Open-Label Extension of the Halt Cardiomyopathy Progression in Duchenne (HOPE-Duchenne) Trial (CAP-1002-DMD-03)
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Sponsor: Capricor Inc.

CLINICAL TRIAL PROTOCOL 
OPEN-LABEL EXTENSION OF THE HALT CARDIOMYOPATHY PROGRESSION 
IN DUCHENNE (HOPE-DUCHENNE) TRIAL 
Protocol Number: 
CAP-1002-DMD-03 
Trial Phase: 
Phase 2 
Product Name: 
CAP-1002 Allogeneic Cardiosphere-
Derived Cells 
IND Number: 
Indication: 
Duchenne Muscular Dystrophy 
Sponsor: 
Capricor, Inc. 
8840 Wilshire Blvd., 2nd Floor 
Beverly Hills, CA  90211 
Sponsor Contact: 
Original Protocol: 
08-Jan-2018
Amendment 1.0 
12-Feb-2018
This document is confidential and proprietary to Capricor, Inc. and its affiliates. Acceptance of this document constitutes agreement by 
the recipient that no unpublished information contained herein will be reproduced, published, or otherwise disseminated or disclosed or 
used without the prior written approval of Capricor, Inc. and its affiliates, except that this document may be disclosed in any medium to 
appropriate clinical Investigators, Institutional Review Boards, and others directly involved in the clinical investigation that is the subject 
of this information under the condition that they keep the information strictly confidential.  
NCT 06304064

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1.1. 
Investigator’s Agreement 
 
 
Trial Title: 
Open Label Extension of the Halt cardiomyOPathy progrEssion in 
Duchenne (HOPE-Duchenne) Trial.   
Short Title: 
Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) 
Protocol Number:  CAP-1002-DMD-03 
Amendment 1.0:  
12-Feb-2018 
 
I have read this clinical trial protocol and agree to conduct the trial according to the 
investigational plan. I agree to maintain the confidentiality of all information received or 
developed in connection with this protocol.  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Printed Name of Investigator 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Signature of Investigator 
 
 
 
 
 
 
 
 
 
Date 
 
 
 
 
 

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1.3. 
Procedures in Case of Emergency 
Please reference the Manual of Procedures for detailed information on serious adverse event 
reporting. 
  
Table 1: 
Emergency Contact Information 
Role in Trial 
Name 
Address and Telephone Number 
 
 
Capricor, Inc.  
8840 Wilshire Blvd., 2nd Floor 
Beverly Hills, CA 90211 
Tel: 
 
Email: 
 
 
 
Capricor, Inc.  
8840 Wilshire Blvd., 2nd Floor 
Beverly Hills, CA 90211 
Tel: 
 
Email: 
 
 

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2. 
SYNOPSIS 
Name of Sponsor: Capricor, Inc.  
Name of Investigational Product: CAP-1002 Allogeneic Cardiosphere-Derived Cells 
Name of Active Ingredient: Cardiosphere-Derived Cells 
Title of Trial: Open Label Extension of the Halt cardiomyOPathy progrEssion in Duchenne (HOPE-
Duchenne) Trial 
Trial Name: Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE) 
Trial Centers: Up to 3 (USA) 
Principal Investigators: This is a multi-center trial with multiple Principal Investigators. 
Studied Period: Approximately 6 months 
Phase of Development: 2 
Objectives:  
Primary: To provide CAP-1002 to subjects who were randomized to the Usual Care Treatment Group 
of the HOPE-Duchenne trial and completed 12 months of follow-up.   
Secondary: To explore the safety and efficacy of intravenous administration of CAP-1002 with repeat 
dose at Month 3.  
Methodology: This Phase 2, multi-center, open label extension trial will provide CAP-1002 to subjects 
that were randomized to the Usual Care Treatment Group of the HOPE-Duchenne trial (CAP-1002-
DMD-01) and explore the safety of two intravenous administrations of CAP-1002, each separated by 
three months. Subjects will undergo targeted tests and procedures during a 30-day screening period to 
determine eligibility based on protocol inclusion and exclusion criteria.  
Eligible subjects will undergo baseline safety and efficacy assessments on Day 1 prior to their first 
infusion of CAP-1002. Administration of CAP-1002 (Day 1) should occur within a maximum of 14 
days following confirmation of eligibility; if a delay of more than 14 days between enrollment and IP 
administration is unavoidable, a conversation between the Investigator and Medical Monitor should 
occur to determine the need for repeat assessments prior to infusion.  
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 
and Month 3. Subjects will be observed in the outpatient setting for at least two hours post infusion and 
then discharged the same day if medically cleared by the site Investigator. 
A safety phone call will be performed 14 days (±3 days) after each CAP-1002 infusion, and if 
clinically indicated, an unscheduled in-person visit will be performed at the investigative site with 
targeted assessments based on presentation of signs and symptoms. 
Blood samples will be collected before each CAP-1002 administration (Day 1 and Month 3), after each 
CAP-1002 infusion (Week 4 and Month 4) and at Month 6, unless otherwise indicated, for donor 
specific antibody (DSA) testing and routine serum chemistries, hematology and cardiac enzymes. HLA 

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typing will occur before the first CAP-1002 administration on Day 1. Collection will occur either at the 
investigative site or remotely at a designated central laboratory patient service center.  
Trial visits at the investigative site will occur at Months 3 and 6 (±14 days, respectively). Subjects will 
complete safety and efficacy assessments prior to CAP-1002 administration at the Month 3 trial visit. 
The primary safety endpoints include the incidence of the following from baseline through the 6-
month timepoint:  
• 
Acute respiratory decompensation within 2 hours following IP administration 
• 
Hypersensitivity reaction  
− 
Hypersensitivity reaction is defined as a clinical syndrome including, but not limited 
to, fever, leukocytosis, or rash with onset ≤ 2 hours post infusion and lasting < 24 
hours, in the absence of clinical signs of concomitant infection.  
• 
All-cause mortality 
• 
Serious adverse events 
• 
Treatment-emergent adverse events related to IP or administration procedure 
• 
Immune sensitization syndrome 
− 
Immune sensitization syndrome shall be defined as: (a) clinical signs and symptoms 
consistent with systemic inflammation (e.g., fever, leukocytosis, rash, or arthralgia) 
with onset ≥ 24 hours post infusion and the absence of clinical signs of concomitant 
infection, AND (b) elevation of anti-human leukocyte antigen (HLA) antibodies 
against the donor cells (i.e., DSAs), detected ≤ 30 days following onset of syndrome, 
of (i) ≥ 2000 mean fluorescent intensity (MFI) if baseline MFI ≤ 1000, or (ii) ≥ 2 times 
baseline otherwise 
The exploratory endpoints include the change from baseline to each assessment timepoint for the 
following assessments: 
• 
All subjects 
− 
Mid-level (elbow) dimension of the Performance of Upper Limb (PUL) 1.2  
− 
Mid- and distal-level (elbow) dimension of the PUL 1.2  
− 
High-level (shoulder) dimension of the PUL 1.2 
− 
Left ventricular structure and function as assessed by cardiac MRI including regional 
systolic left ventricular wall thickening ejection fraction, end-diastolic volume, end-
systolic volume, stroke volume, regional wall thickness, and circumferential strain 
− 
Slow vital capacity (SVC), forced expiratory volume in one second (FEV1), forced 
vital capacity (FVC), peak expiratory flow (PEF), maximum inspiratory pressure 
(MIP), maximum expiratory pressure (MEP), peak cough flow (PCF), and inspiratory 
flow reserve (IFR) 
• 
Ambulatory subjects only  

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− 
6-Minute Walk Test (6MWT)  
Oversight of the trial will be provided by the trial Steering Committee, an independent Data Safety 
Monitoring Board (DSMB), and an independent, Clinical Events Committee (CEC) that will adjudicate 
all potential primary safety endpoints. 
Number of Subjects: Approximately 10 subjects will be enrolled  
Diagnosis and Main Criteria for Inclusion: Subjects randomly assigned to the Usual Care Treatment 
Group of the HOPE-Duchenne trial and who completed the 12-month follow-up period will be 
evaluated for eligibility to participate in the Open Label Extension trial. Eligibility criteria will be 
assessed within 30 days prior to first infusion on Day 1 unless otherwise noted.  
Inclusion Criteria:  
1. Documented enrollment in the Usual Care Treatment Group of the HOPE-Duchenne trial and 
completion of trial follow-up through Month 12  
2. Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and 
assent with parental or guardian informed consent if < 18 years of age 
3. Adequate venous access for intravenous CAP-1002 infusions and routine blood collections in 
the judgement of the Investigator  
4. Assessed by the Investigator as willing and able to comply with the requirements of the trial 
Exclusion Criteria: 
1. Left ventricular ejection fraction (LVEF) < 35% within 6 months of screening 
2. Planned or likely major surgery in the next 6 months after planned first infusion 
3. Risk of near-term respiratory decompensation in the judgment of the investigator, or the need 
for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L 
at screening  
4. History of non DMD-related chronic respiratory disease including, but not limited to, asthma, 
bronchitis, and tuberculosis 
5. Acute respiratory illness within 30 days prior to screening 
6. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products 
7. Treatment with investigational product ≤ 6 months prior to first infusion 
8. History, or current use, of drugs or alcohol that could impair ability to comply with 
participation in the trial 
9. Inability to comply with the investigational plan and follow-up visit schedule for any reason, in 
the judgment of the investigator 
Investigational Product, Dosage and Mode of Administration: 
 
 
 
 
 

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Duration of Treatment: CAP-1002 
 is administered as a single intravenous 
infusion on Day 1 and at Month 3 for a total of 2 administrations. 
Duration of Trial: All subjects will be followed for 6 months post Day 1 infusion. 
Criteria for evaluation: 
Safety 
Safety assessments at Week 4 and Months 3,4 and 6, unless otherwise indicated, will include the 
following: vital signs, height, weight, physical examination, 12-lead ECG, adverse events, DSA 
assessment, and clinical laboratory testing (serum chemistries, hematology, and cardiac enzymes). 
Adverse events and concomitant medications will also be collected during each 14-day post-infusion 
safety phone call, or if clinically indicated, an unscheduled in-person visit will be performed at the 
investigative site with targeted assessments based on presentation of signs and symptoms of subject. 
Efficacy  
The following efficacy outcome measures will be performed at Months 3 and 6 unless otherwise 
specified: PUL 1.2, spirometry, 6MWT when deemed appropriate by the Investigator, and cardiac 
MRI.  
Statistical Methods: 
Sample Size 
Up to 10 subjects that were randomized to Usual Care Treatment Group and completed the 12-month 
follow-up period for the HOPE-Duchenne trial will be enrolled in this trial.  
Analysis Populations  
Intent-to-Treat (ITT) Population: All patients who meet the enrollment criteria and are enrolled in the 
trial. These patients will also constitute the Safety population.  
Per Protocol (PP) Population: All enrolled subjects who have no protocol deviations/violations that 
could significantly impact completeness, accuracy, and/or reliability of the trial data. The list of 
subjects in the per protocol population will be compiled prior to database lock.  

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Safety Analysis 
All primary safety endpoints and other observed adverse events will be documented and reported. 
Adverse events will be summarized by the incidence of events by type and by percentage of subjects 
with those events. Primary safety endpoints that are adjudicated by the Clinical Events Committee 
(CEC) will be further described by the relatedness to the trial treatment and severity level.  
Efficacy Analysis 
Descriptive statistics will be used to summarize changes from baseline in the PUL 1.2 scores 
(including the mid-level dimension, combined mid and distal-level dimensions, and high-level 
dimension), in left ventricular structure and function parameters (including ejection fraction, wall 
thickness, end-diastolic and end-systolic volumes, stroke volume, and circumferential strain, in 
pulmonary function (including SVC, FEV1, FVC, MIP, MEP, PCF, and IFR) and in 6-minute walk test 
distance for ambulatory patients. 

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11.4.1. 
Slow Vital Capacity ....................................................................................................47 
11.4.2. 
Spirometry ..................................................................................................................47 
11.4.3. 
Maximal Inspiratory & Expiratory Pressures .............................................................47 
11.4.4. 
Peak Cough Flow ........................................................................................................48 
11.4.5. 
Inspiratory Flow Reserve ............................................................................................48 
11.5. 
6-Minute Walk Test ....................................................................................................48 
11.6. 
Cardiac MRI ...............................................................................................................48 
12. 
OTHER TRIAL ASSESSMENTS .............................................................................50 
12.1. 
Medical Status Questionnaire .....................................................................................50 
12.2. 
Medical History ..........................................................................................................50 
12.3. 
Prior and Concomitant Medications ...........................................................................50 
12.4. 
Planned Medical/Surgical Procedures ........................................................................50 
13. 
ASSESSMENT OF SAFETY .....................................................................................51 
13.1. 
Safety Parameters .......................................................................................................51 
13.1.1. 
Vital Signs ..................................................................................................................51 
13.1.2. 
Weight and Height ......................................................................................................51 
13.1.3. 
Physical Examination .................................................................................................51 
13.1.4. 
Electrocardiogram (ECG) ...........................................................................................52 
13.1.5. 
Immunologic Assessments .........................................................................................52 
13.1.6. 
Clinical Laboratory Assessments ...............................................................................52 
13.1.6.1. 
Hematology .................................................................................................................52 
13.1.6.2. 
Serum Chemistry ........................................................................................................52 
13.1.6.3. 
Cardiac Enzymes ........................................................................................................53 
13.2. 
Adverse and Serious Adverse Events .........................................................................53 
13.2.1. 
Definition of Adverse Events .....................................................................................53 
13.2.1.1. 
Adverse Event (AE) ....................................................................................................53 
13.2.1.2. 
Serious Adverse Event (SAE) ....................................................................................53 
13.2.1.3. 
Expected Adverse Events ...........................................................................................54 
13.2.1.4. 
Unexpected Adverse Events .......................................................................................54 
13.2.1.5. 
Other Adverse Event (OAE) .......................................................................................54 
13.2.2. 
Relationship to Investigational Product ......................................................................55 
13.2.3. 
Recording Adverse Events .........................................................................................56 
13.2.4. 
Reporting Adverse Events ..........................................................................................56 

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13.2.5. 
Pregnancy Reporting ..................................................................................................57 
13.2.6. 
Adverse Event Follow-up ...........................................................................................57 
14. 
STATISTICS ..............................................................................................................59 
14.1. 
General Considerations ...............................................................................................59 
14.2. 
Sample Size ................................................................................................................59 
14.3. 
Analysis Population ....................................................................................................59 
14.4. 
Safety Analysis ...........................................................................................................59 
14.4.1. 
Safety Endpoints .........................................................................................................59 
14.4.2. 
Medications .................................................................................................................60 
14.4.3. 
Clinical Laboratory Evaluations .................................................................................60 
14.4.4. 
Donor-Specific Antibodies .........................................................................................60 
14.4.5. 
Physical Examination .................................................................................................60 
14.5. 
Efficacy Analysis ........................................................................................................60 
14.5.1. 
Exploratory Efficacy Endpoints .................................................................................60 
14.6. 
Statistical Methods ......................................................................................................60 
14.6.1. 
Multiplicity .................................................................................................................61 
14.6.2. 
Missing Data ...............................................................................................................61 
15. 
TRIAL OVERSIGHT .................................................................................................62 
15.1. 
Steering Committee ....................................................................................................62 
15.2. 
Clinical Events Committee (CEC) ..............................................................................62 
15.3. 
Data Safety Monitoring Board ....................................................................................62 
16. 
SOURCE DATA AND TRIAL DOCUMENTS ........................................................63 
16.1. 
Electronic Data Capture ..............................................................................................63 
16.2. 
Trial Monitoring .........................................................................................................63 
16.2.1. 
Source Document Requirements ................................................................................64 
16.3. 
Audits and Inspections ................................................................................................65 
16.4. 
Retention of Records ..................................................................................................65 
17. 
QUALITY CONTROL AND QUALITY ASSURANCE .........................................66 
17.1. 
Qualifications and Trainings .......................................................................................66 
17.2. 
Good Clinical Practices (GCP) ...................................................................................66 
17.3. 
HIPAA or Other Privacy Training ..............................................................................66 
17.4. 
Site Initiation ..............................................................................................................66 
17.5. 
Protocol Deviations ....................................................................................................67 

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18. 
ETHICS ......................................................................................................................68 
18.1. 
Ethics Review .............................................................................................................68 
18.2. 
Ethical Conduct of the Trial .......................................................................................68 
18.3. 
Written Informed Consent and Assent .......................................................................68 
18.3.1. 
Obtaining Informed Consent and Assent ....................................................................69 
18.4. 
Subject Confidentiality ...............................................................................................69 
19. 
PUBLICATION POLICY ..........................................................................................70 
20. 
REFERENCES ...........................................................................................................71 
 

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LIST OF TABLES 
Table 1: 
Emergency Contact Information ...................................................................................4 
Table 2: 
Abbreviations and Specialist Terms ...........................................................................17 
Table 3: 
Schedule of Assessments ............................................................................................33 
 
 
Table 5: 
Height Calculation from Ulna Length ........................................................................51 
 

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LIST OF FIGURES 
Figure 1: 
Trial Design ................................................................................................................32 
 
 
 
  

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4. 
LIST OF ABBREVIATIONS AND DEFINITIONS OF TERMS 
The following abbreviations and specialist terms are used in this trial protocol. 
Table 2: 
Abbreviations and Specialist Terms  
Term 
Explanation 
 
Term 
Explanation 
6MWT 
Six-Minute Walk Test 
 
ESR 
Expedited Safety Report 
AE 
Adverse Event 
 
ET 
Early Termination 
ALT 
Alanine Aminotransferase 
 
ETFE 
Ethylene Tetrafluoroethylene 
AST 
Aspartate Aminotransferase 
 
FDA 
U.S. Food and Drug 
Administration 
BUN 
Blood Urea Nitrogen 
 
FEV1 
Forced Expiratory Volume in 1 
Second 
C 
Celsius 
 
FVC 
Force Vital Capacity 
CBC 
Complete Blood Count 
 
GCP 
Good Clinical Practice 
CDC 
Cardiosphere-Derived Cells 
 
GMP 
Good Manufacturing Practice 
CEC 
Clinical Events Committee 
 
HED 
Human Equivalent Dose 
CFR 
Code of Federal Regulations 
 
HEENT 
Head, Eyes, Ears, Nose, and 
Throat 
CK-MB 
Creatine kinase MB 
Isoenzyme 
 
HFrEF 
Heart Failure with Reduced 
Ejection Fraction 
CLIA 
Clinical Laboratory 
Improvement Amendments 
 
HIPAA 
Health Insurance Portability and 
Accountability Act 
CRF 
Case Report Form 
 
HLA 
Human Leukocyte Antigen 
CRO 
Clinical Research 
Organization 
 
HSA  
Human Serum Albumin 
CT 
Computerized Tomography 
 
IB 
Investigator's Brochure 
DCM 
Dilated Cardiomyopathy 
 
ICH 
International Conference of 
Harmonisation 
DMD 
Duchenne Muscular 
Dystrophy 
 
IFR 
Inspiratory Flow Reserve 
DMSO 
Dimethyl Sulfoxide 
 
IND 
Investigational New Drug 
Application 
DSA 
Donor-Specific Antibody 
 
IP 
Investigational Product 
DSMB 
Data Safety Monitoring 
Board 
 
IRB 
Institutional Review Board 
ECG 
Electrocardiogram 
 
ITT 
Intent-to-Treat 
EDC 
Electronic Data Capture 
 
IV 
Intravenous 

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Term 
Explanation 
 
Term 
Explanation 
IWRS 
Interactive Web Response 
System 
 
PI 
Principal Investigator 
LV 
Left Ventricle / Left 
Ventricular 
 
PP 
Per Protocol 
LVEDV 
Left Ventricular End-
Diastolic Volume 
 
PT 
Preferred Term 
LVEF 
Left Ventricular Ejection 
Fraction 
 
PUL 
Performance of the Upper Limb 
LVESV 
Left Ventricular End-Systolic 
Volume 
 
QOL 
Quality of Life 
M 
Million 
 
RBC 
Red Blood Cell 
MACE 
Major Adverse Cardiac Event 
 
RV 
Residual Volume 
MCB 
Master Cell Bank 
 
SAE 
Serious Adverse Event 
MedDRA 
Medical Dictionary for 
Regulatory Activities 
 
SD 
Standard Deviation 
MEP 
Mean Expiratory Pressure 
 
SOC 
System Organ Class 
MFI 
Mean Fluorescence Intensity 
 
SpO2 
Peripheral Capillary Hemoglobin 
Oxygen Saturation 
MHC 
Major Histocompatibility 
Complex 
 
SVC 
Slow Vital Capacity 
MI 
Myocardial Infarction 
 
TEAE 
Treatment-Emergent Adverse 
Events 
MIP 
Mean Inspiratory Pressure 
 
Term 
Explanation 
mITT 
Modified Intent-to-Treat 
 
TIMI 
Thrombolysis in Myocardial 
Infarction 
mL 
Milliliter 
 
TLC 
Total Lung Capacity 
MOP 
Manual of Procedures 
 
v/v 
Volume to Volume 
MRI 
Magnetic Resonance Image 
 
WBC 
White Blood Cell 
MSC 
Mesenchymal Stem Cells 
 
WHO-DD 
World Health Organization Drug 
Dictionary 
OAE 
Other Adverse Events 
 
 
 
OLE 
Open Label Extension 
 
 
 
PCF 
Peak Cough Flow 
 
 
 
PE 
Physical Exam 
 
 
 
PEF 
Peak Expiratory Flow 
 
 
 
PFT 
Pulmonary Function Test 
 
 
 

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5. 
INTRODUCTION 
5.1. 
Background 
Duchenne muscular dystrophy (DMD) is a severe, X-linked, progressive disease affecting 
approximately one in 3,600 to 9,200 male births (Mah et al., 2014). It is caused by mutations in 
the dystrophin gene resulting in the absence of or non-functional dystrophin protein (Hoffman et 
al., 1988). 
Dystrophin is a cytoplasmic protein encoded by the dmd gene, which links cytoskeletal actin 
filaments to membrane proteins. The dystrophin protein acts as a shock absorber during muscle 
fiber contraction by linking the actin of the contractile apparatus to the layer of connective tissue 
that surrounds each muscle fiber (Koenig et al., 1988; Fairclough et al., 2013; Aartsma-Rus et 
al., 2016). 
Due to the lack of dystrophin, the connection between the actin cytoskeleton and connective 
tissue is lost, inducing excessive membrane fragility and permeability, dysregulation of calcium 
homeostasis, and oxidative damage, which results in muscle cell necrosis and a chronic 
inflammatory state. Initially, muscle necrosis is followed by regeneration, but with age, the 
regenerative ability of myofibers is lost and muscle fibers are gradually replaced by connective 
and adipose tissue. It has been postulated that chronic injury and regeneration induce satellite 
cell exhaustion. Recent studies suggest that the absence of dystrophin in satellite cells impairs 
their ability to divide properly, thus reducing the generation of myogenic progenitors that are 
needed for proper muscle regeneration (Dumont et al., 2015). As a consequence, muscle function 
is lost (Muntoni et al., 2003; Deconinck and Dan, 2007; Falzarano et al., 2015). 
DMD symptoms begin in early childhood, with degeneration occurring progressively in the 
skeletal musculature and ultimately in the heart and respiratory muscles, resulting in premature 
death (Hendriksen et al., 2015). Progressive weakness and muscle atrophy caused by 
degenerating muscle fibers begins in the lower extremities and pelvis before spreading into the 
upper extremities. Other symptoms include loss of some reflexes, a waddling gait, frequent falls, 
difficulty when rising from a sitting or lying position or when climbing stairs, changes to overall 
posture, and impaired breathing. Many children precipitously lose the ability to run or jump. The 
atrophied muscles, in particular the calf muscles, and less commonly, muscles in the buttocks, 
shoulders, and arms, may be enlarged by an accumulation of fat and connective tissue, causing 
them to look larger and healthier than they actually are (“pseudohypertrophy”). Bone thinning 
and scoliosis are common. Ultimately, a wheelchair becomes necessary, in most cases between 
12 to15 years of age (Henricson et al., 2013).  
As the disease progresses, the muscles in the diaphragm that assist in breathing and coughing 
become weaker. Affected individuals experience breathing difficulties, respiratory infections, 
and swallowing problems. Almost all DMD patients will develop cardiomyopathy (Aartsma-Rus 
et al., 2016). Pneumonia, compounded by cardiac involvement, is the most frequent cause of 
death, which typically occurs in the late teens or early 20s. However, improvements in 
multidisciplinary care, in particular respiratory care and various forms of assisted ventilation, 
have extended the life expectancy; numerous individuals with DMD now survive into their 30s, 
and some even into their 40s (Emery, 2002; Bushby et al., 2010; Bushby et al., 2010). 

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No pharmacologic or biological therapies proven to stop or reverse the progression of DMD have 
been approved in the US. Disease management consists primarily of preventive measures as well 
as active interventions to address the primary and secondary aspects of the disorder.   

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5.7. 
Trial Population 
The target population for this trial is pediatric and adult males with a diagnosis of DMD who 
were randomized to the Usual Care Treatment Group of the HOPE-Duchenne trial and 
completed the 12-month follow-up period, and meet the OLE trial eligibility criteria (see 
Section 8) that focus on minimizing risk of participation in the extension trial.     

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6. 
TRIAL OBJECTIVES AND PURPOSE 
6.1. 
Primary Objective 
The primary objective of this trial is to provide CAP-1002 to subjects who were randomized to 
the Usual Care Treatment Group of the HOPE-Duchenne trial and completed the 12-month 
follow-up period.      
6.2. 
Secondary Objectives 
The secondary objectives of this trial are to explore the safety and efficacy of intravenous 
administration of CAP-1002 with repeat dosing at Month 3.     

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7. 
INVESTIGATIONAL PLAN 
7.1. 
Overall Trial Design 
This Phase 2, multi-center, open label extension trial will provide CAP-1002 to subjects who 
were randomized to the Usual Care Treatment Group and completed 12 months of follow-up in 
the HOPE-Duchenne trial. The trial will assess the safety of CAP-1002, administered as two IV 
infusions, each separated by 3 months. All subjects will undergo targeted tests and procedures 
within 30 days prior to first infusion, unless otherwise stated, to confirm eligibility based on 
protocol inclusion and exclusion criteria.  
Eligible subjects will undergo baseline safety and efficacy assessments (Table 3) prior to the first 
infusion of CAP-1002. Administration of CAP-1002 (Day 1) should occur within a maximum of 
14 days following confirmation of eligibility; if a delay of more than 14 days between enrollment 
and CAP-1002 administration is unavoidable, a conversation between the Investigator and 
Medical Monitor should occur to determine the need for repeat assessments prior to infusion. For 
the purposes of this trial, enrollment is defined as the time consent is signed.  
All intravenous infusions will be conducted in an outpatient setting at the investigative site on 
Day 1 and at Month 3. Subjects will be observed in the outpatient setting for at least two hours 
post infusion and then discharged the same day if medically cleared by the site Investigator.  
A safety phone call will be performed 14 days (±3 days) after each CAP-1002 infusion, and if 
clinically indicated, an unscheduled in-person visit will be performed at the investigative site 
with targeted assessments based on presentation of signs and symptoms. 
Blood samples will be collected at Week 4 and Month 4 (±7 days, respectively) for DSA testing 
and safety laboratory assessments. Collection will occur either at the investigative site or 
remotely at a designated central laboratory patient service center.  
Subsequent trial visits at the investigative site will occur at Months 3 and 6 (±14 days, 
respectively). Subjects will complete safety and efficacy assessments at each of these visits and 
prior to CAP-1002 administration in the case of the Month 3 trial visit. 
7.2. 
Trial Endpoints 
7.2.1. 
Safety Endpoints 
The primary safety endpoints include the incidence of the following from baseline through the 
6-month timepoint:  
• Acute respiratory decompensation within 2 hours following IP administration 
• Hypersensitivity reaction  
− Hypersensitivity reaction is defined as a clinical syndrome including, but not 
limited to, fever, leukocytosis, or rash with onset ≤ 2 hours post infusion and 
lasting < 24 hours, in the absence of clinical signs of concomitant infection.  
• All-cause mortality 
• Serious adverse events 

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• Treatment-emergent adverse events related to IP or administration procedure 
• Immune sensitization syndrome 
− Immune sensitization syndrome shall be defined as: (a) clinical signs and 
symptoms consistent with systemic inflammation (e.g., fever, leukocytosis, rash, 
or arthralgia) with onset ≥ 24 hours post infusion and the absence of clinical signs 
of concomitant infection, AND (b) elevation of anti-human leukocyte antigen 
(HLA) antibodies against the donor cells (i.e., DSAs), detected ≤ 30 days 
following onset of syndrome, of (i) ≥ 2000 mean fluorescent intensity (MFI) if 
baseline MFI ≤ 1000, or (ii) ≥ 2 times baseline otherwise 
7.2.2. 
Exploratory Endpoints 
The exploratory endpoints include the change from baseline to each assessment timepoint for 
the following assessments: 
• All subjects 
− Mid-level (elbow) dimension of the PUL 1.2  
− Mid- and distal-level (elbow) dimension of the PUL 1.2  
− High-level (shoulder) dimension of the PUL 1.2 
− Left ventricular structure and function as assessed by cardiac MRI including 
regional systolic left ventricular wall thickening, ejection fraction, end-diastolic 
volume, end-systolic volume, stroke volume, regional wall thickness, and 
circumferential strain 
− Slow vital capacity (SVC), forced expiratory volume in one second (FEV1), 
forced vital capacity (FVC), peak expiratory flow (PEF), maximum inspiratory 
pressure (MIP), maximum expiratory pressure (MEP), peak cough flow (PCF), 
and inspiratory flow reserve (IFR) 
• Ambulatory subjects only  
− 6-Minute Walk Test (6MWT)  
7.3. 
Number of Subjects 
Up to 10 subjects will be enrolled.  
7.4. 
Treatment Assignment 
All subjects will receive open-label CAP-1002 (
 at Day 1 and Month 3 via IV 
infusion.    
7.5. 
Dose Adjustment Criteria 
There are no planned dose adjustments. Should an acute toxicity arise during the infusion (e.g., 
hypersensitivity reaction, pulmonary decompensation, etc.), the infusion should be terminated 
immediately, and the actual total dose administered recorded. Any decision about re-challenging 

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in a subsequent infusion should be made after discussions with the Investigator, Medical 
Monitor, Data Safety Monitoring Board (DSMB), and other medical experts that may be 
required to make an informed decision. 
7.6. 
Criteria for Trial Termination 
The trial may be terminated at any time and for any reason, including, but not limited to, a 
recommendation by the DSMB for safety reasons, an action by the FDA, or decision by 
Capricor. 

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Figure 1: 
Trial Design 
 

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12 Subjects will undergo cardiac MRI if they are physically capable as determined by an Investigator. Subjects that cannot complete a cardiac MRI as part of the 
trial will complete an echocardiogram at screening using the site’s local equipment, acquisition protocol, and evaluation procedures. The reported LVEF from a 
local interpretation, whether it’s measured via cardiac MRI or echocardiogram, will be used to assess eligibility (exclusion criterion 1). 

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8. 
SELECTION AND WITHDRAWAL OF SUBJECTS 
Subjects who meet all inclusion criteria and no exclusion criteria will be eligible for the trial.  
8.1. 
Subject Inclusion Criteria 
Inclusion criteria will be assessed within 30 days prior to first infusion on Day 1 unless otherwise 
noted:  
1. Documented enrollment in the Usual Care Treatment Group of the HOPE-Duchenne trial 
and completion of trial follow-up through Month 12.  
2. Willing and able to provide informed consent to participate in the trial if ≥ 18 years of 
age, and assent with parental or guardian informed consent if < 18 years of age   
3. Adequate venous access for intravenous CAP-1002 infusions and routine blood 
collections in the judgement of the Investigator  
4. Assessed by the Investigator as willing and able to comply with the requirements of the 
trial 
8.2. 
Subject Exclusion Criteria 
Exclusion criteria will be assessed within 30 days prior to first infusion on Day 1 unless 
otherwise indicated: 
1. Left ventricular ejection fraction (LVEF) < 35% within 6 months of screening 
2. Planned or likely major surgery in the next 6 months after planned first infusion 
3. Risk of near-term respiratory decompensation in the judgment of the investigator, or the 
need for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 
29 mmol/L at screening  
4. History of non DMD-related chronic respiratory disease including, but not limited to, 
asthma, bronchitis, and tuberculosis 
5. Acute respiratory illness within 30 days prior to screening 
6. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products 
7. Treatment with investigational product ≤ 6 months prior to first infusion 
8. History, or current use, of drugs or alcohol that could impair their ability to comply with 
participation in the trial 
9. Inability to comply with the investigational plan and follow-up visit schedule for any 
reason, in the judgment of the investigator 
8.3. 
Screen Failures 
Any subject who provides written informed consent/assent and is ultimately not infused for 
whatever reason will be classified as a screen failure. All subjects, including screen failures, 
must be accounted for in the clinical database.  

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8.4. 
Subject Withdrawal Criteria 
Every effort will be made to have each subject complete all elements of the trial. If a subject has 
started at least one IP infusion and withdraws prior to trial completion, all attempts must be made 
to perform the trial assessments indicated for the Month 6 visit (i.e., final comprehensive visit). 
Criteria for withdrawal from trial participation include the following reasons: 
• A subject may withdraw his consent at any time without prejudice to his care.  
• At the discretion of the Investigator, the subject may be withdrawn from the trial for 
lack of adherence to the investigational plan.  
• A subject may be withdrawn from the trial for an acute reaction to CAP-1002 or other 
safety issue that prevents repeat infusions. In this instance, the subject should be 
followed on trial for at least 3 months after the last CAP-1002 infusion and then 
complete a final comprehensive visit.  

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donor used for CAP-1002 production (i.e., DSA) will be assessed at screening and throughout 
the course of the trial per Section 13.1.5.   

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reconcile all CAP-1002 received from Capricor’s drug depot. Further information on CAP-1002 
accountability is found in the Investigational Product Manual.    
10.10. 
Handling and Disposal   
All CAP-1002 materials will be disposed following each infusion. Site personnel will follow 
institutional policies on the proper disposal of containers and disposables coming into contact 
with the CAP-1002. Generally, disposal like other biohazard red-bag trash that is ultimately 
incinerated should be sufficient to meet local institutional policies and any other regulations or 
laws. 

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11.
ASSESSMENT OF EFFICACY
Assessments to evaluate efficacy will be performed at trial visits as indicated in Table 3.  
11.1. 
Sequence of Assessments 
Subjects will complete assessments in the following sequence and prior to IP infusion (if 
applicable):  
•
Safety and other trial assessments, excluding blood and urine collections (e.g., vital
signs, height, weight, 12-lead ECG, physical exam etc.)
•
Pulmonary function testing
•
PUL 1.2
•
6MWT
•
Blood and urine collections
•
Cardiac MRI
At the Month 3 follow-up visit, subjects will complete the efficacy assessments designated 
for that visit prior to CAP-1002 administration. 
11.2. 
Efficacy Assessment Training 
Site personnel must complete trial-specific training prior to conducting efficacy assessments for 
the trial. Standardization and consistency are essential. All efforts must be made to have the 
same clinical evaluators conduct the PFT, PUL, and 6MWT assessments and the same imaging 
technologist conduct the cardiac MRI for a subject throughout the duration of the trial. 
Additional details regarding training requirements can be found in Section 17.1 and the trial’s 
Manual of Procedures (MOP).  
11.3. 
Performance of the Upper Limb 
The Performance of the Upper Limb (PUL) was designed specifically for assessing upper limb 
function in ambulant and non-ambulant DMD patients (Pane et al., 2014). All the tasks included 
in the PUL were selected to address patient prioritized activities of daily living that are typical 
regardless of age, including preschool children. The PUL includes an entry item to define the 
starting functional testing level for a subject. The remaining PUL items are divided into three 
regional dimensions: high-level (shoulder), mid-level (elbow), and distal-level (wrist and hand). 
Trial subjects will complete testing of the 1.2 module using the same preferred arm throughout 
the course of the trial when PUL testing is required.  
Elbow contractures will be measured in each arm of a subject prior to the start of PUL testing at 
each visit that requires testing.  
PUL equipment will be standardized across investigative sites and provided by Capricor. 
Additional details regarding the PUL requirements can be found in the trial’s MOP.  

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11.4. 
Pulmonary Function Testing 
Pulmonary function testing (PFT) in this trial will measure slow vital capacity (SVC), forced 
vital capacity (FVC), forced expiratory volume in one second (FEV1), peak expiratory flow 
(PEF), maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP), peak cough 
flow (PCF), and inspiratory flow reserve (IFR). PFTs will be performed using equipment that 
meets or exceeds the minimal performance recommendations of the American Thoracic 
Society/European Respiratory Society and that will be provided by a centralized core lab.  
Site personnel must complete a certification exam prior to conducting PFTs with a trial subject.  
Subjects will complete PFTs in the seated position prior to any other functional outcome 
measurements (i.e., PUL 1.2, 6MWT, and cardiac MRI). Every effort should be made to 
complete a subject’s PFTs at approximately the same time in the morning at each trial visit. 
Additional details regarding each PFT technique and requirements are included in the study 
reference manual provided by the centralized core lab. This manual should be followed for all 
pulmonary function testing.   
11.4.1. 
Slow Vital Capacity  
SVC maneuvers will be performed prior to spirometry (forced maneuvers) because of the 
potential for muscular fatigue and volume history effects.  
Subjects will perform at least three acceptable SVC maneuvers (with no more than five total 
attempts) until the highest SVC is no more than 0.150 L greater than the next highest 
measurement. The largest acceptable SVC value will be reported.  
11.4.2. 
Spirometry 
At least three acceptable spirometry efforts (with no more than five attempts) will be obtained 
for FEV1, FVC, and PEF determinations. Acceptable forced maneuvers will have a satisfactory 
start of test and end of test (i.e., a plateau in the volume-time curve) and be free from artifacts 
due to cough, early termination, poor effort, obstructed mouthpiece, equipment malfunction, or 
other reasons. 
Quality control standards will be implemented within a testing session. The largest FEV1, FVC, 
and PEF from the 3 acceptable efforts will be reported, even if they do not come from the same 
effort. The two largest accepted FVC values should be within ± 15% of each other. Additionally, 
the two largest accepted PEF values should be within ± 15% of each other.  
11.4.3. 
Maximal Inspiratory & Expiratory Pressures 
Maximal inspiratory pressure (MIP) and maximal expiratory mouth pressure (MEP) are simple 
tests in which patients generate as much inspiratory or expiratory pressure as possible against a 
blocked mouthpiece. Because lung volume cannot change significantly during measurement, 
results are to a large extent independent of the properties of the lungs. They are general tests of 
neuromuscular function of the combined diaphragm, abdominal, intercostal, and accessory 
muscles (Evans and Whitelaw, 2009). 
Subjects will perform the MIP maneuvers (from residual volume (RV)) first, and the MEP 
maneuvers (from total lung capacity (TLC)) second.  

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There must be at least 3 acceptable maneuvers from a maximum of five per session.  
The two largest accepted values should be within ± 15% of each other.  
Subjects will sustain maximal efforts for a total of 3-4 seconds until a plateau in pressure is 
achieved for at least 1.5 seconds.  
Site personnel must carefully judge outlier efforts, which need to be eliminated if the pressure 
reading showed no gradual increase to the plateau value, suggesting a sharp peak value. 
11.4.4. 
Peak Cough Flow 
Subjects will perform at least three cough maneuvers (with no more than five total attempts) 
from TLC for PCF determination. Site personnel must evaluate each maneuver to determine if it 
meets acceptability criteria. The largest, acceptable PCF value will be reported. 
11.4.5. 
Inspiratory Flow Reserve 
Subjects will perform at least three maximum inspiratory flow-volume maneuvers (with no more 
than five total attempts).  From stable tidal breathing, subjects will expire to RV and then inspire 
to TLC with maximum effort. Site personnel must evaluate each maneuver to determine if it 
meets acceptability criteria. The largest, acceptable inspiratory flow reserve value will be 
reported. 
11.5. 
6-Minute Walk Test  
Subjects assessed by the Investigator as capable of performing the 6MWT will do so on Day 1 
and the protocol-specified follow-up visits. Subjects unable to perform the 6MWT on Day 1 will 
not be required to perform the test at any subsequent visit. Detailed information on performing 
the 6MWT are found in the trial MOP.  
11.6. 
Cardiac MRI 
Subjects will undergo cardiac MRI at the Screening and Month 6 visits (or, Early Termination 
Visit) if they are physically capable as determined by an Investigator. Subjects that complete the 
cardiac MRI will have sufficient attention span, ability to maintain a breath-hold, lack significant 
contractures that would otherwise make lying flat difficult and fit properly within the MRI 
scanner.  
A subject’s screening cardiac MRI case will require evaluation by a site’s radiologist using 
institutional procedures. The reported LVEF from the local interpretation will be used to assess 
eligibility (exclusion criterion 1). 
It is anticipated that the duration of each MRI session will be 45-60 minutes. Acquisition 
parameters and techniques are specified in the MRI Imaging Manual. The site-based MRI 
technologists will be trained and certified in the common acquisition protocol prior to initiation 
of enrollment at each site. All trial images and applicable imaging data will be sent to and 
centrally read at an independent, central, imaging core by DMD cardiovascular MRI and 
imaging experts.  
Subjects that cannot complete a cardiac MRI as part of the trial will complete an echocardiogram 
at screening using the site’s local equipment, acquisition protocol, and evaluation procedures. 

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The reported LVEF from the local interpretation will be used to assess eligibility (exclusion 
criterion 1).    

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12. 
OTHER TRIAL ASSESSMENTS 
Other trial assessments will be performed as indicated in Table 3.  
12.1. 
Medical Status Questionnaire 
Subjects will be evaluated for the following at Day 1 and throughout the course of the trial: 
• Frequency of manual/power wheelchair use 
• Age permanently transitioned to a wheelchair full time 
• Ventilatory support 
• Frequency of falls 
12.2. 
Medical History 
Relevant and significant medical/surgical history will be confirmed at screening.  
12.3. 
Prior and Concomitant Medications 
All medications taken within one month prior to the screening visit through trial completion will 
be captured. Medications will be reviewed at each trial visit and any medication changes, 
including new and discontinuations, will be recorded.  
For each medication, generic name, indication, dose, frequency, route, and starting and stopping 
dates/times (if applicable) will be collected. The trade name of the medication is to be reported 
for combination therapies (e.g., Alka-Seltzer, Advair, etc.). 
All efforts should be made to maintain the same DMD medication regimen throughout the 
course of the trial. This should be discussed with the subject’s primary medical doctor and 
caregivers at the outset of the trial and during the trial follow-up period.   
12.4. 
Planned Medical/Surgical Procedures 
Data associated with any elective medical and/or surgical procedure (e.g., wisdom tooth 
extraction) that is not the result of an adverse event will also be captured.   
For each procedure, the type of procedure, indication, and start date/time will be recorded. Any 
administered medication(s) related to the planned medical/surgical procedure(s) will be captured 
as a concomitant medication.  

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13. 
ASSESSMENT OF SAFETY 
13.1. 
Safety Parameters 
Safety assessments will be performed as indicated in Table 3. 
13.1.1. 
Vital Signs 
Heart rate, systolic and diastolic blood pressure, respiratory rate, body temperature, and blood 
oxygen saturation (SpO2) will be measured after the subject has rested for approximately 5 
minutes and before performing ECG and spirometry testing. A single set of values will be 
captured.  
A subject’s SpO2 will be monitored for at least 30 minutes following each CAP-1002 infusion 
(see Section 10.7). 
Vital signs will be performed using equipment provided by investigative sites that has been 
properly calibrated per institutional guidelines.  
13.1.2. 
Weight and Height 
Weight and height measurements will be performed.  
Investigative sites will make every effort to perform a weight measurement without the subject’s 
wheelchair or other assistive device (e.g., walker), if applicable.  
If standing height cannot be measured, height will be calculated using a measurement of ulna 
length (Gauld et al., 2004). It is critical to reduce variability in ulna length measurements across 
investigative sites. Therefore, site personnel must complete trial-specific training prior to 
measuring a subject’s ulna length for the purposes of calculating height. 
Table 5: 
Height Calculation from Ulna Length 
Height (cm) = [4.605 x Ulna Length (cm)]  
 
+ [1.308 x Age (years)] 
 
+ 28.003  
Centimeter and year entries must include decimal places. If a subject is 18 years or 
older, enter “18” in the formula for age.  
13.1.3. 
Physical Examination 
The physical exam (PE) is not considered a standard of care assessment as the examiner will be 
assessing for research events. Therefore, the physical exam is to be conducted only by an 
Investigator, or designated site personnel (e.g., nurse practitioners, physician assistants, research 
fellows) listed on the Delegation of Authority Log.    
The physical examination will be a review of the major organ systems including: general 
appearance, HEENT, lymphatic, respiratory, cardiovascular, chest, abdomen, gastrointestinal, 
and musculoskeletal.  
Clinically significant findings prior to investigational product administration are to be captured 
as medical history.  

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Clinically significant findings after the start of investigational product administration are 
captured and reported as AEs, if they meet the definition of an AE per Section 13.2.1. 
13.1.4. 
Electrocardiogram (ECG) 
All ECG measurements will be obtained in the supine position with standardized equipment that 
meet institutional standards for clinical use. Site personnel must complete trial-specific training 
prior to ECG measurements. A 12-lead ECG measurement and rhythm strip (10 seconds) will be 
obtained after measurement of vital signs and before spirometry testing.  
The Investigator, a designated Sub-Investigator, or other appropriately trained site personnel will 
be responsible for performing 12-lead ECG assessments. The Investigator must provide his/her 
dated signature on the original paper tracing, attesting to the authenticity of the ECG machine 
interpretation, or their over-read and reinterpretation. 
ECG data will be electronically transmitted to an independent cardiologist, contracted by 
Capricor, and evaluated. The independent cardiologist, blinded to treatment assignment, will 
conduct an over-read of the ECG measurements required for the trial. Investigative sites will 
receive a report with the independent cardiologist’s ECG interpretations that must be reviewed, 
signed, and dated by an Investigator.  
13.1.5. 
Immunologic Assessments 
Serum for DSA testing and whole blood for HLA typing will be collected following the standard 
institutional procedures for blood collection and submitted to a central laboratory for analysis. 
Instructions on collection, processing, storage and shipping the samples to the central laboratory 
are located in the Laboratory Manual.      
DSA collection for the Week 4 and Month 4 (±7 days, respectively) visits will occur either at the 
investigative site or remotely at a designated central laboratory patient service center.   
DSA samples may also be collected in the setting of clinical suspicion of immune sensitization 
syndrome in the judgment of the Investigator. See Section 7.2.1 for additional details regarding 
immune sensitization syndrome as a safety endpoint.  
13.1.6. 
Clinical Laboratory Assessments 
All blood samples are to be collected following the standard institutional procedures for blood 
collection and submitted to a central laboratory for analysis.  
Instructions on collection, processing, storage and shipping the samples to the central laboratory 
are located in the Laboratory Manual. 
13.1.6.1. Hematology 
Hematological testing will include complete blood count (CBC) with white blood cell (WBC) 
differential, hemoglobin, hematocrit, and platelet count 
13.1.6.2. Serum Chemistry 
Serum chemistry testing will include: basic metabolic panel (Glucose, Sodium, Potassium, 
Chloride, Bicarbonate, BUN, Creatinine, Calcium), comprehensive hepatic panel (Albumin, 

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Alkaline Phosphatase, Total Protein, ALT, AST, Direct Bilirubin, Total Bilirubin), and Creatine 
Kinase 
13.1.6.3. Cardiac Enzymes 
Cardiac enzyme testing will include CK-MB and Troponin I 
13.2. 
Adverse and Serious Adverse Events 
13.2.1. 
Definition of Adverse Events 
13.2.1.1. Adverse Event (AE) 
An AE is any untoward medical occurrence associated with the use of a drug in humans, whether 
or not considered drug related. It may be indicated by physical sign, symptom, clinically 
significant laboratory abnormalities, and/or disease temporally associated with a medical 
(investigational) treatment, procedure, or product, whether or not related to the medical 
(investigational treatment, procedure or product.  This definition includes intercurrent illnesses 
or injuries, exacerbation of pre-existing conditions, or events occurring due to abuse or overdose.   
Any condition that was pre-existing is not an adverse event unless there is a change in the nature, 
severity, or degree of the condition.  
Clinical laboratory abnormalities are considered AEs when deemed clinically significant by the 
Investigator and/or lead to a change in the subject’s functional status.  
An AE does not include:  
• Medical or surgical procedures (e.g., colonoscopy, biopsy). The medical condition 
that leads to the procedure is an AE. 
• Social or convenience hospital admissions where an untoward medical occurrence did 
not occur. 
• Day-to-day fluctuations of pre-existing disease or conditions present or detected at 
the start of the trial that do not worsen. 
• The disease/disorder being studied or expected progression, signs, or symptoms of the 
disease/disorder being studied unless more severe than expected for the subject’s 
condition. 
All Investigators conducting investigative studies supported by Capricor must report both 
expected and unexpected SAEs to Capricor, or designee, and their individual Institutional 
Review Board (IRB) in compliance with their institutional policies. Please see Section 13.2.4 or 
further details on event reporting.  
13.2.1.2. Serious Adverse Event (SAE) 
An AE is considered “serious” if, in the view of either the Investigator or Capricor, it results in 
any of the following outcomes:  
• Death 
• Life-threatening adverse event 

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• Inpatient hospitalization or prolongation of existing hospitalization 
• Persistent or significant incapacity or substantial disruption of the ability to conduct 
normal life functions 
• Congenital anomaly/birth defect 
Important medical events that may not result in death, be life-threatening, or require 
hospitalization may be considered serious when, based upon appropriate medical judgment, they 
may jeopardize the subject and may require medical or surgical intervention to prevent one of the 
outcomes listed in this definition.  
Examples of such medical events include bronchospasm requiring intensive treatment in an 
emergency room or at home, blood dyscrasias, or convulsions that do not result in inpatient 
hospitalization, or the development of drug dependency or drug abuse.   
Complications that occur during a hospitalization are AEs.  When the hospitalization is 
prolonged due to the complication or the complication fulfills any other serious criteria, the event 
is reported as an SAE.  Hospitalization for elective treatment of a pre-existing condition that did 
not worsen from baseline is not considered an SAE.  
13.2.1.3. Expected Adverse Events 
As CAP-1002 is comprised of allogeneic cells, immunologic reactions to the product are a 
possible adverse event. However, to date, no significant immune system adverse events have 
been identified with any likelihood of relationship to CAP-1002.  
There is some risk of developing transient DSA with the CAP-1002 allogeneic product. All 
subjects will be monitored during the trial for immune sensitization as outlined in Table 3. 
Other risks of the infusion procedure include those risks that are possible with the intravenous 
administration. These include risks related to infection, bleeding, pain, and bruising and/or 
hematoma at the vascular access site(s).   
13.2.1.4. Unexpected Adverse Events 
An AE is considered “unexpected” if it is not listed in the Investigator’s Brochure or is not listed 
at the specificity or severity that has been observed, if the Investigator’s Brochure is not required 
or available, or if it is not consistent with the risk information described in the general 
investigational plan or elsewhere in the current application. 
“Unexpected,” as used in this definition, also refers to adverse events or suspected adverse 
reactions that are mentioned in the Investigator’s Brochure as occurring with a class of drugs or 
as anticipated from the pharmacological properties of the drug, but are not specifically 
mentioned as occurring with the particular drug under investigation. 
Expedited reporting is required for serious unexpected AEs as discussed in Section 13.2.4.   
13.2.1.5. Other Adverse Event (OAE) 
OAEs will be identified by the Medical Monitor during the evaluation of safety data for the 
Clinical Study Report.  Significant adverse events of particular clinical importance, other than 

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SAEs and those AEs leading to discontinuation of the subject from the trial, will be classified as 
OAEs.   
For each OAE, a narrative may be written and included in the Clinical Study Report. 
13.2.2. 
Relationship to Investigational Product 
The Investigator will assess the relationship (causality) of an AE to the investigational product 
and administration procedure.  
Causality will be defined as follows:  
• Probable: adverse events that, after careful medical evaluation, are considered with a 
high degree of certainty to be related to the investigational product or administration 
procedure. The following characteristics will apply: 
− A reasonable temporal relationship exists between the event and the 
investigational product or administration procedure, and 
− The event is a known reaction to the investigational product or administration 
procedure, which cannot be explained by an alternative etiology commonly 
occurring in the population/individual.  
• Possible: adverse events that, after careful medical evaluation, do not meet the criteria 
for a probable relationship to the investigational product or administration procedure, 
but for which a connection has reasonable certainty. The following characteristics 
apply: 
− The event occurs after exposure to the investigational product or administration 
procedure, and  
− The event is not a known reaction to the investigational product or administration 
procedure, but cannot be explained by a commonly occurring alternative etiology, 
or 
− In the absence of a temporal relationship, the event cannot reasonably be 
explained by an alternative etiology.  
• Unlikely: adverse events that, after careful medical evaluation, do not meet the 
criteria for possible or probable relationship to investigational product or 
administration procedure and for which a connection is unlikely. The following 
characteristics will apply: 
− The event does not follow a reasonable temporal sequence from administration of 
the investigational product or administration procedure, or  
− May be explained by commonly occurring alternative etiology in the 
population/individual, or  
− May have been produced by environmental factors, and there is no apparent 
pattern of response to the investigational product or administration procedure.  
An adverse event will be reported as “related” when causality is evaluated by an Investigator as 
probably or possibly related to the investigational product and/or the administration procedure. 

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Related adverse events indicate a potential cause-and-effect relationship between the 
investigational product and/or administration procedure and the occurrence of the adverse event.  
An adverse event will be reported as “unrelated” when causality is evaluated by an Investigator 
as unlikely related to the investigational product and/or administration procedure by the 
Investigator. Unrelated adverse events indicate no relationship between the occurrence of the 
adverse event and the investigational product and/or administration procedure.  
13.2.3. 
Recording Adverse Events 
Investigators will monitor all subjects for AEs during the trial and establish a diagnosis for an 
event based on signs, symptoms, and/or other clinical information. It is important to distinguish 
that individual signs and symptoms of the event are not adverse events and should not be 
reported.  
For each AE, the Investigator will evaluate the causality and severity, report the action taken and 
event outcome and disclose whether or not it caused the subject to discontinue trial participation. 
The following severity scale will be used as a guideline to differentiate the severity of adverse 
events:  
• Mild (Grade 1): Transient or mild discomfort; no limitation in activity; no medical 
intervention/therapy required 
• Moderate (Grade 2): Mild to moderate limitation in activity – some assistance may 
be needed; no or minimal medical intervention/therapy required 
• Severe (Grade 3): Marked limitation in activity, some assistance usually required; 
medical intervention/therapy required and often requiring hospitalization or 
prolongation of hospitalization 
• Life-Threatening or Disabling (Grade 4): Extreme limitation in activity, significant 
assistance required; significant medical intervention/therapy required; hospitalization, 
prolongation of hospitalization, or hospice care 
• Fatal (Grade 5) 
An AE that is assessed as severe should not be confused with an SAE. Severity is a category 
utilized for rating the intensity of an event. An event is described as “serious” when it meets one 
of the pre-defined outcomes noted in Section 13.2.1.2. Both an AE and SAE can be assessed as 
severe. However, an AE of severe intensity may not meet SAE definition requirements.  
13.2.4. 
Reporting Adverse Events 
All adverse events are collected from the time of signing informed consent for trial participation 
until completion of 6 months or early termination, whichever occurs first. All AEs occurring 
after the initiation of the IV catheter placement for the initial dose of CAP-1002 will be 
considered treatment emergent. Any ongoing adverse event that has not been resolved at the time 
of trial completion or early termination for a subject will be marked as ongoing on the adverse 
event case report form (CRF).  
All AEs will be entered into the electronic data capture (EDC) system by trained site personnel at 
the investigative site.   

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Expected and unexpected SAEs must be reported to Capricor and entered into the EDC system 
within 24 hours of discovery of the event. For events that do not have complete information 
available at the time of initial report, the investigative site will submit all available information at 
the time of the submission. All SAE Report Forms must be signed by an Investigator and 
submitted with available source documentation. All source documentation must be de-identified 
prior to submission.  
ALL SAES and subsequent follow-up information must be reported to Capricor, or designee, via 
email or fax as outlined in the Manual of Procedures and Safety Plan.  
All SAEs must be reported to the respective IRB in accordance with the investigative site’s 
policies. Copies of the submission will be collected by Capricor.   
All SAEs will be reported to the DSMB at least semi-annually, or more frequently at the 
discretion of the Medical Monitor.  
Capricor will promptly upon discovery, report serious and unexpected adverse events for which 
there is a reasonable possibility that the investigative therapy (i.e. administration product and/or 
investigative product) caused the events, to the Food and Drug Administration (FDA) in 
accordance with 21 CFR 312.32 regulations and ICG E2A guidelines.  
For trials conducted under an investigational new drug (IND) application, FDA regulations 
require reporting of any suspected adverse reaction. A serious adverse reaction is defined as any 
adverse event for which there is a reasonable possibility that the drug/biologic caused the AE. 
For the purposes of IND safety reporting, “reasonable possibility” means there is evidence to 
suggest a causal relationship between the drug/biologic and the adverse event. Serious, 
unexpected suspected adverse reactions (SUSARs) are SAEs that are unexpected and are 
possibly or probably related to participation in the research. Expedited reporting is required for 
all SUSARs. Capricor will send an initial Expedited Safety Report (ESR) or MedWatch Form 
3500A to the FDA within 5 business day of determination that the event qualifies for reporting.  
13.2.5. 
Pregnancy Reporting 
Should a female be impregnated by a trial subject, investigative sites are required to notify 
Capricor within 24 hours of learning about the pregnancy. The investigative site will receive the 
Pregnancy Reporting Form to complete and submit to Capricor, or designee.  
All pregnancies will be followed until the pregnancy outcome is known. In addition, pregnancies 
that are ongoing at the time of trial completion will be followed until the outcome is known. The 
investigative site is responsible for outcome reporting via the Pregnancy Reporting Form, 
including spontaneous or voluntary termination, details of birth, and the presence or absence of 
any birth defects, congenital abnormalities, or maternal and newborn complications.     
The pregnancy is not an adverse event for the male subject unless there is a suspicion that the 
investigational product interfered with the effectiveness of a contraceptive medication. 
13.2.6. 
Adverse Event Follow-up 
All AEs are followed by the investigative site until an outcome is known or the subject’s 
participation in the trial concludes at either 6 months or early termination, whichever occurs first.  

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The investigative site is expected to review all ongoing AEs at each visit. AEs are followed until 
resolution or until no further changes in the event are expected (i.e., the point at which a subject 
experiencing an AE is treated successfully and stabilized even though he may continue to 
experience lingering sequelae that may never resolve), or it is agreed that further follow-up of the 
event is not warranted (e.g., non-serious, IP unrelated, ongoing at final visit). See Section 13.2.3 
for reporting ongoing AEs at the end of trial participation.   
For SAEs that were incomplete or ongoing at the time of initial submission, the investigative site 
is required to submit follow-up SAE Report Forms when event information is available to the 
research site personnel and/or an outcome is known.  

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14. 
STATISTICS 
14.1. 
General Considerations 
1. Descriptive statistics will be used to summarize all subject baseline and outcome data 
collected during the trial. Continuous variables will be summarized using mean, standard 
deviations, medians, and ranges. Categorical variables will be summarized in frequency 
distributions or as rates.  
2. Statistical analyses will be performed by validated software (e.g., SAS, IBM/SPSS, or 
Cytel Software). 
3. There is no formal hypotheses or statistical tests associated with trial endpoints. 
Statistical tests will be performed, however, for informational value as part of 
summarizing trial results. Parametric and non-parametric tests will be used that are 
appropriate to the outcome measure being summarized. Reported p-values will be 
considered nominal and unadjusted for multiple testing, without conclusions regarding 
statistical significance levels.  
4. Copies of databases used to prepare clinical report summaries will be archived to enable 
any statistical analyses performed to be replicated.  
5. A full data listing will be prepared, including an electronic version in a standard 
computer-accessible format (e.g., SAS) at the completion of the study. Listings of data 
represented on the case report forms (eCRF) will be provided for all key baseline, 
demographic and outcome variables to facilitate further investigation of tabulated values 
and to allow for clinical review of safety variables. 
14.2. 
Sample Size 
Up to 10 subjects that were randomized to Usual Care Treatment Group and completed the 12-
month follow-up period for the HOPE-Duchenne trial will be enrolled in this trial.   
14.3. 
Analysis Population 
Intent-to-Treat (ITT) Population:  All subjects who meet the enrollment criteria and are enrolled 
in the study.  These subjects will also constitute the Safety population. 
Per Protocol (PP) Population:  All enrolled subjects who have no protocol deviations/violations 
that could significantly impact the completeness, accuracy and/or reliability of the trial data. The 
list of subjects in the per protocol population will be compiled prior to database lock. 
14.4. 
Safety Analysis 
14.4.1. 
Safety Endpoints 
All primary safety endpoints and other observed adverse events will be documented and 
reported.   Adverse events will be summarized by the incidence of events by type and by the 
percentages (rates) of subjects with those events.   Primary safety endpoints that are adjudicated 
by the Clinical Events Committee (CEC) will be further described by the relatedness to the study 
treatment and severity level.  

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14.4.2. 
Medications 
A listing of prescription and over the counter medications used by individual trial subject will be 
prepared, including information on generic name. dosage, frequency of administration, reason 
for use, observed side effects, and any related adverse events.  Study level summaries by type of 
medication may also be prepared. 
14.4.3. 
Clinical Laboratory Evaluations 
All laboratory evaluations will be documented.   A listing of results by subject will be prepared, 
with any abnormalities noted at baseline or during the follow-up period. 
14.4.4. 
Donor-Specific Antibodies 
Donor-specific antibodies (DSA) will be assessed as screening and periodically during the 
follow-up period or upon clinical suspicion of an immune sensitization response, and will be 
summarized by the specific CDC donors contributing to the CAP-1002 doses used by individual 
subjects.  
14.4.5. 
Physical Examination 
Medical status and baseline physical exam results, including vital signs, 12-lead ECG, and 
weight, will be summarized using descriptive statistics and any observed abnormalities.  Any 
changes over the follow-up period will be documented and reported. 
14.5. 
Efficacy Analysis 
Efficacy will be evaluated at the 3 and 6-month follow-up visit, with changes from baseline 
assessed for pre-specified exploratory endpoints.  Analyses will be performed both in the ITT 
and PP populations. 
14.5.1. 
Exploratory Efficacy Endpoints 
Descriptive statistics will be used to summarize changes from baseline in the PUL 1.2 scores 
(including the mid-level dimension, combined mid and distal-level dimensions, and high-level 
dimension), in left ventricular structure and function parameters (including ejection fraction, wall 
thickness, end-diastolic and end-systolic volumes, stroke volume, and circumferential strain, in 
pulmonary function (including SVC, FEV1, FVC, MIP, MEP, PCF, and IFR) and in 6-minute 
walk test distance for ambulatory subjects.  
14.6. 
Statistical Methods 
All subject baseline and study outcomes for safety and efficacy will be summarized using 
descriptive statistics.  These descriptive statistics and any exploratory statistical analyses will be 
appropriate to the data type involved.   
Mean changes from baseline in continuous measures (PUL 1.2, cardiac parameters, and 
pulmonary parameters) will be evaluated as a function of follow-up time.   Variability in 
outcomes will be summarized with the associated 95% confidence intervals for the mean 
estimates.  Statistical tests may be performed to assess whether observed mean changes are 
different than zero, but without assignment of statistical significance. 

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Additional analyses may be performed to examine the relationship of patient factors to study 
outcomes, to estimate the correlations between different study outcomes, or to examine the 
consistency of outcomes between subject subgroups or study sites. 
Standard statistical tests for outliers may be performed in those cases where suspect lab tests or 
study outcomes are observed. 
14.6.1. 
Multiplicity 
There are no pre-specified test hypotheses or assignments of statistical significance to study 
results requiring a consideration of adjustments for multiple testing.  
14.6.2. 
Missing Data 
There are no plans for imputation of missing data.  The reasons for missing data will be 
documented.  Sensitivity analyses, however, may be performed using various assumptions for 
missing data to assess the impact on summary results. 

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15. 
TRIAL OVERSIGHT  
15.1. 
Steering Committee 
The Steering Committee will provide the overall scientific direction for the trial. The 
responsibilities of the Steering Committee are to: (a) maintain contact with trial Investigators to 
ensure high quality data collection; (b) approve and implement major protocol changes; (c) 
collaborate in data analysis, interpretations, and publications; (d) establish criteria for authorship 
on all manuscripts, publications and presentations that arise from the trial.  
15.2. 
Clinical Events Committee (CEC) 
The purpose of CEC adjudication is to provide consistent and unbiased adjudication of clinical 
outcomes and specified events through the independent review of source documentation. The 
charge of the CEC is to review source documents and to adjudicate the classifications of all 
potential primary safety endpoint events. Sites will be provided instructions in the MOP on how 
to collect and submit event information required for CEC review. The CEC will remain blinded 
to subject treatment assignments.  The individuals that serve on the committee will be appointed 
by Capricor, are independent from all other trial activities, and are not affiliated with any 
investigative site. The committee will consist of, at a minimum, a neurologist and pulmonologist 
with experience in treating patients with DMD. Additional experts in immunology will be 
consultants to the committee as necessary. The frequency of CEC meetings is detailed in the 
CEC Charter.  
15.3. 
Data Safety Monitoring Board 
To meet the trial's ethical responsibility to its subjects, an independent Data Safety Monitoring 
Board (DSMB) will monitor results during the trial.  The board consists of physicians and 
biostatistician(s) appointed by Capricor, who have no formal involvement or conflict of interest 
with the Investigators, investigative sites, subjects, or Capricor. The DSMB will act in a senior 
advisory capacity to Capricor regarding data and safety matters throughout the duration of the 
trial. The board will meet on a periodic basis according to the DSMB charter to monitor the 
available information regarding safety, efficacy, and quality of trial conduct. The DSMB will 
communicate their recommendations directly to Capricor. The investigative sites will have no 
contact with the members of DSMB and no voting member of the committee may participate in 
the trial as an Investigator. 

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16. 
SOURCE DATA AND TRIAL DOCUMENTS 
16.1. 
Electronic Data Capture 
All trial data will be entered into the EDC system. Site personnel requiring access will have their 
own Login/Password. Access to trial information will be based on individual roles and 
responsibilities. The application employs fine-grained role-based access control for data entry, 
viewing and reporting options. All trial data will be transmitted over an encrypted SSL (Secure 
Sockets Layer) connection that requires user authentication.  
This application is designed to be in full compliance with the International Conference on 
Harmonization and Good Clinical Practices (ICH-GCP), the FDA’s CFR 21 Part 11 Electronic 
Record and Electronic Signatures, the FDA’s “Guidance: Computerized Systems Used in 
Clinical Studys,” and Health Insurance Portability and Accountability Act (HIPAA).  
EDC supports efficient data collection and management and facilitates rapid data closure. A 
strong advantage of web-based design is that Capricor, or designee, has immediate access to the 
data from all investigative sites so that queries can be generated and distributed to the sites in 
real-time and the frequency of missing data can be reduced.     
16.2. 
Trial Monitoring 
In accordance with 21 CFR 312.56, ICH- GCP, and local regulations, trial monitors will 
periodically complete on-site monitoring of data with a focus on safety, trial endpoints, data 
completion, data outliers and data integrity. Trial monitors will schedule an on-site visit with a 
site coordinator for an appropriate duration based on the scope of data anticipated to be collected. 
Prior to the visit, a confirmation letter will be sent to the investigative site, which will include a 
listing of which CRFs and source documents will be reviewed.  
Site coordinators are to have all source documents up to date and easily accessible to a monitor.      
During an on-site monitoring visit, trial monitors are to:  
• Track the overall monitoring process including data collected and entered, visit 
schedules, and subject screening and enrollment. 
• Verify and ensure compliance with the protocol according to GCP and HIPAA 
requirements. 
• Ensure that appropriate data corrections are made, dated, explained, and initialed by 
the Investigator or representative. 
• Assess the impact of any personnel changes on the investigative site’s ability to 
conduct the trial.  
• Verify a minimum of the following data points for all subjects: date of birth, signed 
informed consent, eligibility criteria, medical history, date of enrollment, serious 
AEs, and mortalities.  
• Perform review of informed consent process and review documentation of informed 
consent for completeness and correctness.  

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• Perform on-site validation checks of recoded data by reviewing source documents to 
determine whether the data reported in the EDC system are complete and accurate. 
Source documents include medical charts, screening records and/or logs, research 
procedure records and/or files, and other trial related notes.  
• Monitor subject safety by verifying that any AE, therapy modification, or 
concomitant medications are reported in accordance with the protocol.  
• Determine whether all AEs, protocol deviations, and protocol violations are 
appropriately reported within the required time periods according to applicable 
regulatory requirements as outlined in the protocol and by regulatory agencies.  
• Verify that any missed visits, tests, and examinations that were not performed, as well 
as trial withdrawals and/or dropouts are explained and clearly reported. 
• Inform the site PI about any deviations from or violations of the trial protocol, GCP 
and/or regulatory requirements in order for appropriate actions to be taken to prevent 
recurrence of the deviation and/or violation.  
• Infor the Investigator of any major data entry error, delays in data entry, omissions, or 
eligibility requirement errors.  
• Verify that regulatory documentation is accurate, complete, current, and properly 
maintained.  
• Very that PI oversight of trial conduct is documented via signature or initials and date 
on documentation regarding eligibility, AEs, and abnormal laboratory values.  
The monitor may also inspect the investigative site’s facilities to verify that proper space for 
study documents, equipment and investigational product is available.  
A debriefing meeting with the PI, site coordinator(s), and the Monitor to review any notable 
findings will be scheduled toward the end of the on-site visit. The monitor will submit a written 
monitoring visit report to Capricor and send a follow-up letter with findings to the PI. The report 
and letter will include a summary of documentation reviewed by the monitor, any significant 
findings, AEs, protocol deviations and violations, missing regulatory documents and actions 
taken, to be taken or recommended to ensure compliance. The report and letter will include a list 
of action items. Investigative sites are expected to complete the list of actions items within 30 
days of receipt.  
16.2.1. 
Source Document Requirements 
It is highly recommended that the investigative site uses the CRFs and schedule of assessments 
(Table 3) to develop a plan for identifying and standardizing where source documentation for 
data verification will be collected across all trial participants at their site. As part of trial start-up 
and prior to first enrollment, investigative sites are encouraged to conduct a gap analysis to 
identify any data points that are not routinely documented in the medical record.  
The medical record is the gold standard for source documentation. However, Capricor 
understands that there may be data points required for this trial that are not collected as routine 
practice at the investigative site in the medical record for this patient population. Investigative 
sites may use the provided source document worksheets or create source documents for the 

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purposes of collecting source data that are not included in the medical record. It is important to 
remember that “source” documentation is where the information is first recorded.  
The investigative site must ensure that all subject source documentation is complete, orderly, and 
stored in a secure location. For electronic records, the investigative site should abide by the 
institutional policies for the storage of private health information (PHI). For any paper records 
containing any PHI, the investigative sites must ensure that the files are double-locked, that is, in 
a locked filing cabinet within a locked office or suite.  
All source documentation is to be de-identified of all unique patient and hospital identifiers by 
the investigative site prior to review or submission to Capricor or their designee. 
16.3. 
Audits and Inspections 
Authorized representatives of Capricor, a regulatory authority, an Independent Ethics Committee 
or an Institutional Review Board may visit the site to perform audits or inspections, including 
source data verification.  The purpose of a Capricor audit or inspection is to systematically and 
independently examine all trial-related activities and documents to determine whether these 
activities were conducted, and data were recorded, analyzed, and accurately reported according 
to the protocol, Good Clinical Practice guidelines of the International Conference on 
Harmonization, and any applicable regulatory requirements.  The Investigator should contact 
Capricor immediately if contacted by a regulatory agency about an inspection. 
16.4. 
Retention of Records 
The investigative sites must maintain all documentation relating to the trial for a period of two 
years after the last marketing application approval, or if not approved, two years following the 
discontinuance of the test article for investigation.  If it becomes necessary for Capricor or the 
Regulatory Authority to review any documentation relating to the trial, the Investigator must 
permit access to such records. 

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17. 
QUALITY CONTROL AND QUALITY ASSURANCE 
Capricor will implement and maintain quality control and quality assurance procedures to ensure 
compliance with the protocol, Good Clinical Practices, and all applicable regulatory 
requirements, and may conduct a quality assurance audit(s).  Please see Section 16.3 for more 
details regarding the audit process. 
17.1. 
Qualifications and Trainings 
Clinical Investigators will be physicians with expertise in the clinical care of patients with DMD 
in a multi-disciplinary clinical setting that includes neuromuscular medicine, pulmonary, 
cardiology, and physical therapy.   
Each investigative site MRI lab involved in image acquisition for this trial will be certified by the 
MRI core lab in accordance with the MRI Imaging Manual.  
Each investigative site pulmonary function testing lab involved in conducting pulmonary 
function testing for this trial will be certified by the Pulmonary Function Core Lab as detailed in 
the Manual of Procedures.   
Investigative site personnel delegated to perform the ECG assessment will be certified to 
perform and transmit the assessment by a centralized vendor.   
All Investigators and coordinators will be trained by Capricor, or designee, in the specifics of the 
protocol, investigational product and administration procedure at the site initiation visit in 
advance of the first subject enrollment. The Investigators and coordinators will also undergo a 
separate training to gain familiarity with the electronic data capture system.   
17.2. 
Good Clinical Practices (GCP)  
All Investigators, coordinators and other site personnel involved in care of trial subjects, and/or 
research data collection must provide certification that they have successfully completed their 
institutionally required GCP or other Human Subject Protection courses. 
17.3. 
HIPAA or Other Privacy Training 
All Investigators and coordinators must provide documentation that they have successfully 
completed the institutional requirements to ensure subject rights, privacy and security under 
HIPAA.  
17.4. 
Site Initiation 
IRB approval and the clinical trial agreement between the investigative site and Capricor must be 
signed and executed prior to the site initiation. Additionally, the completed Form FDA 1572, 
applicable CVs and other regulatory documents must be on file with Capricor prior to site 
initiation. A representative from Capricor, or designee, will conduct a site initiation prior to 
enrollment of the first subject. Investigators, study coordinator(s), investigational pharmacist(s), 
infusion suite personnel, clinical evaluator(s), MRI technologist(s), and laboratory personnel will 
be required to attend the initiation. All other site personnel who may be involved in the trial will 
be encouraged to attend.  

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17.5. 
Protocol Deviations 
Efforts to maximize adhere to the protocol will be made through careful and comprehensive 
training, review of trial data collected via the EDC, and routine communication with all site 
Investigators.    
All protocol deviations and violations are to be documented and captured in the EDC. The 
investigative site is responsible for reporting deviations and violations to the IRB per the IRB’s 
reporting guidelines. Capricor will ensure reporting to the proper local and federal regulatory 
authorities in accordance with all applicable federal and local regulations.  
Capricor will determine the course of action based on the severity of the deviation or violation. 
These may include but are not limited to, withdrawal of the subject, additional training at the 
site, additional site monitoring, and/or other appropriate courses of action.  In addition, the 
Medical Monitor and biostatistician will review the circumstances of each deviation and 
violation (in a blinded fashion) to determine whether data can reasonably be included in any trial 
analyses. 

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18. 
ETHICS 
18.1. 
Ethics Review 
Investigative sites are required to follow their institutional guidelines for obtaining initial 
approval by the IRB and for submitting continuing reviews to the IRB. Subject enrollment at an 
investigative site will not commence until initial IRB approval documentation has been received 
and reviewed by Capricor. The composition and conduct of this committee must conform to the 
United States CFR and ICH E6.  
The informed consent must be reapproved in accordance with the investigative site’s IRB 
policies or at least annually.  
Capricor will provide the investigative sites with DSMB approval letters, serious adverse drug 
reactions and any other applicable correspondences during the trial. Investigative sites are to 
follow their institutional policies for reporting these correspondences and documents to their 
IRB.  
All IRB approvals and all materials approved/acknowledged by the IRB for this trial, including 
the subject consent/assent form, recruitment materials, or safety event notifications, must be 
maintained by the Investigator and made available for inspection. 
18.2. 
Ethical Conduct of the Trial 
The trial will be performed in accordance with ethical principles that have their origin in the 
Declaration of Helsinki and are consistent with ICH/Good Clinical Practice, and applicable 
regulatory requirements. Please reference Section 17 further information.  
In accordance with FDA regulatory requirements, 21 CFR 54.4, the Investigators at each site will 
be required to complete a financial disclosure form provided by Capricor prior to participation in 
the trial. Each Investigator shall provide Capricor sufficient accurate financial information to 
allow Capricor to submit complete and accurate certification or disclosure statements (Forms 
3454 and/or 3455) as required by the FDA regulations. Investigators shall promptly update this 
information if any relevant changes occur in the course of the trial or for 1 year following 
completion of the trial 
18.3. 
Written Informed Consent and Assent 
The Investigator is responsible for ensuring that the informed consent process is conducted and 
documented appropriately by trained site personnel. A signed informed consent, which has been 
approved by Capricor and the individual site IRB, is required for trial participation. The consent 
form must incorporate a clinical research authorization for use and disclosure of private health 
information and a release of medical information that authorizes release of medical records to the 
trial Investigators, monitors, and Capricor. The Investigators or designated and qualified 
individual, will provide a thorough explanation of objectives, subject responsibilities, risks and 
benefits of the trial, and will fully address all concerns raised by the subject and/or legal 
guardian. After all issues have been adequately resolved, and the Investigator confirms that the 
subject has been fully consented, the subject or their legal guardian will be asked to sign the 
informed consent. The consent process must be documented in the medical chart and a signed 
copy of the consent and/or assent must be given to the subject and/or legal guardian.  

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18.3.1. 
Obtaining Informed Consent and Assent 
The Investigators at each center will ensure that the subject and legal guardian (applicable only if 
the subject is < 18 years of age) are given full and adequate oral and written information about 
the nature, purpose, possible risk and benefit of the trial.  Subjects and legal guardian must also 
be notified that they are free to discontinue from the trial at any time.  The subject and legal 
guardian should be given the opportunity to ask questions and allowed time to consider the 
information provided. 
The subject must sign and date the informed consent form prior to performing any trial 
procedures. If the subject is <18 years of age, the subject must sign and date the assent form and 
a legal guardian must sign and date the informed consent form prior to performing any trial 
procedures. Specific requirements and guidelines for providing assent will be determined by the 
investigative site’s IRB.   
18.4. 
Subject Confidentiality 
Confidentiality of all subject records will be maintained according to HIPAA guidelines. 
Investigators, investigative site IRBs, Capricor, CEC, and the FDA may review source 
documentation for enrolled subjects as necessary, but all unique patient and hospital identifiers 
will be removed prior to review. If the results of this trial are published, the data will be 
presented in aggregate, with all subject identifiers removed.  

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19. 
PUBLICATION POLICY 
Recognizing the importance of communicating clinical trial results to the public and the medical 
and scientific communities in an accurate and complete manner, the first publication of the trial, 
to include results from all of the investigative centers in a multi-center publication, will be 
authored by the lead or national Principal Investigator, and/or other designees assigned by the 
Steering Committee, for publication in a peer-reviewed scientific journal. All participating 
Investigators, key site personnel, committees and committee members will be listed in an 
appendix as part of the main manuscript.  
An individual Investigator has the right to publish his/her data after the multi-center publication, 
unless no such multi-center publication is so published before the first anniversary of the 
finalization of the multi-center database, in which case the Investigator may publish or submit 
for publication a manuscript without further delay according to the terms and conditions in the 
Clinical Trial Agreement. 
Additional manuscripts targeting exploratory endpoints or other endpoints or data not included in 
the first multi-center publication are anticipated and encouraged. In such cases, the 
Investigator(s) should submit ideas for these additional manuscripts to the Steering Committee 
that will serve as the clearing house to approve topics, ensure that activity between the 
Investigator(s) in analyzing the data is coordinated, prioritize data analyses and help determine 
authorship. 

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20. 
REFERENCES 
1. Aartsma-Rus A., J.C. Van Deutekom, I.F. Fokkema, G.J. Van Ommen, J.T. Den Dunnen 
(2006). "Entries in the Leiden Duchenne muscular dystrophy mutation database: an 
overview of mutation types and paradoxical cases that confirm the reading-frame rule." 
Muscle Nerve 34(2):135-44.  
2. Aartsma-Rus, A. (2014). "Dystrophin Analysis in Clinical Trials." J Neuromuscul Dis 
1(1):41-53. 
3. Aartsma-Rus, A., I. B. Ginjaar and K. Bushby (2016). "The importance of genetic 
diagnosis for Duchenne muscular dystrophy." J Med Genet 53(3): 145-151. 
4. Aminzadeh, M. A., R. G. Rogers, K. Gouin, M. Fournier, R. E. Tobin, X. Guan, M. K. 
Childers, A. M. Andres, D. J. Taylor, A. G. Ibrahim, X. Ding, A. Torrente, J. I. 
Goldhaber, R. A. Victor, R. A. Gottlieb, M. Lewis and E. Marban (2017). Reversal of 
cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-
derived cells and their exosomes in mdx dystrophic mice and in human Duchenne 
cardiomyocytes. http://biorxiv.org/content/early/2017/04/20/128900 
5. Bonios, M., J. Terrovitis, C. Y. Chang, J. M. Engles, T. Higuchi, R. Lautamaki, J. Yu, J. 
Fox, M. Pomper, R. L. Wahl, B. M. Tsui, B. O'Rourke, F. M. Bengel, E. Marban and M. 
R. Abraham (2011). "Myocardial substrate and route of administration determine acute 
cardiac retention and lung bio-distribution of cardiosphere-derived cells." J Nucl Cardiol 
18(3): 443-450. 
6. Bulfield, G., W. G. Siller, P. A. Wight and K. J. Moore (1984). "X chromosome-linked 
muscular dystrophy (mdx) in the mouse." Proc Natl Acad Sci U S A 81(4): 1189-1192. 
7. Bushby, K., R. Finkel, D. J. Birnkrant, L. E. Case, P. R. Clemens, L. Cripe, A. Kaul, K. 
Kinnett, C. McDonald, S. Pandya, J. Poysky, F. Shapiro, J. Tomezsko, C. Constantin and 
D. M. D. C. C. W. Group (2010). "Diagnosis and management of Duchenne muscular 
dystrophy, part 1: diagnosis, and pharmacological and psychosocial management." 
Lancet Neurol 9(1): 77-93. 
8. Bushby, K., R. Finkel, D. J. Birnkrant, L. E. Case, P. R. Clemens, L. Cripe, A. Kaul, K. 
Kinnett, C. McDonald, S. Pandya, J. Poysky, F. Shapiro, J. Tomezsko, C. Constantin and 
D. M. D. C. C. W. Group (2010). "Diagnosis and management of Duchenne muscular 
dystrophy, part 2: implementation of multidisciplinary care." Lancet Neurol 9(2): 177-
189. 
9. Chambers, D. C., D. Enever, S. Lawrence, M. J. Sturm, R. Herrmann, S. Yerkovich, M. 
Musk and P. M. Hopkins (2017). "Mesenchymal Stromal Cell Therapy for Chronic Lung 
Allograft Dysfunction: Results of a First-in-Man Study." Stem Cells Transl Med 6(4): 
1152-1157. 
10. Deconinck, N. and B. Dan (2007). "Pathophysiology of duchenne muscular dystrophy: 
current hypotheses." Pediatr Neurol 36(1): 1-7. 
11. den Dunnen J.T. (2014). "Leiden DMD Mutation Database" www.dmd.nl 

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12. Dumont, N. A., Y. X. Wang, J. von Maltzahn, A. Pasut, C. F. Bentzinger, C. E. Brun and 
M. A. Rudnicki (2015). "Dystrophin expression in muscle stem cells regulates their 
polarity and asymmetric division." Nat Med 21(12): 1455-1463. 
13. Emery, A. E. (2002). "The muscular dystrophies." Lancet 359(9307): 687-695. 
14. Evans, J.A., W.A. Whitelaw (2009). "The assessment of maximal respiratory mouth 
pressures in adults." Respir Care 54(10):1348-59. 
15. Fairclough, R. J., M. J. Wood and K. E. Davies (2013). "Therapy for Duchenne muscular 
dystrophy: renewed optimism from genetic approaches." Nat Rev Genet 14(6): 373-378. 
16. Falzarano, M. S., C. Scotton, C. Passarelli and A. Ferlini (2015). "Duchenne Muscular 
Dystrophy: From Diagnosis to Therapy." Molecules 20(10): 18168-18184. 
17. Gauld, L. M., J. Kappers, J.B. Carlin, C.F. Robertson (2004). "Height prediction from 
ulna length." Dev Med Child Neurol 46(7):475-80. 
18. Hendriksen, R. G., G. Hoogland, S. Schipper, J. G. Hendriksen, J. S. Vles and M. W. 
Aalbers (2015). "A possible role of dystrophin in neuronal excitability: a review of the 
current literature." Neurosci Biobehav Rev 51: 255-262. 
19. Henricson, E. K., R. T. Abresch, A. Cnaan, F. Hu, T. Duong, A. Arrieta, J. Han, D. M. 
Escolar, J. M. Florence, P. R. Clemens, E. P. Hoffman, C. M. McDonald and C. 
Investigators (2013). "The cooperative international neuromuscular research group 
Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful 
functional milestones and reduces rate of disease progression as measured by manual 
muscle testing and other commonly used clinical trial outcome measures." Muscle Nerve 
48(1): 55-67. 
20. Hoffman, E. P., R. H. Brown and L. M. Krunkel (1987). "Dystrophin: the protein product 
of the Duchenne muscular dystrophy locus." Cell 51(6): 919-928. 
21. Hong, S. J., D. Hou, T. J. Brinton, B. Johnstone, D. Feng, P. Rogers, W. F. Fearon, P. 
Yock and K. L. March (2014). "Intracoronary and retrograde coronary venous 
myocardial delivery of adipose-derived stem cells in swine infarction lead to transient 
myocardial trapping with predominant pulmonary redistribution." Catheter Cardiovasc 
Interv 83(1): E17-25. 
22. Ibrahim, A. G., K. Cheng and E. Marban (2014). "Exosomes as critical agents of cardiac 
regeneration triggered by cell therapy." Stem Cell Reports 2(5): 606-619. 
23. Kanazawa, H., E. Tseliou, K. Malliaras, K. Yee, J. F. Dawkins, G. De Couto, R. R. 
Smith, M. Kreke, J. Seinfeld, R. C. Middleton, R. Gallet, K. Cheng, D. Luthringer, I. 
Valle, S. Chowdhury, K. Fukuda, R. R. Makkar, L. Marban and E. Marban (2015). 
"Cellular postconditioning: allogeneic cardiosphere-derived cells reduce infarct size and 
attenuate microvascular obstruction when administered after reperfusion in pigs with 
acute myocardial infarction." Circ Heart Fail 8(2): 322-332. 
24. Koenig, M., A. P. Monaco and L. M. Kunkel (1988). "The complete sequence of 
dystrophin predicts a rod-shaped cytoskeletal protein." Cell 53(2): 219-228. 

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25. Mah J.K., L. Korngut, J. Dykeman, L. Day, T. Pringsheim, N. Jette. (2014). "A 
systematic review and meta-analysis on the epidemiology of Duchenne and Becker 
muscular dystrophy." Neuromuscul Disord 24(6):482-91. 
26. Malliaras, K., R. R. Smith, H. Kanazawa, K. Yee, J. Seinfeld, E. Tseliou, J. F. Dawkins, 
M. Kreke, K. Cheng, D. Luthringer, C. S. Ho, A. Blusztajn, I. Valle, S. Chowdhury, R. R. 
Makkar, R. Dharmakumar, D. Li, L. Marban and E. Marban (2013). "Validation of 
contrast-enhanced magnetic resonance imaging to monitor regenerative efficacy after cell 
therapy in a porcine model of convalescent myocardial infarction." Circulation 128(25): 
2764-2775. 
27. Malliaras, K., T. S. Li, D. Luthringer, J. Terrovitis, K. Cheng, T. Chakravarty, G. Galang, 
Y. Zhang, F. Schoenhoff, J. Van Eyk, L. Marban and E. Marban (2012). "Safety and 
efficacy of allogeneic cell therapy in infarcted rats transplanted with mismatched 
cardiosphere-derived cells." Circulation 125(1): 100-112. 
28. Muntoni, F., S. Torelli and A. Ferlini (2003). "Dystrophin and mutations: one gene, 
several proteins, multiple phenotypes." Lancet Neurol 2(12): 731-740. 
29. Pane M., E.S. Mazzone, L. Fanelli, R. De Sanctis, F. Bianco, S. Sivo, A. D'Amico, S. 
Messina, R. Battini, M. Scutifero, R. Petillo, S. Frosini, R. Scalise, G. Vita, C. Bruno, M. 
Pedemonte, T. Mongini, E. Pegoraro, F. Brustia, A. Gardani, A. Berardinelli, V. 
Lanzillotta, E. Viggiano, F. Cavallaro, M. Sframeli, L. Bello, A. Barp, S. Bonfiglio, E. 
Rolle, G. Colia, M. Catteruccia, C. Palermo, G. D'Angelo, A. Pini, E. Iotti, K. Gorni, G. 
Baranello, L. Morandi, E. Bertini, L. Politano, M. Sormani, E. Mercuri (2014). 
"Reliability of the Performance of Upper Limb assessment in Duchenne muscular 
dystrophy." Neuromuscul Disord 24(3):201-6.  
30. Reich, H., E. Tseliou, G. de Couto, D. Angert, J. Valle, Y. Kubota, D. Luthringer, J. 
Mirocha, B. Sun, R. R. Smith, L. Marban and E. Marban (2016). "Repeated 
transplantation of allogeneic cardiosphere-derived cells boosts therapeutic benefits 
without immune sensitization in a rat model of myocardial infarction." J Heart Lung 
Transplant 35(11): 1348-1357. 
31. Weiss, D. J., R. Casaburi, R. Flannery, M. LeRoux-Williams and D. P. Tashkin (2013). 
"A placebo-controlled, randomized trial of mesenchymal stem cells in COPD." Chest 
143(6): 1590-1598. 
32. White S.J., J.T. den Dunnen (2006). "Copy number variation in the genome; the human 
DMD gene as an example." Cytogenet Genome Res 115(3-4):240-6. 
33. Wilson, J. G., K. D. Liu, H. Zhuo, L. Caballero, M. McMillan, X. Fang, K. Cosgrove, R. 
Vojnik, C. S. Calfee, J. W. Lee, A. J. Rogers, J. Levitt, J. Wiener-Kronish, E. K. Bajwa, 
A. Leavitt, D. McKenna, B. T. Thompson and M. A. Matthay (2015). "Mesenchymal 
stem (stromal) cells for treatment of ARDS: a phase 1 clinical trial." Lancet Respir Med 
3(1): 24-32.