There is a strong rationale for systemic gene delivery in young DMD boys based on studies 
of the natural history of this disease. Gene replacement therapy has been studied for the past 
10 to 15 years and shows very favorable results in nonclinical studies in mice and canine species 
deficient in dystrophin.  
The goal of SRP-9001 therapy is to increase the expression level of the micro-dystrophin protein 
in skeletal and cardiac muscle in order to increase strength and protect from contraction-induced 
injury. Correction of the underlying genetic defect in DMD using gene replacement with 
SRP-9001 is the most promising treatment. Duchenne muscular dystrophy affects all skeletal 
muscles in the body, in addition to the diaphragm and heart. As such, a systemic approach is 
necessary in order to provide the best possible prospect of direct benefit to patients. Utilizing the 
rAAVrh74 serotype allows for efficient transduction of cardiac, skeletal, and diaphragm muscle 
without the increased risk of regional delivery strategies. Increased dystrophin expression in vivo 
may potentially improve patient’s muscle function. SRP-9001 appears to have a favorable safety 
profile and to be generally well-tolerated in nonclinical studies. 
All patients will be screened for pre-existing immunity to rAAVrh74 to avoid potential immune 
reaction and rejection following transfer of the gene.  
2.2. 
Background 
Duchenne muscular dystrophy is the most common, severe childhood form of muscular 
dystrophy. Inheritance follows an X-linked recessive pattern. Birth prevalence has been 
estimated at 1 in 3500 to 5000 live male births (Mendell 2010). Approximately one-third of cases 
represent new mutations of the DMD gene with the remaining inherited on the X chromosome 
from a carrier mother. Questions usually begin to surface between 3 to 5 years of age regarding 
reduced motor skills that alert a need for diagnostic evaluation. Earlier recognition results from 
delay in motor milestones or family history. At times aminotransaminases are found to be 
elevated that lead to detection of creatine kinase (CK) elevation and subsequent further testing. 
Duchenne muscular dystrophy is relentlessly progressive with loss of ambulation by 12 years of 
age (Brooke 1983). Historically, patients died from respiratory complications. Now, a variety of 
factors protect the respiratory system related to improved supportive equipment, antibiotics, 
vaccines, and other ancillary methods (Eagle 2002). Consequently, patients tend to live longer 
but eventually succumb to cardiac failure (D’Amario 2017). 

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2.3. 
Benefit/Risk Assessment 
Nonclinical data and results from clinical study SRP-9001-101 suggest a positive benefit/risk 
relationship and support clinical investigation of SRP-9001 gene replacement in DMD patients.  
More detailed information about the known and expected benefits and risks and reasonably 
expected adverse events (AEs) of SRP-9001 may be found in the Investigator’s Brochure. 

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3. 
STUDY DESIGN 
3.1. 
Overall Design 
This is a randomized, double-blind, placebo-controlled, 3-part clinical study of systemic gene 
delivery of SRP-9001 in up to 44 DMD patients 4 to 7 years of age (inclusive) who either have 
a confirmed frameshift deletion or duplication between exons 18 to 58, or premature stop codon 
mutation between exons 18 to 58. Patients meeting all eligibility criteria will be randomized to 
SRP-9001 or placebo by the interactive voice/web response system (IXRS). All patients will 
have the opportunity to receive intravenous (IV) SRP-9001 (1.33 × 1014 vg/kg) in either Part 1 or 
Part 2. Part 3 of the study will be an open-label follow-up period.  
The Sponsor has developed a quantitative polymerase chain reaction (qPCR) method using 
a linear standard for dose titering. This method replaces the previously used qPCR method 
which used a supercoiled standard for dose titering developed by Nationwide Children’s 
Hospital. The scale for the linear standard method is different from the previous supercoiled 
standard method. For patients who received a dose in this study based on Nationwide Children’s 
Hospital qPCR method, the dose used was 2 × 1014 vg/kg which corresponds to 
1.33 × 1014 vg/kg as measured by the Sponsor’s qPCR method using a linear standard. Note that 
both numbers (ie, 2 × 1014 and 1.33 × 1014 vg/kg) refer to the same quantity of drug given to the 
patients; only the effective scale for the 2 methods is different. Hence, as of Amendment 5, 
Version 6, the reassigned dose for the additional patients in the study is 1.33 × 1014 vg/kg, and 
for the duration of this study, all references to dosing will use 1.33 × 1014 vg/kg. 
3.1.1. 
Screening/Baseline Period 
The Screening/Baseline period will be up to 4 weeks before Day -1 in Part 1. After obtaining 
informed consent, patients will be evaluated for eligibility. Screening will include collection 
of demographics and medical history, vital sign measurements, a physical examination, 
electrocardiogram (ECG), and an echocardiogram (ECHO). Blood and urine samples will be 
collected for clinical and safety laboratory assessments. Blood samples will also be collected 
for hepatitis B and C, human immunodeficiency virus (HIV), and antibodies to rAAVrh74 and 
antigen-specific T-cells to rAAVrh74 capsid and micro-dystrophin. A pretreatment muscle 
biopsy will involve the gastrocnemius muscle, or a muscle selected by the Principal Investigator 
(PI) and will be performed after eligibility but before Day -1 in Part 1. The parent/caregiver will 
be asked to complete questionnaires. 
Physical functional assessments will include the time to rise from the floor, time to ascend 
4 steps, North Star Ambulatory Assessment (NSAA), the 10-meter timed test, and the 100-meter 
timed test. 
3.1.2. 
Treatment Period 
3.1.2.1. 
Part 1 
Part 1 is a 48-week randomized, double-blind, placebo-controlled period. Patients meeting 
eligibility requirements will be randomized prior to or on Day -1 to SRP-9001 or placebo in a 
1:1 ratio by IXRS. Up to 22 patients will receive IV SRP-9001 (1.33 × 1014 vg/kg) and up to 

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22 patients will receive a matching volume (up to 10 mL/kg) of placebo (lactated Ringer’s 
solution). 
Patients may be admitted to the hospital the day before the study treatment is infused at the 
discretion of the Investigator. On Day -1, the patient will receive a physical examination, have 
vital signs collected, and provide blood and urine samples.  
The day prior to the study treatment infusion (SRP-9001 or placebo), patient’s background dose 
of steroid for DMD will be increased to at least 1 mg/kg of a glucocorticoid (prednisone 
equivalent) daily and continue at this level for at least 60 days after the infusion unless earlier 
tapering is judged by the PI to be in the best interest of the patient. 
SRP-9001 or placebo will be administered IV on Day 1 over 1 to 2 hours. Vital signs will be 
monitored during and after the infusion according to the Study Operations Manual.  
On the day after the infusion (Day 2), patients will receive a physical examination, have vital 
signs collected, and provide blood and urine samples before being discharged or in clinic, as 
applicable.  
Patients will be followed for 48 weeks in Part 1. Patients will complete clinic follow-up visits at 
the following time points: 
relative to the infusion on 
Day 1). In addition, at 
, patients will complete follow-up visits for assessment of 
liver function tests. If the 
 visits occur in the clinic, a blood sample for the 
assessment of glutamate dehydrogenase (GLDH) will be collected.  
All patients will have a muscle biopsy performed at Week 12 in Part 1. 
 
 
 
  
All patients, parents, caregivers, the Examining Team, the Treating Team, physiotherapists, and 
clinical staff will be blinded to the treatment the patient receives (SRP-9001 or placebo). The 
study will be unblinded when the last patient completes the 
 visit. Refer to the 
study unblinding plan for additional details. 
Safety will be assessed by monitoring of treatment-emergent adverse events (TEAEs), serious 
adverse events (SAEs), and select laboratory assessments. 
Refer to Section 6 Study Assessments and Procedures for additional information.  
3.1.2.2. 
Part 2 
Part 2 of the study will begin after the patient completes Part 1. Patients who were randomized 
to placebo during Part 1 will receive IV SRP-9001 (1.33 × 1014 vg/kg) in Part 2. To maintain 
blinding throughout the study, patients previously randomized and treated with SRP-9001 in 
Part 1 will receive up to 10 mL/kg placebo (lactated Ringer’s solution). Infusions will be given 
in the same manner as in Part 1. 
 
 
 
 
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• 
 
• 
 
• 
 
 
 
 
 
Prior to dosing in Part 2, if a patient has rAAVrh74 antibody titers > 1:400, consultation with the 
unblinded contract research organization (CRO) Medical Director, blinded Sponsor Medical 
Director, and PI is required prior to dosing. Patients who received placebo in Part 1 and who 
have rAAVrh74 antibody titers > 1:400 will not be dosed with SRP-9001 nor will they receive 
a second placebo infusion in Part 2. The patient will be encouraged to continue their follow-up 
through the last planned visit based on the protocol. If the patient decides to withdraw from the 
study, they should complete the last planned visit based on the protocol as their final visit.  
The day prior to the study treatment (SRP-9001 or placebo) infusion in Part 2, the patient’s dose 
of steroids will be increased to at least 1 mg/kg of a glucocorticoid (prednisone equivalent) daily 
and continue at this level for at least 60 days after the infusion unless earlier tapering is judged 
by the PI to be in the best interest of the patient. 
 
 
 
 
On the day after the infusion, patients will receive a physical examination, have vital signs 
collected, and provide blood and urine samples before being discharged or in clinic, as 
applicable. 
 
 
 
 
 
 
 
 
  
Safety will be assessed by monitoring of TEAEs, SAEs, and select laboratory assessments. 
Refer to Section 6 Study Assessments and Procedures for additional information. 
3.1.2.3. 
Part 3 
Part 3 of the study will be an open-label follow-up period. The study will be unblinded once the 
last patient completes the 
 visit; refer to the study unblinding plan for additional 
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details. Patients will have the opportunity to complete their 5-year post-infusion follow-up in 
a long-term extension study. The long-term extension study has been designed to provide a 
uniform approach to monitoring long-term safety and efficacy in patients who received an 
infusion of SRP-9001 in a clinical trial.  
Patients must complete the Week 130 visit at a minimum before rolling over into the long-term 
extension study. After completion of the Week 130 visit, if patients decide not to continue their 
follow-up in the long-term extension study, patients will complete an end of study visit. 
3.2. 
Scientific Rationale for Study Design 
Based on the hypothesis that micro-dystrophin expression can improve sarcolemmal integrity 
resulting in improved cardiac and skeletal muscle function as well as preliminary results from the 
ongoing study (Study SRP-9001-101), this study will further assess the safety and efficacy of 
exogenous gene transfer in DMD patients measuring biologic and clinical efficacy endpoints.  
3.3. 
 
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3.4. 
End of Study Definition 
The end of the study is defined as the date of last patient, last visit. Patients will have the 
opportunity to complete their 5-year post-infusion follow-up in a long-term extension study. 
The long-term extension study has been designed to provide a uniform approach to monitoring 
long-term safety and efficacy in subjects who received an infusion of SRP-9001 in a clinical 
trial.  
Patients must complete the Week 130 visit at a minimum before rolling over into the long-term 
extension study. After completion of the Week 130 visit, if subjects decide not to continue their 
follow-up in the long-term extension study, subjects will complete an end of study visit. 
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4. 
STUDY POPULATION 
Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as 
protocol waivers or exemptions, are not permitted. 
4.1. 
Inclusion Criteria 
Participants are eligible to be included in the study only if all of the following criteria apply: 
1. Male, 4 to 7 years of age, inclusive, at the time of screening in Part 1. 
2. Molecular characterization of the DMD gene with either frameshift (deletion or 
duplication) between exons 18 to 58, or premature stop codon mutation between 
exons 18 to 58. 
3. Indication of symptomatic muscular dystrophy:  
  
 
4. Ability to cooperate with motor assessment testing. 
5. Stable dose equivalent of oral corticosteroids for at least 12 weeks before screening and 
the dose is expected to remain constant (except for potential modifications to 
accommodate changes in weight) throughout Part 1 and 2 of the study. 
4.2. 
Exclusion Criteria  
Participants are excluded from the study if any of the following criteria apply: 
1. Signs of cardiomyopathy, including ECHO 
 
2. Serological evidence of HIV infection, or Hepatitis B or C infection. 
3. Diagnosis of (or ongoing treatment for) an autoimmune disease.  
4. Concomitant illness or requirement for chronic drug treatment that in the opinion of the 
PI creates unnecessary risks for gene transfer.  
5. Has a medical condition or extenuating circumstance that, in the opinion of the 
Investigator, might compromise the patient’s ability to comply with the protocol required 
testing or procedures or compromise the patient’s wellbeing, safety, or clinical 
interpretability. 
6. Severe infection (eg, pneumonia, pyelonephritis, or meningitis) within 
 before 
study treatment infusion. 
7. Demonstrates cognitive impairment that could confound motor development in the 
opinion of the Investigator. 
8. Has received any investigational medication (other than corticosteroids) or exon skipping 
medications (including EXONDYS 51®), experimental or otherwise, within 6 months of 
screening.  
9. Has received any type of gene therapy, cell-based therapy (eg, stem cell transplantation), 
or CRISPR/Cas9 therapy. 
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10. Abnormal laboratory values considered clinically significant: 
11. Patients with rAAVrh74 antibody titers > 1:400 as determined by enzyme-linked 
immunosorbent assay (ELISA). 
12. Family does not want to disclose patient’s study participation with primary care physician 
and other medical providers. 
4.3. 
Lifestyle Considerations 
 Every effort should 
be made to collect these samples on the first day of a 2-day study visit; however, per PI 
discretion, the samples may be collected during a 1-day study visit. 
 samples must 
be collected before any functional assessments are performed. Parents or caregivers should be 
asked to limit patient’s physical activity level over the 3 days before a visit where 
 samples 
will be collected. 
4.4. 
Screen Failures 
Screen failures are defined as participants who consent to participate in the clinical study but are 
not subsequently randomly assigned to study intervention.  
Individuals who do not meet the eligibility criteria may be rescreened at the Investigator’s 
discretion.  
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5. 
STUDY INTERVENTION 
Study intervention is defined as any investigational intervention(s), glucocorticoid(s), or placebo 
intended to be administered to a study participant according to the study protocol. 
5.1. 
Study Intervention(s) Administered 
 
Micro-dystrophin 
Placebo 
Corticosteroid 
Intervention Name SRP-9001 gene vector 
Lactated Ringer’s 
solution 
Glucocorticoid 
Type  
Vector 
Placebo 
Immunosuppressant 
Dose Formulation 
IV infusion 
IV infusion 
Oral or IV 
Dose a 
1.33 × 1014 vg/kg  
NA 
1 mg/kg (prednisone 
equivalent) 
Dosage Level(s) 
Single dose  
Single dose 
Day before infusion and 
continuing for at least 
60 days after the infusion 
unless earlier tapering is 
judged by the Principal 
Investigator to be in the best 
interest of the patient 
Route of 
Administration 
IV infusion (peripheral 
limb vein)  
IV infusion (peripheral 
limb vein)  
Oral or IV 
IV = intravenous; NA = not applicable; qPCR = quantitative polymerase chain reaction. 
a For patients who received a dose in this study based on Nationwide Children’s Hospital qPCR method, the dose 
used was 2 × 1014 vg/kg which corresponds to 1.33 × 1014 vg/kg as measured by the Sponsor’s qPCR method using 
a linear standard.  
5.1.1. 
Pre-infusion Immunosuppressants 
The day before the infusion, the patient’s background dose for DMD will be increased to at least 
1 mg/kg of a glucocorticoid (prednisone equivalent). 
5.1.2. 
Post-infusion Immunosuppressants 
Patients will remain on their stable dose of corticosteroids throughout Part 1 and 2 of the study 
but will be increased to at least 1 mg/kg of a glucocorticoid (prednisone equivalent) for at least 
60 days after the infusion unless earlier tapering is judged by the PI to be in the best interest of 
the patient. Additional glucocorticoid may be utilized if GGT level is > 150 U/L or there are 
other clinically significant liver function abnormalities following infusion. A tapering dose of 
glucocorticoid will be implemented based on individual patients’ immune response to the 
infusion, assessed by liver function monitoring with GGT. This monitoring will be done by the 
Treating Team who will also monitor AEs. If there are signs of immune response in specific 

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organ systems like the liver, the Treating Team will closely monitor and make adjustments in 
glucocorticoid dosing. 
5.1.3. 
Intravenous Infusion (Day 1, Part 1 and Part 2)  
SRP-9001 or placebo administration will be through a peripheral limb vein. Administration will 
be performed according to the Site-Specific Pharmacy and/or Dose Administration Manual. 
5.1.4. 
Post Infusion Monitoring 
5.1.4.1. 
Immediately Following Infusion 
Vital signs will be monitored during and after the infusion according to the Study Operations 
Manual. Concomitant medications and all AEs and SAEs will also be monitored and documented 
following injection.  
5.1.4.2. 
Extended Follow-up 
Patients will return for clinic follow-up visits as outlined in the Schedule of Activities. Patients 
must complete the Week 130 visit at minimum before rolling over into the long-term extension 
study. After completion of the Week 130 visit, if patients decide not to continue their follow-up 
in the long-term extension study, patients will complete an end of study visit.  
 
 
 
 
 Unscheduled visits and additional blood draws may 
occur if the Investigator determines they are necessary for the patients’ safety and wellbeing.  
5.2. 
Preparation/Handling/Storage/Accountability 
Compliance standards for Biosafety Level 1 vectors must be followed according to standard 
pharmacy protocol for handling investigational products similar to SRP-9001.  
5.3. 
Measures to Minimize Bias: Randomization and Blinding 
5.3.1. 
Establish Patient Identification Number 
All patients will be given a unique sequentially assigned patient identifier. Patients will be 
identified only by this number in order to protect identity. 
5.3.2. 
Assignment to a Treatment Group 
This is a double-blind study. Patients who have signed the informed consent and who meet all 
eligibility will be randomized using the IXRS to 1 of the 2 treatment groups prior to or on Day -1 
in Part 1. 
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5.3.3. 
Randomization 
Patients will be randomized in a 1:1 ratio by the IXRS in Part 1 of the study to receive either 
SRP-9001 or placebo. Patients randomized to SRP-9001 in Part 1 of the study will receive 
placebo in Part 2. Patients randomized to placebo in Part 1 of the study will have the opportunity 
to receive SRP-9001 in Part 2.  
Randomization will be stratified by age group at Baseline (4-5 vs 6-7 years). To ensure 
approximately equal numbers of patients are assigned to the 2 treatment groups (SRP-9001 vs 
placebo) in Part 1, each age group must have a multiple of 4 patients. 
5.3.4. 
Blinding/Unblinding 
All patients and study staff will be blinded to treatment assignments with the exception of the 
pharmacist until the last patient completes the 
 visit. The blind of the treatment 
may be broken only in exceptional circumstances, such as when knowledge of the study 
treatment is essential for treating a patient due to an SAE. If time permits, the Investigator must 
contact the unblinded CRO Medical Director before unblinding of the patient. If time does not 
permit, the Investigator may authorize breaking of the blind and then notify the unblinded CRO 
Medical Director. The study treatment information should be disclosed only to personnel who 
need the information for the medical care of the patient. If the study treatment is unblinded, the 
patient number, time, date, and reason for unblinding must be recorded in the study records. 
Study personnel who were unblinded should be identified. Refer to the study unblinding plan for 
further details regarding the unblinding of the study for Part 3. 
Separate study personnel will be designated to conduct functional assessments and to treat 
patients in order to protect against possible unblinding of treatment assignment during regular 
clinical care of patients. 
The PI will designate the following investigational site personnel: 
• An Examining Team 
• A Treating Team 
The Examining Team will be: 
• Composed of physiotherapists trained in the proper execution of the physical functional 
assessments (NSAA, 10-meter timed test, 100-meter timed test, time to rise from floor, 
and time to ascend 4 steps) 
The Treating Team will be: 
• Composed of clinicians who will be responsible for the following: 
− Administering the study treatment 
− Conducting the patient’s follow-up visits but not the physical functional 
assessments at those visits per the SoA (Section 1.3) 
− Review of all clinical and safety laboratory results 
− Glucocorticoid dose adjustments 
− Assessment of AEs, SAEs, and TEAEs 
− Management of the routine care of the patient 
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The physiotherapists from the Examining Team will be responsible for conducting the physical 
functional assessments (NSAA, 10-meter timed test, 100-meter timed test, time to rise from 
floor, and time to ascend 4 steps). They will not have access to any patient data and must be 
restricted from having access to AEs, concomitant medications, laboratory data, and any other 
data that have the potential of revealing the treatment assignment. The physiotherapists should be 
instructed not to have conversations with the patients and parents/caregivers about their progress 
in the study. 
The Treating Team members will be responsible for the clinical care of the patient. They will 
manage the study drug infusion, steroid dosing changes, review all laboratory data, assess AEs 
and SAEs, and perform all assessments except for the physical functional assessments (NSAA, 
10-meter timed test, 100-meter timed test, time to rise from floor, and time to ascend 4 steps). 
The Treating Team will not have access to the data collected by the Examining Team. 
The study will be unblinded once the last patient completes the 
 visit; refer to the 
study unblinding plan for further details.  
5.4. 
Concomitant Therapy 
To be collected from the time of consent until final study visit, recorded on the case report form 
(CRF). 
Any medication or vaccine (including over the counter or prescription medicines, vitamins, 
and/or herbal supplements) that the participant is receiving at the time of enrollment or receives 
during the study must be recorded in the CRF. 
If a reaction to the infusion occurs, the Treating Team should treat the reaction. The date and 
time of medication administration as well as the name and dosage regimen must be recorded. 
5.4.1. 
Rescue Medicine 
There are no specific rescue medications for use in this study that will turn off SRP-9001 
transgene expression. Corticosteroid therapy, at the PI’s discretion, may be used to manage 
possible liver-associated adverse effects of SRP-9001.  
5.5. 
Dose Modification 
5.5.1. 
Stopping Rules 
Study enrollment will be interrupted when any patient experiences 1 or more Grade 3 (severe) or 
higher AEs that are unexpected as related to gene therapy or DMD. This will include any patient 
death, important clinical laboratory findings, or any severe local complication in the injected area 
related to administration of the study agent. The findings will be reported to the data monitoring 
committee (DMC). If after review by the DMC, the recommendation is made to continue, the 
Sponsor may decide that the study will proceed.  
5.5.2. 
Dosing Schedule 
Based on the safety profile of systemic gene delivery clinical trial (Study SRP-9001-101) for 
DMD using SRP-9001, there will be at least a 1-week interval between dosing each patient for 
the first 4 patients.  
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5.6. 
Study Discontinuation 
If the Sponsor, Investigator, Medical Monitor, DMC, Study Monitor, IRB/IEC, and/or 
appropriate regulatory officials discover conditions arising during the study that indicate the 
study should be halted or that the study center should be terminated, appropriate action may be 
taken after consultation among the Sponsor, Investigator, and others as needed. 
Conditions that may warrant termination of the study or an individual site include, but are not 
limited to, the following: 
• The discovery of an unexpected, serious, or unacceptable risk to subjects enrolled in 
the study 
• A decision by the Sponsor to suspend or discontinue testing, evaluation, or 
development of the study drug or stop the study 
• Inability of the Investigational site to identify potential subjects for the clinical trial  
• Failure of the Investigator to comply with pertinent regulations of IRB/IEC or 
appropriate regulatory authorities 
• Failure of the Investigator to comply with the protocol 
• Submission of knowingly false information from the research facility to the Sponsor, 
the Study Monitor, IRB/IEC, or regulatory authority 
• Insufficient adherence or noncompliance with the clinical study protocol and global 
GCP regulations, directives, guidelines, and laws such as the Pharmaceutical and 
Medical Device Act, and the clinical study protocol as applicable. 
Study termination and follow-up will be performed in compliance with the conditions set forth in 
International Council for Harmonisation (ICH) E6 on Good Clinical Practice (GCP) as well as 
21 CFR 312.56b and other applicable national/local regulations, which require a Sponsor to 
ensure an Investigator’s compliance with these requirements and to promptly secure a plan for 
compliance or discontinue shipments of the study drug to the Investigator and end the 
Investigator’s participation in the study. 
If the Sponsor terminates the study for any reason, if possible, an early termination visit should 
be conducted, as shown in the SoA (Section 1.3). See the SoA (Section 1.3) for data to be 
collected at the time of study discontinuation or termination and for any further evaluations that 
need to be completed. 
5.7. 
Intervention after the End of the Study 
There will be no intervention following the end of the study. 

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6. 
STUDY ASSESSMENTS AND PROCEDURES 
• Study procedures and their timing are summarized in the SoA (Section 1.3). Protocol 
waivers or exemptions are not allowed. 
• Immediate safety concerns should be discussed with the Sponsor immediately upon 
occurrence or awareness to determine if the participant should continue or 
discontinue study intervention. 
• Adherence to the study design requirements, including those specified in the SoA, is 
essential and required for study conduct. 
• All screening evaluations must be completed and reviewed to confirm that potential 
participants meet all eligibility criteria. The Investigator will maintain a screening log 
to record details of all participants screened and to confirm eligibility or record 
reasons for screening failure, as applicable. 
• Procedures conducted as part of the participant’s routine clinical management 
(eg, blood count) and obtained before signing of the informed consent form (ICF) 
may be utilized for screening or baseline purposes provided the procedures met the 
protocol-specified criteria and were performed within the time frame defined in the 
SoA. 
6.1. 
Efficacy Assessments  
Planned time points for all efficacy assessments are provided in the SoA (Section 1.3). 
6.1.1. 
Muscle Biopsy  
A muscle biopsy will be performed at Screening/Baseline after establishing eligibility. Muscle 
biopsies will be used to quantify transgene expression by Western blot comparing the baseline 
biopsy with the biopsies collected at Week 12 in Part 1 
 
. Additional information can be found in the 
Biopsy Manual. 
6.1.2. 
Immunofluorescence Fiber Intensity / Immunofluorescence PDPF 
Muscle biopsies will be used to quantify transgene expression by immunofluorescence (IF) fiber 
intensity and IF percent dystrophin positive fibers comparing the baseline biopsy with both the 
biopsies collected at Week 12 in Part 1 
.  
6.1.3. 
 
 
 
6.1.4. 
 
 
 
 
  
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6.1.5. 
Physical Functional Assessments 
Efficacy will be measured by the following functional tests: time to rise from the floor, ascend 
4 steps, NSAA, 10-meter timed test, and 100-meter timed test. Assessments will be administered 
by a physiotherapist. The physiotherapist will not have access to information that can disclose 
study treatment, laboratory results, or adverse events. 
The patient’s functional assessments will be videotaped for quality control and may be viewed 
by government agencies if the study goes through a quality review. These videos may also be 
used to demonstrate any functional outcomes related to the study drug and shared at scientific 
meetings and/or with regulatory authorities. 
6.1.5.1. 
Time to Rise from the Floor 
This assessment is performed as a part of the NSAA. It measures the time taken to rise from 
supine to standing. 
6.1.5.2. 
Ascend 4 Steps 
This assessment measures the time taken to climb 4 steps. 
6.1.5.3. 
North Star Ambulatory Assessment 
The NSAA is a healthcare provider administered scale that rates patient performance on various 
functional activities (Mazzone 2010). During this assessment, patients will be asked to perform 
17 different functional activities, including a 10-meter walk/run, rising from a sitting position to 
standing position, standing on 1 leg, climbing a box step, descending a box step, rising from 
lying to sitting, rising from the floor, while supine lifting head off floor, standing on heels, and 
jumping.  
6.1.5.4. 
10-meter Timed Test 
This assessment measures the time needed to move 10 meters. 
6.1.5.5. 
100-meter Timed Test 
This assessment measures the time needed to move 100 meters. 
6.2. 
Safety Assessments 
Planned time points for all safety assessments are provided in the SoA (Section 1.3). 
Safety parameters evaluated during the conduct of the study include AEs, SAEs, TEAEs, clinical 
laboratory values, antibody titers, vital signs, ECGs, ECHOs, physical examination findings, and 
use of concomitant medications.  
6.2.1. 
Physical Examinations 
Physical examinations will be conducted at the time points specified in the SoA (Section 1.3). 
Physical examinations will include examination of general appearance, head, ears, eyes, nose, 
and throat, heart, chest, abdomen, skin, lymph nodes, extremities, musculoskeletal, and 
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Any physical examination finding assessed as clinically significant should be recorded as an AE 
or SAE. If SAE criteria are met, the event should be recorded and reported according to the SAE 
reporting process (see Section 6.3). 
6.2.2. 
Vital Signs 
Vital signs (blood pressure, heart rate, respiration, and oral temperature) will be measured at the 
time points specified in the SoA (Section 1.3). 
Vital signs including blood pressure, heart rate, respiratory rate, and temperature will be 
performed after study treatment infusion according to the Study Operations Manual.  
Any vital signs assessed as clinically significant should be recorded as an AE or SAE. If SAE 
criteria are met, the event should be recorded and reported according to the SAE reporting 
process (see Section 6.3). 
6.2.3. 
Electrocardiograms and Echocardiograms 
Single ECGs will be obtained at the time points specified in the SoA (Section 1.3) using an ECG 
machine that automatically calculates the heart rate and measures PR, QRS, and QT intervals. 
ECGs will be performed at a consistent time of day throughout the study. The Investigator, or 
designee, will review the results of the ECG report and determine if the findings are clinically 
significant. 
A standard 2-dimensional ECHO will be obtained at the time points specified in the SoA 
(Section 1.3). Echocardiograms will be performed at a consistent time of day throughout the 
study and before any invasive procedures (eg, blood sampling, study drug infusion, or biopsy). 
The ECHO will be reviewed and interpreted by local medically qualified personnel. Left 
ventricular ejection fraction will be noted. The Investigator, or designee, will review the results 
of the ECHO report and determine if the findings are clinically significant.  
Clinical significance is defined as any variation in assessment results that has medical relevance 
resulting in an alteration in medical care. If clinically significant deterioration from baseline 
levels is noted, the Investigator will continue to monitor the patient with additional assessments 
until:  
• Values have reached normal range and/or baseline levels; or  
• In the judgment of the Investigator together with the Sponsor’s Medical Monitor, 
abnormal values are assessed to be not related to the administration of study treatment 
or other protocol-specific procedures, and additional assessments are not medically 
indicated. In the judgment of the Investigator, an unexplained laboratory abnormality 
that could potentially lead to a poor outcome should result in interruption of 
treatment. 
If assessed as clinically significant, the event should be recorded as an AE or SAE as required in 
Section 6.3. 
6.2.4. 
Clinical Safety Laboratory Assessments 
The following routine clinical laboratory tests will be performed at the time points specified in 
the SoA (Section 1.3). Samples will be collected and processed according to the local laboratory 

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procedures, the Study Operations Manual, and/or the Central Laboratory Manual provided for 
the study: 
Chemistry: 
Sodium, chloride, potassium, carbon dioxide, glucose, creatinine, 
blood urea nitrogen, total protein, albumin, total bilirubin, alkaline 
phosphatase, amylase, ALT, AST, GGT, lactate dehydrogenase, 
C-reactive protein, 
 and serum cystatin C, complement (CH50, 
C3, C4, and factor B), and GLDH 
Note: CH50 may be collected and analyzed by the clinical site; 
however, C3, C4, and factor B will be collected and analyzed by the 
central laboratory only. 
Note: The GLDH assay will be performed by a central laboratory and 
only at clinic visits. 
Hematology: 
Red blood cells, total WBCs, hemoglobin, hematocrit, neutrophils, 
lymphocytes, monocytes, eosinophils, basophils, platelets, and 
abnormal cells 
Coagulation Screen: 
Prothrombin time, partial thromboplastin time, and international 
normalized ratio  
 
 
 
Urinalysis: 
pH, specific gravity, protein, glucose, ketones, cytology, and 
hemoglobin 
Any laboratory abnormality deemed clinically significant by the Investigator should be recorded 
as an AE. A clinically significant abnormality is an abnormality confirmed by repeat testing, that 
in the judgment of the Investigator warrants a change in management. This alteration may 
include monitoring the laboratory test further, initiating other diagnostic tests or procedures, 
changing ongoing treatment, or administering new treatment(s).  
Whenever possible, the underlying medical diagnosis (eg, anemia) should be recorded as the AE 
term. Repeated additional tests and/or other evaluations required to establish the significance and 
etiology of an abnormal result should be obtained when clinically indicated. 
Patients enrolled under this clinical protocol are expected to present clinically with adverse 
events related to natural progression of the disease. The draft guidance entitled “Duchenne 
Muscular Dystrophy Developing Drugs for Treatment over the Spectrum of Disease” 
(https://www.regulations.gov/document?D=FDA-2014-D-1264-0002) provides the basis of 
expected disease-related AEs. The expectedness of all AEs will be determined according to the 
most recent versions of the Investigator’s Brochure (Section 6.3.3.7). 
Asymptomatic elevations in transaminases are a feature of Duchenne muscular dystrophy, as 
they are related to release of AST and ALT from muscle tissue and correlate with levels of CK. 
These are a feature of the disease itself. Levels of AST and ALT up to 10× the ULN are expected 
to be seen at Baseline and are not indicative of muscle or liver injury. Elevated AST/ALT levels 
are thus not exclusionary for enrollment and, depending on circumstances, will not be recorded 
as adverse events during the course of the trial.  
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6.2.5. 
Safety Monitoring 
6.2.5.1. 
Safety Monitoring for Liver Chemistry Tests 
Liver chemistry tests need to be monitored as specified in the SoA (Section 1.3). Initial abnormal 
liver chemistry test result(s) need to be confirmed if: 
• GGT or GLDH is 
× ULN at any time during the study 
• AST or ALT measurement is 
× baseline value if the baseline value is >ULN;  
Patients with confirmed liver chemistry test results (as above) should have their liver tests 
 
 
 retested at a minimum of 
 and additional testing 
beyond that is at the discretion of the Investigator. Frequency of retesting can decrease to once a 
week or less if abnormalities stabilize and the patient is asymptomatic. 
Additional Investigations 
Patients with confirmed abnormal liver chemistry test results (as above) are recommended to 
have the following evaluations performed:  
• Obtaining a more detailed history of symptoms and prior or concurrent diseases  
• Obtaining a history of concomitant drug use (including nonprescription medications 
and herbal and dietary supplement preparations), alcohol use, recreational drug use, 
and special diets  
• Ruling out acute viral hepatitis types A, B, C, D, and E; autoimmune hepatitis; 
Non-Alcoholic Steato Hepatitis; hypoxic/ischemic hepatopathy; and biliary tract 
disease  
• Consideration of other viral illnesses that have been associated with hepatitis (eg, 
Epstein-Barr virus, cytomegalovirus, Human Herpesvirus 6) 
• Obtaining a history of exposure to environmental chemical agents  
Additional liver evaluations, including 
 
 may be performed at the discretion of the Investigator, in 
consultation with the Sponsor Medical Monitor. 
Refer to Section 6.3.6.2 for the list of adverse events of special interest (AESIs) related to liver 
chemistry tests. 
6.2.5.2. 
Safety Monitoring for Hypersensitivity and Complement-Mediated Reactions 
Patients will be monitored for hypersensitivity and complement-mediated reactions as follows: 
• Hypersensitivity: Patients will be monitored for occurrence of allergic reactions by 
monitoring AEs as specified in the SoA (Section 1.3). Patients will be instructed to 
promptly report any signs or symptoms of fever or constitutional symptoms that may 
arise during the study, and the Investigator needs to closely evaluate all potential 
causes, including concomitant illness.  
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• Complement-mediated reactions: Patients will be monitored for occurrence of 
complement-mediated reactions by monitoring AEs as specified in the SoA 
(Section 1.3). In addition to monitoring AEs, routine laboratory monitoring for 
complement levels (CH50, C3, C4, and factor B) will be performed as outlined in the 
SoA (Section 1.3). 
Additional Investigations 
• Hypersensitivity: Patients who experience significant or persistent constitutional 
symptoms, including any Grade ≥ 3 hypersensitivity reactions, need to be discussed 
with the Sponsor Medical Monitor to determine whether additional monitoring or 
laboratory tests are required. Additional evaluations including 
 
 
 may be performed at the discretion of the 
Investigator in consultation with the Sponsor Medical Monitor. 
• Atypical hemolytic uremic syndrome (aHUS): Patients diagnosed with aHUS need to 
be discussed with the Sponsor Medical Monitor to determine additional monitoring 
and laboratory testing. Additional evaluations including 
 
 
 
 
 
 
 
 may be performed at the discretion of the 
Investigator in consultation with the Sponsor Medical Monitor. 
Refer to Section 6.3.6.2 for the list of AESIs related to hypersensitivity. 
6.2.5.3. 
Safety Monitoring for Platelet Count Results 
Platelet counts will be monitored according to the SoA (Section 1.3). Patients with a confirmed 
occurrence of platelets 
 need to have the following evaluations performed: 
• 
 
• 
 
• 
 
 
• 
 
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Additional Investigations 
Additional platelet evaluations for confirmed, unexplained significant platelet count reductions, 
 
 
 may be 
performed at the discretion of the Investigator in consultation with the Sponsor Medical Monitor. 
Refer to Section 6.3.6.2 for the list of AESIs related to thrombocytopenia. 
6.2.5.4. 
Safety Monitoring for Rhabdomyolysis 
Rhabdomyolysis will be monitored by urine dipstick and assessments of AEs per the time points 
in the SoA (Section 1.3). Patients who have confirmed heme+ dipstick urinalysis need to be 
evaluated by urine microscopy and for the following AEs: 
Additional Investigations 
In case of rhabdomyolysis, myoglobinuria, or chromaturia, patients will need to have evaluations 
of 
 
 
 these 
evaluations should be undertaken at the discretion of the Investigator. 
In addition, Investigators should obtain a more detailed history of symptoms, preceding activity 
and hydration status, concomitant drug use, and recent or concurrent infections. Additional 
evaluations, 
 may 
be performed at the discretion of the Investigator in consultation with the Sponsor Medical 
Monitor. 
Refer to Section 6.3.6.2 for the list of AESIs related to rhabdomyolysis.  
6.2.6. 
 
 
 
  
 
 
 
 
 
  
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6.2.7. 
 
 
 
 
  
 
6.2.8. 
 
 
 
 
 
 
6.3. 
Adverse Events and Serious Adverse Events 
6.3.1. 
Collection of Adverse Events 
Over the entire duration of the study, site personnel will ensure that all AEs are recorded 
appropriately. If an AE occurs, the primary concern is for patient safety, and the Investigator will 
use his/her judgment and expertise to determine the appropriate course of action. 
All AEs from the time of informed consent through the last follow-up visit will be recorded in 
each enrolled patient’s CRF. For consented patients who are found to be ineligible for the study 
during the Screening period and are not enrolled (ie, Screening failures), only SAEs will be 
collected. 
If, at any time after the patient has completed participation in the study, the Investigator or study 
staff becomes aware of an SAE that the Investigator believes is possibly/probably or definitely 
related to the investigational product (IP) or is possibly/probably or definitely related to a study 
procedure, then the event and any known details must be reported promptly to the Sponsor. 
6.3.2. 
Definition of Adverse Events 
6.3.2.1. 
Adverse Event 
An AE is any untoward medical occurrence in a clinical trial participant, which does not 
necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any 
unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs 
during or after administration of an IP whether or not considered related to the IP. 
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Adverse events include: 
• Symptoms described by the patient or signs observed by the Investigator or medical 
staff. 
• Test abnormalities (laboratory tests, ECG, X-rays, etc.) that result in an alteration in 
medical care (diagnostic or therapeutic). 
Abnormalities present at Screening are considered AEs only if they reoccur after resolution or 
worsen during the AE collection period. 
6.3.2.2. 
Serious Adverse Events 
An SAE is defined as any AE that results in any of the following: 
• Death: The patient died as the result of the event. 
• Life-threatening event: Any AE that places the patient, in the view of the 
Investigator or Sponsor, at immediate risk of death from the AE as it occurred, 
ie, does not include an AE that had it occurred in a more severe form, might have 
caused death. 
• Required or prolonged inpatient hospitalization: The AE resulted in 
hospitalization or prolonged an existing hospitalization. Since hospitalization may be 
part of the study, only hospitalizations that are longer than expected per protocol 
procedures, based on Investigator judgment, will be considered prolonged 
hospitalizations. 
• Persistent or significant disability/incapacity: An AE that results in persistent or 
significant disability or disruption of a person’s ability to conduct normal life 
functions. 
• Important medical events: An AE that may not result in death, be life-threatening, 
or require hospitalization may be considered an SAE when, based upon appropriate 
medical judgment, the event may jeopardize the patient and may require medical or 
surgical intervention to prevent one of the outcomes listed above.  
6.3.2.3. 
Adverse Events of Special Interest 
An AESI is any AE (serious or nonserious) that is of scientific and medical interest specific to 
the study treatment for which ongoing and/or rapid communication by the Investigator to the 
Sponsor is appropriate. See Section 6.3.6.2 for the list of AESIs for this study. 
6.3.3. 
Classification of Adverse Events 
Each AE whether serious or non-serious will be classified by the Investigator according to the 
following rules and definitions. 
6.3.3.1. 
Relationship to Investigational Product 
For each AE, the Investigator will determine whether there is a reasonable likelihood that the AE 
may have been caused by the study treatment according to the categories below: 

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Unrelated: 
The event is clearly not related to the study treatment. 
Possibly/probably related: 
The event could be related/is likely to be related to the 
study treatment. 
Definitely related: 
The event is clearly related to the study treatment. 
6.3.3.2. 
Relationship to Study Procedures 
For each AE the Investigator will determine whether there is a reasonable possibility that the AE 
may have been caused by the study procedures according to the categories below: 
Unrelated: 
The event is clearly not related to the study procedures. 
Possibly/probably related: 
The event could be related/is likely to be related to study 
procedures. 
Definitely related: 
The event is clearly related to the study procedures. 
6.3.3.3. 
Relationship to Underlying Disease 
For each AE the Investigator will determine whether there is a reasonable possibility that the AE 
may be related to the underlying disease according to the categories below: 
Unrelated: 
The event is clearly not related to the underlying disease 
Possibly/probably related: 
The event could be related/is likely to be related to the 
underlying disease 
Definitely related: 
The event is clearly related to the underlying disease 
Events of disease progression may be considered AEs, based on the Investigator’s discretion. 
6.3.3.4. 
Severity of Adverse Events 
Note that severity is not the same as “seriousness”. 
The Investigator will assess the severity of all AEs as Mild, Moderate, or Severe, based on the 
following definitions: 
Mild: 
The event does not interfere with the patient’s usual activities. 
Moderate: 
The event interferes with the patient’s usual activities. 
Severe: 
The event prevents the patient from undertaking their usual activities and 
requires therapeutic intervention or cessation of the study treatment. 
6.3.3.5. 
Outcome 
An outcome describes the status of the AE. The Investigator will provide information regarding 
the patient outcome of each AE. Outcome categories will include recovered, recovered with 
sequelae, not recovered, fatal, and unknown. 

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6.3.3.6. 
Action Taken Regarding the Investigational Product 
The Investigator will provide information regarding the action taken with respect to the study 
treatment in response to the AE. Categories for action taken regarding study treatment will 
include none, drug interrupted, drug withdrawn, and not applicable. 
6.3.3.7. 
Expectedness of an Adverse Event 
The expectedness of all AEs will be determined according to the most recent versions of the 
Investigator’s Brochure. 
6.3.3.8. 
Suspected Unexpected Serious Adverse Reactions  
Suspected unexpected serious adverse reactions (SUSARs) will be reported within the required 
timelines in an unblinded fashion to regulatory authorities and Institutional Review Board 
(IRB)/Independent Ethics Committee (IEC) per the requirements of the concerned competent 
authorities. SUSARs will also be reported in a blinded fashion to study Investigators 
6.3.4. 
Recording of Adverse Events 
All AEs from the time of informed consent/assent through the last follow-up visit will be 
recorded in each enrolled patient’s CRF. For patients who are found to be ineligible for the study 
during the Screening period and are not enrolled (ie, Screening failures), only SAEs will be 
recorded. 
Information should include: a concise description of the event; date of event onset and 
resolution; determination of seriousness, severity, corrective treatment (if any), outcome, and 
relationship to IP or study procedure or underlying disease; and any action taken will be 
recorded. Resolution occurs when the patient has returned to his baseline state of health or 
further improvement or worsening of the event is not expected. 
Whenever possible, a diagnosis will be recorded as an AE, rather than symptoms or isolated 
laboratory abnormalities related to that diagnosis. Several symptoms or laboratory results that are 
related to the same diagnosis can thus be part of the same AE. A medical or surgical procedure is 
not an AE; rather the condition leading to the procedure should be recorded as the AE. Similarly, 
death is not an AE, but is rather the outcome of the AE(s) that resulted in death. If the AE(s) 
leading to death are not known, then death must be reported as an AE. All AEs will be followed 
until the resolution of AE, completion of the patient’s study participation, or study termination, 
whichever occurs first. SAEs will be followed until resolution or until the condition stabilizes or 
returns to baseline status. 
6.3.5. 
Reporting Serious Adverse Events 
It is the responsibility of the Investigator that reporting is done adequately. In order to meet 
Regulatory reporting timelines, the study site is obligated to report any SAE(s) to the Sponsor or 
designee immediately and no later than 24 hours after receiving information of an event that 
meets at least one of the criteria for seriousness. 

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6.3.6. 
Special Situations 
6.3.6.1. 
Overdose 
Refer to Section 6.4 for the definition of treatment overdose. An overdose is not an AE. An 
overdose will be reported even if it does not result in an AE. An overdose will be recorded on the 
Clinical Trial Safety Reporting Form and sent to the Sponsor or designee within 24 hours. 
6.3.6.2. 
Adverse Events of Special Interest 
All AESIs need to be reported to the Sponsor via email to 
 within 24 hours 
of awareness, irrespective of the seriousness of the AE. In this study, the following AEs are 
considered of special interest: 
Hepatotoxicity 
Hypersensitivity 
Thrombocytopenia 
Rhabdomyolysis 
6.3.6.3. 
Death 
Death is an outcome of an event. All causes of death are SAEs. In the event of death, every effort 
should be made to obtain a death certificate and if possible, an autopsy report. If the cause of 
death is unknown, death will be recorded as the event. 
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6.3.6.4. 
Unblinding due to a Medical Emergency 
In the event of a medical emergency, such as when knowledge of the patient’s treatment 
assignment is essential for treating a patient due to an SAE, the Investigator has the option to 
unblind treatment assignment through the IXRS. If time permits, the Investigator must contact 
the unblinded CRO Medical Director before the unblinding of the patient. If time does not 
permit, the Investigator may authorize breaking of the blind, and then notify the unblinded CRO 
Medical Director. 
The reasons for unblinding and the study personnel who were unblinded must be noted in the 
source documentation. The Investigator must not disclose information about treatment 
assignment to anyone who does not need the information for patient care. Disposition of patients 
who become unblinded due to medical emergency will be determined following discussion with 
the Sponsor. 
The study will be unblinded once the last patient completes the 
 visit; refer to the 
study unblinding plan for further details. 
6.3.7. 
Responsibilities of the Investigator 
The responsibilities of the Investigator include but are not limited to the following: 
• Monitor and record all AEs. 
• Determine seriousness, severity, and relationship to IP and/or study procedure and/or 
underlying disease. 
• Determination of the onset and end date of each event. 
• Provide initial report on all SAEs within 24 hours of first knowledge to the Sponsor 
via email to 
 or designee. 
• Provide follow-up information on SAEs in a timely and proactive manner. 
• Respond to queries regarding AEs and SAEs in a timely manner. 
• Ensure source documentation for all AEs are accurate and complete. 
• Ensure that the study is conducted as defined in this protocol. 
Investigators may also report improvement of pre-existing DMD conditions or unexpected 
therapeutic responses. 
6.3.8. 
Responsibilities of the Sponsor 
The responsibilities of the Sponsor (Sarepta and designees) include, but are not limited to the 
following: 
• Training of Investigator and site staff on AE/SAE definitions, safety assessments, and 
site obligations related to safety monitoring and reporting of AE/SAEs. 
• Accurate and timely reporting of SUSARs to all applicable regulatory bodies, clinical 
trial Investigators, and other parties as appropriate and required within the regulated 
timelines. 
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• Ensuring accurate recording of AEs and SAEs.  
• Notification of expedited SUSARs to sites. 
• Annual safety reporting to regulatory authorities and IRBs/IECs according to regional 
requirements. 
6.4. 
Treatment Overdose 
For this study, any dose of SRP-9001 
 for a specific patient 
will be considered an overdose. 
There is no recommended specific treatment for an overdose. 
In the event of an overdose, the Investigator should: 
1. Contact the CRO’s unblinded Medical Director immediately. 
2. Closely monitor the participant for any AE/SAE and laboratory abnormalities until 
resolution. 
3. Document the quantity of the excess dose as well as the duration of the overdose on the 
Overdose Form and send to the Sponsor immediately. The overdose should also be 
entered in the CRF. 
4. Provide appropriate medical care to patient based on clinical judgement. 
Decisions regarding dose interruptions or modifications will be made by the Investigator in 
consultation with the CRO’s unblinded Medical Director based on the clinical evaluation of the 
participant. 
6.5. 
Pharmacokinetics 
Pharmacokinetic parameters are not being evaluated in this study. 
6.6. 
Pharmacodynamics 
Creatine kinase levels will be measured at time points according to the SoA (Section 1.3). 
Muscle biopsies will be performed at Screening/Baseline and at Week 12 in Part 1 
 
. 
6.7. 
 
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6.7.1. 
 
6.7.2. 
 
6.8. 
Biomarkers 
The collection of muscle biopsy samples 
 to assess micro-dystrophin 
 will be obtained; however, they are considered efficacy endpoints and described 
in Section 6.1.  
6.9. 
 
6.9.1. 
 
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6.9.2. 
 
 
 
 
 
 
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7. 
STATISTICAL CONSIDERATIONS 
7.1. 
Statistical Hypotheses 
The study has 2 primary endpoints: change in quantity of micro-dystrophin protein expression 
from Baseline to Week 12 (Part 1) as measured by Western blot and change in NSAA total score 
from Baseline to Week 48 (Part 1). Each primary endpoint will be tested for statistical 
significance based on a multiplicity-adjusted testing procedure that will control the overall 
Type I error rate at a 2-sided level of 0.05. 
 
 
 
 
 
 Additional details will be specified in the Statistical Analysis Plan (SAP). 
The statistical analyses of endpoints in Part 2 will also be described in the SAP. 
For the primary endpoint of change in quantity of micro-dystrophin protein expression from 
Baseline to Week 12 as measured by Western blot, the null hypothesis is that treatment 
assignment has no effect on the endpoint, and the alternative hypothesis is that treatment 
assignment affects the endpoint. Even though the alternative hypothesis is 2-sided, only 
superiority of SRP-9001 over placebo will be of interest.  
The statistical hypotheses for other micro-dystrophin expression endpoints can be stated in a 
similar manner as those for change in quantity of micro-dystrophin protein expression from 
Baseline to Week 12 as measured by Western blot. 
For the primary efficacy endpoint of change in NSAA total score from Baseline to Week 48 
(Part 1), the null hypothesis is that the population means for the 2 treatments are equal and the 
alternative hypothesis is that the population means for the 2 treatments are not equal. Even 
though the alternative hypothesis is 2-sided, only superiority of SRP-9001 over placebo will be 
of interest.  
The statistical hypotheses for secondary endpoints on physical function can be stated in a similar 
manner as those for change in NSAA total score from Baseline to Week 48 (Part 1). 
7.2. 
Sample Size Determination 
A dramatic treatment difference is expected for the primary endpoint of change from Baseline 
to Week 12 in quantity of micro-dystrophin protein expression as measured by Western blot. 
Therefore, the sample size is based on the power for the primary efficacy endpoint of change 
in NSAA total score from Baseline to Week 48 (Part 1) with Type I error of 0.05 (2-sided), 
assuming the alpha allocated for the other primary endpoint will be recycled. 
 
 
 
 
 
 
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7.3. 
Analysis Populations 
For purposes of statistical analyses, the following analysis populations are defined: 
Population 
Description 
Intent-to-Treat (ITT) 
All randomized patients who received study treatment during Part 1, 
with treatment group designated according to randomization. The ITT 
population will be the main analysis population for endpoints for 
Part 1. 
Per-Protocol  
All patients in the ITT population who do not have important protocol 
deviations that may substantially affect the study results. Eligibility for 
the Per-Protocol population will be determined prior to breaking the 
study blind.  
Safety 
All patients who received study treatment during Part 1 or Part 2, with 
treatment group designated according to the treatment that they 
actually received.  
7.4. 
Statistical Analyses 
An SAP will be developed and finalized before database lock and unblinding of the study data. It 
will describe the details of the statistical analyses. This section is a summary of the planned 
statistical analyses of the primary and secondary endpoints as well as the safety analyses.  
7.4.1. 
Efficacy Analyses 
The study has 2 primary endpoints: change in quantity of micro-dystrophin protein expression 
from Baseline to Week 12 (Part 1) as measured by Western blot and change in NSAA total score 
from Baseline to Week 48 (Part 1). Each primary endpoint will be tested for statistical 
significance based on the multiplicity-adjusted testing procedure that will control the overall 
Type I error at a 2-sided level of 0.05. 
Endpoint 
Statistical Analysis Methods 
Primary  
For the primary endpoint of change in quantity of micro-dystrophin protein 
expression from Baseline to Week 12 (Part 1) as measured by Western blot, 
summary statistics will be provided by treatment group for Baseline, Week 12, and 
change from Baseline to Week 12 for quantity of micro-dystrophin protein 
expression as measured by Western blot.  
 
 
 
 
 
  
For the primary efficacy endpoint of change in NSAA total score from Baseline to 
Week 48 (Part 1), summary statistics will be provided by treatment group for 
Baseline, each post-Baseline visit, and change from Baseline to each post-Baseline 
visit.  
A mixed model with repeat measures or another test deemed appropriate, to be 
specified in the SAP, will be performed to compare the treatment groups for change 
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Endpoint 
Statistical Analysis Methods 
in NSAA total score from Baseline to Week 48 (Part 1). The superiority of SRP-9001 
over placebo will be concluded if the test achieves statistical significance based on 
the multiplicity-adjusted testing procedure that will be specified in the SAP. 
Secondary  
The secondary endpoints will be analyzed using similar statistical methods as the 
primary endpoints, as appropriate, with details specified in the SAP. 
 
  
7.4.2. 
Safety Analyses 
Four sets of safety analyses will be performed: (1) Part 1, this will provide summaries of safety 
endpoints in a side-by-side comparison between the two treatment groups that should be similar 
at Baseline; 
 
 
 (3) Part 1 
, this will provide summaries of safety endpoints 
in a side-by-side comparison between the two treatment groups through Part 1 
; 
(4) Time since SRP-9001 treatment, this will provide summaries of safety endpoints across all 
patients who received SRP-9001 in either Part 1 
, starting from the time of SRP-9001 
infusion and continue through the end of the study. 
Endpoint 
Statistical Analysis Methods 
Safety: TEAEs, 
and SAEs  
Adverse events will be coded by the Medical Dictionary for Regulatory Activities. 
TEAEs include all AEs that occurred or increased in severity since the study 
treatment. A treatment-related TEAE will be defined as a TEAE that the Investigator 
considered possibly/probably or definitely related to the study treatment.  
TEAEs will be summarized by treatment group by the number and percentage of 
patients who reported TEAEs, with grouping by system organ class and preferred 
term. SAEs will be summarized in the same way. TEAEs will also be summarized by 
treatment group, with grouping by system organ class, preferred term, and severity. 
Further, treatment-related TEAEs will be summarized in the same way as TEAEs.  
Safety: Clinical 
laboratory 
assessments 
Clinical laboratory variables will be presented in 3 ways.  
First, change from Baseline to each scheduled assessment will be summarized 
descriptively by treatment group. Baseline will be defined as the value of the clinical 
laboratory variable obtained immediately before dosing of study drug (Day -1 for 
Part 1 and Day -1 for Part 2). For Part 1, if Day -1 value is unavailable, the value 
obtained at the Screening Visit will be used. 
Second, treatment-emergent potentially clinically significant (PCS) laboratory values 
will be identified. Criteria for PCS laboratory values will be defined in the SAP. 
Treatment-emergent PCS laboratory values will be defined as post-Baseline PCS 
laboratory values for patients whose baseline value for the same laboratory variable 
did not meet the PCS criteria. The number and percentage of patients with a 
treatment-emergent PCS laboratory value will be summarized by treatment group for 
each clinical laboratory variable. 
Third, the number and percentage of patients with abnormal, clinically significant 
laboratory values (per Investigator judgment) will be summarized by treatment group 
for each clinical laboratory variable. 
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7.5. 
Interim Analyses  
Up to  planned interim analyses may be performed. The SAP will describe the planned interim 
analyses in greater detail, including the testing procedure to adjust for multiplicity to control the 
overall Type I error rate at a 2-sided level of 0.05.  
7.5.1. 
Interim Analysis at Week 12 in Part 1 
An interim analysis for micro-dystrophin expression will be performed after the blinded assays 
for micro-dystrophin expression for Baseline and Week 12 (Part 1) have been completed. Unless 
an unexpected outcome occurs at the interim analysis, there is no plan to change the conduct of 
the study after the interim analysis. A procedure will be put in place prior to unblinding of any 
personnel to micro-dystrophin expression data to ensure that the study continues to be conducted 
as a double blinded study after this interim analysis. 
7.5.2. 
Interim Analysis at the Completion of Part 1 
An interim analysis will be performed after all patients have completed Part 1 of the study. This 
will be the primary analysis of the study. A procedure will be put in place prior to unblinding of 
a limited number of personnel to ensure that the study continues to be conducted as a double 
blinded study after this interim analysis. 
7.5.3. 
 
 
 
7.5.4. 
Interim Analysis Based on Regulatory Interactions 
An additional interim analysis may be performed based on regulatory interactions. The decision 
to perform this interim analysis may take into account information external to this study and/or 
blinded study data review. If this interim analysis is to be performed during Part 1 or Part 2, a 
procedure will be put in place prior to this interim analysis to ensure that the study continues to 
be conducted as a double blinded study after this interim analysis. 
7.6. 
Data Monitoring Committee 
An independent DMC will be established to periodically review the safety data of this study and 
provide recommendations to the Sponsor. The DMC will operate under a DMC charter to be 
approved by the DMC members and the Sponsor.  
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8. 
DISCONTINUATION OF STUDY INTERVENTION AND 
PARTICIPANT DISCONTINUATION/WITHDRAWAL 
8.1. 
Discontinuation of Study Intervention 
Participants will receive a single-dose of study treatment in Part 1 and Part 2 of the study; 
therefore, discontinuations from the study intervention are not anticipated. 
8.2. 
Participant Discontinuation/Withdrawal from the Study 
• A participant may withdraw from the study at any time at his own or legal guardian’s 
request or may be withdrawn at any time at the discretion of the Investigator for 
safety, behavioral, compliance, or administrative reasons. This is expected to be 
uncommon. 
• At the time of discontinuing from the study or if the sponsor terminates the study for 
any reason, if possible, an early termination visit should be conducted, as shown in 
the SoA (Section 1.3). See the SoA (Section 1.3) for data to be collected at the time of 
study discontinuation or termination and for any further evaluations that need to be 
completed. 
• If the participant (or legal guardian) withdraws consent for disclosure of future 
information, the Sponsor may retain and continue to use any data collected before 
such a withdrawal of consent. 
• If a participant withdraws from the study, he (or legal guardian) may request 
destruction of any samples taken and not tested, and the Investigator must document 
this in the site study records. 
8.3. 
Lost to Follow-up 
A participant will be considered lost to follow-up if he repeatedly fails to return for scheduled 
visits and is unable to be contacted by the study site. 
The following actions must be taken if a participant fails to return to the clinic for a required 
study visit: 
• The site must attempt to contact the participant (or if applicable, the legal guardian 
who signed the informed consent on behalf of the participant) and reschedule the 
missed visit as soon as possible and counsel the participant (or if applicable, the legal 
guardian who signed the informed consent on behalf of the participant) on the 
importance of maintaining the assigned visit schedule and ascertain whether or not 
the participant wishes to and/or should continue in the study. 
• Before a participant is deemed lost to follow up, the Investigator or designee must 
make every effort to regain contact with the participant (or if applicable, the legal 
guardian who signed the informed consent on behalf of the participant) (where 
possible, 3 telephone calls and, if necessary, a certified letter to the participant’s last 
known mailing address or local equivalent methods). These contact attempts should 
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• Should the participant continue to be unreachable, he/she will be considered to have 
withdrawn from the study. 

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9. 
SUPPORTING DOCUMENTATION AND OPERATIONAL 
CONSIDERATIONS 
9.1. 
Appendix 1: Regulatory, Ethical, and Study Oversight 
Considerations 
9.1.1. 
Clinical Monitoring of the Study 
The study will be monitored in compliance with the relevant parts of 21 Code of Federal 
Regulations (CFR) and according to the International Council on Harmonisation (ICH) Good 
Clinical Practices (GCP) Guidelines. The procedures outlined in the protocol and case report 
forms will be carefully reviewed by the PI and staff prior to study initiation to ensure appropriate 
interpretation and implementation. No deviations from the protocol shall be made except in 
emergency situations where alternative treatment is necessary for the protection, proper care and 
wellbeing of patients. 
9.1.2. 
Regulatory and Ethical Considerations 
• This study will be conducted in accordance with the protocol and with the following: 
− Consensus ethical principles derived from international guidelines including the 
Declaration of Helsinki and Council for International Organizations of Medical 
Sciences International Ethical Guidelines 
− Applicable ICH GCP Guidelines 
− Applicable laws and regulations 
• The protocol, protocol amendments, ICF, Investigator Brochure, and other relevant 
documents (eg, advertisements) must be submitted to an IRB/IEC by the Investigator 
and reviewed and approved by the IRB/IEC before the study is initiated.  
• Any amendments to the protocol will require IRB/IEC approval before 
implementation of changes made to the study design, except for changes necessary 
to eliminate an immediate hazard to study participants.  
• The Investigator will be responsible for the following: 
− Providing written summaries of the status of the study to the IRB/IEC annually or 
more frequently in accordance with the requirements, policies, and procedures 
established by the IRB/IEC 
− Notifying the IRB/IEC of SAEs or other significant safety findings as required by 
IRB/IEC procedures 
− Providing oversight of the conduct of the study at the site and adherence to 
requirements of 21 CFR, ICH guidelines, the IRB/IEC, European regulation 
536/2014 for clinical studies (if applicable), and all other applicable local 
regulations 

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9.1.3. 
Financial Disclosure 
Investigators and sub-Investigators will provide the Sponsor with sufficient, accurate financial 
information as requested to allow the Sponsor to submit complete and accurate financial 
certification or disclosure statements to the appropriate regulatory authorities. Investigators and 
sub-Investigators are responsible for providing information on financial interests during the 
course of the study and for 1 year after completion of the study. 
9.1.4. 
Informed Consent Process 
Legally effective and properly executed written informed consent, in compliance with 
21 CFR 50 and the ICH guidelines, will be obtained from each patient, or legal guardian 
where applicable, before the patient is entered into the trial or before any unusual or non-routine 
procedure is performed that involves risk to the patient. The informed consent will be signed 
prior to study procedures and will require IRB approval. Attention will be directed to the basic 
elements that are required for incorporation into the informed consent under United States 
Federal Regulations for Protection of Human Subjects [21CFR 50.25(a)]. The final 
IRB-approved document as well as any subsequent approved modified consent document(s) 
must be provided to correspondent agencies for regulatory purposes. If new information related 
to the study arises, patients, or legal guardians where applicable, will be asked to sign a revised 
document. Signed consent forms will remain in each patient's research chart and will be available 
for verification by study monitors at any time. Patients, or legal guardians where applicable, will 
be given a signed, dated copy of their consent form. 
• The Investigator or his/her representative will explain the nature of the study to the 
participant or his legally authorized representative and answer all questions regarding 
the study.  
• Participants, and legal guardians where applicable, must be informed that their 
participation is voluntary. Participants or their legally authorized representative will 
be required to sign a statement of informed consent that meets the requirements of 
21 CFR 50, local regulations, ICH guidelines, Health Insurance Portability and 
Accountability Act requirements, where applicable, and the IRB/IEC or study center.  
• The medical record must include a statement that written informed consent was 
obtained before the participant was enrolled in the study and the date the written 
consent was obtained. The authorized person obtaining the informed consent must 
also sign the ICF. 
• Participants must be re-consented to the most current version of the ICF(s) during 
their participation in the study.  
• A copy of the ICF(s) must be provided to the participant or the participant’s legally 
authorized representative.  
The ICF will contain a separate section that addresses the use of remaining mandatory samples 
for 
. The Investigator or authorized designee will explain to each 
participant the objectives of the 
. Participants, and their legal guardians 
where applicable, will be told that they are free to refuse to participate and may withdraw their 
consent at any time and for any reason during the storage period. 
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9.1.5. 
Data Protection 
9.1.5.1. 
Data Safety Monitoring Plan 
All data and observations will be documented on CRFs by source documentation. A Study 
Monitor will have access to the data to monitor adherence to the protocol and to applicable 
regulations, and the maintenance of adequate and accurate clinical records. A CRF will be 
completed for every patient that was randomized for participation in the study. The CRFs will be 
completed as data is generated. Additional information can be found in the CRF Completion 
Guidelines.  
9.2. 
Committees Structure 
9.2.1. 
Data Monitoring Committee  
The independent DMC will act in an advisory capacity to review participant safety and study 
progress for the clinical trial. For additional information, refer to the Study Operations Manual 
and DMC Charter.  
9.2.2. 
Dissemination of Clinical Study Data 
9.2.2.1. 
Final Study Report 
The final study report will include data through the final study visit.  
9.2.3. 
Source Documents 
• Source documents provide evidence for the existence of the participant and 
substantiate the integrity of the data collected. Source documents are filed at the 
Investigator’s site. 
• Data reported on the CRF or entered in the electronic CRF that are transcribed from 
source documents must be consistent with the source documents or the discrepancies 
must be explained. The Investigator may need to request previous medical records or 
transfer records, depending on the study. Also, current medical records must be 
available. 
9.2.4. 
Publication Policy 
The information that is developed during the conduct of this clinical study is considered to be 
strictly confidential. This information may be disclosed only as deemed necessary by Sarepta 
Therapeutics, Inc. At the conclusion of this clinical study, a clinical study report will be 
prepared. In addition, a manuscript may be prepared for publication in a reputable scientific 
journal under the direction of Sarepta. Sarepta Therapeutics, Inc. will publish and communicate 
the clinical study results, irrespective of positive or negative findings. Data generated for this 
study will be exclusively owned by Sarepta Therapeutics, Inc., as detailed in the Clinical Trial 
Agreement. The study will be registered on ClinicalTrials.gov. After completion of the study, 
results will be disseminated through ClinicalTrials.gov.  

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9.3. 
Appendix 2: 
  
 
 
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10. 
REFERENCES 
 
 
 
Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy: 2. 
Determination of the “power” of therapeutic trials based on the natural history. Muscle Nerve. 
1983;6(2):91-103 
D’Amario D, Amodeo A, Adorisio R, et al. A current approach to heart failure in Duchenne 
muscular dystrophy. Heart. 2017;103(22):1770-1779. 
Eagle M, Baudouin SV, Chandler C, et al. Survival in Duchenne muscular dystrophy: 
improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. 
Neuromuscul Disord. 2002;12(10):926-929. 
 
 
 
Mazzone E, Martinelli D, Berardinelli A, et al. North Star Ambulatory Assessment, 6-minute 
walk test and timed items in ambulant boys with Duchenne muscular dystrophy. Neuromuscul 
Disord. 2010;20(11):712-716. 
Mendell JR, Campbell K, Rodino-Klapac L, et al. Dystrophin immunity in Duchenne’s muscular 
dystrophy. N Engl J Med. 2010;363(15):1429-1437. 
 
 
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