HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) (HOPE)

A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy; Cardiomyopathy
• Intervention / Treatment: Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)

Detailed Description

Approximately 24, and not more than 30, participants will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2.

The first 6-8 randomized participants will comprise Group 1, and will include a minimum of 3 participants completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused participants and for at least 6 participants overall.

Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 participants. Screening and randomization will continue until at total of 12 participants are infused with CAP 1002 or 30 participants are randomized into the study, whichever comes first.

All participants assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior).

Participants randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the participant on an ongoing basis, and will receive no infusion.

Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible participants randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible participants randomized to the usual care arm will occur 7 days after the date of randomization. All randomized participants will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90048
      • Cedars-Sinai Medical Center
  • Florida
    • Gainesville, Florida, United States, 32611
      • University of Florida
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male participants 18 years of age or older must be able to provide informed consent and follow up with protocol procedures. Male participants at least 12 years of age but younger than 18 years of age must be able to provide assent with parent or guardian providing permission for study participation. Only male participants will be randomized into this study.
2. Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.
3. Cardiomyopathy with left ventricular scar by late gadolinium enhancement (LGE) in at least 4 segments as assessed by contrast-enhanced MRI and EF >35% at the time of screening.
4. Use of evidence based medical-therapy in accordance with the "DMD Care Considerations Working Group" guidelines for the management of DMD, for at least three months prior to signing the consent form (or, providing assent) or documented contraindication or intolerance or patient preference.
5. Participants must be taking systemic glucocorticoids for at least six months prior to screening.
6. Participants must be 12 years of age or older at time of screening
7. Participants must be appropriate candidates for cardiac catheterization and intracoronary infusion of CAP-1002, in the judgement of the site's interventional cardiologist.

Exclusion Criteria:

1. Therapy with intravenous inotropic or vasoactive medications at the time of screening.
2. Inability to undergo cardiac catheterization and/or MRI without general anesthesia.
3. Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling.
4. Planned or likely major surgery in the next 12 months after planned randomization.
5. Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD.
6. Contraindication to cardiac MRI.
7. Known hypersensitivity to contrast agents.
8. Estimated glomerular filtration rate (GFR) <60 mL/min, as calculated by the CKD-EPI cystatin C equation (Inker, Schmid et al. 2012).
9. Active infection not responsive to treatment.
10. Active systemic allergic reaction(s), connective tissue disease or autoimmune disorder(s).
11. History of cardiac tumor or cardiac tumor demonstrated on screening MRI.
12. History of previous stem cell therapy.
13. History of use of medications listed in Appendix 3 within 3 months prior to signing the Inform Consent Form / Assent through completion of the study infusion.
14. Known moderate-to-severe aortic stenosis/insufficiency or severe mitral stenosis/regurgitation.
15. Current active alcohol or drug abuse.
16. Known history of Human Immunodeficiency Virus (HIV) infection.
17. Known history of chronic viral hepatitis.
18. Abnormal liver function (alanine aminotransferase (ALT)/aspartate aminotransferase (AST) >10 times the upper reference range) and/or abnormal hematology (hematocrit <25%, White Blood Cells <3000 μl, platelets <100,000 μl) studies without a reversible, identifiable cause.
19. Known hypersensitivity to bovine products.
20. Known hypersensitivity to dimethyl sulfoxide (DMSO).
21. Uncontrolled diabetes (HbA1c >9.0).
22. Inability to comply with protocol-related procedures, including required study visits.
23. Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study.

24. Currently receiving investigational treatment on another clinical study or expanded access protocol, including any of the following:

    • Received investigational intervention within 30 days prior to randomization
    • Treatment and/or an incomplete follow-up to treatment with any investigational cell based therapy within 6 months prior to randomization
    • Active participation in other research therapy for cardiovascular repair/regeneration

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Allogeneic Cardiosphere-Derived Cells (CAP-1002); CAP-1002 is an investigational product consisting of allogeneic cardiosphere-derived cells (CDCs). All subjects assigned to the active treatment arm will receive an intended total dose of 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior). If any of the three coronary arteries are deemed by the infusing Investigator to supply less than 30% of the left ventricular myocardium, the infusing Investigator may choose to infuse only 12.5M cells into that coronary artery or arteries. Therefore the full dose of CAP-1002 delivered may range from 50M cells to 75M cells provided that all three arteries are infused.	Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002); Intracoronary delivery of Allogeneic Cardiosphere-Derived Cells (CAP-1002); Other Names: CAP-1002
No Intervention: Usual Care; Subjects randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the subject on an ongoing basis, and will receive no infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Experiencing Any of the Adjudicated Events	Adjudicated Events reported included: New thrombolysis in myocardial infarction (TIMI) flow 0-2 or TIMI myocardial perfusion grade (TMPG) 0-2noted immediately following infusion and persisting greater than (>) 3 minutes, despite intracoronary vasodilator administration; sudden unexpected death within 72 hours of intracoronary infusion; and Major adverse cardiac event (MACE) within 72 hours of intracoronary infusion, including death, non-fatal myocardial infarction and hospitalization for cardiovascular event (including heart failure hospitalizations).	Within 72 hours post-infusion
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	An Adverse Event (AE) was any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily had to have causal relationship with treatment. TEAEs were defined as AEs that occurred or worsened in severity between the first dose of the investigational medicinal product (IMP) until the end of study. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.	Up to Month 12 post-infusion
Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12	Clinical chemistry parameters assessed were chloride, potassium and sodium.	Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12	Clinical chemistry parameter assessed was albumin.	Baseline, Month 6 and Month 12
Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12	Clinical chemistry parameter assessed was glucose.	Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12	Hematological parameters assessed were: platelets, white blood cells, basophils, eosinophils, lymphocytes, monocytes and neutrophils.	Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12	Hematological parameter assessed was hemoglobin.	Baseline, Month 6 and Month 12
Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12	Hematological parameter assessed was red blood cells.	Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12	Vital signs assessed were systolic and diastolic blood pressure.	Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12	Vital signs assessed was heart rate.	Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12	Vital signs assessed was respiratory rate.	Baseline, Month 6 and Month 12
Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12	Vital signs assessed was temperature.	Baseline, Month 6 and Month 12
Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12	Cardiac physical examination parameters assessed were: jugular vein distension, edema, heart sounds, murmur, breath sounds.	Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12	ECG parameters assessed were: PR Interval, QRS Duration, QT Interval, QTc interval and QT interval.	Baseline, Month 6 and Month 12
Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12	ECG parameter assessed was ventricular rate; which depends on the degree of atrioventricular conduction.	Baseline, Month 6 and Month 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Cardiac Structural composite assessed as: Absolute and relative change in parameters measured by cardiac MRI	12 months post infusion of CAP-1002 or randomization to the usual care arm
Functional composite assessed as: Serial change in mobility measurements and Performance of Upper Limb (PUL) scale, spirometry, and 6-minute walk test (6MWT) when deemed appropriate by the Investigator.	12 months post infusion of CAP-1002 or randomization to the usual care arm
Quality of Life composite assessed as: Change in PedsQL (Pediatric Quality of Life Inventory), including the cardiac module, and PODCI Adolescent Questionnaire.	12 months post infusion of CAP-1002 or randomization to the usual care arm
Biomarkers composite assessed as: Osteopontin, ST2, IL-10, Galectin-3, and exploratory biomarkers (if consented/provided assent).	12 months post infusion of CAP-1002 or randomization to the usual care arm

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Absolute Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12	LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). LVEF expressed as percentage ejection fraction was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.	Baseline, Month 6 and Month 12
Percent Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 6 and 12	LVEF is the fraction of chamber volume ejected in systole (stroke volume) in relation to the volume of the blood in the ventricle at the end of diastole (end-diastolic volume). Percent change in LVEF was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100%.	Baseline, Month 6 and Month 12
Absolute Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12	LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. LVEDV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.	Baseline, Month 6 and Month 12
Percent Change From Baseline in Left Ventricular End Diastolic Volume (LVEDV) at Month 6 and 12	LVEDV is the amount of blood in the heart's left ventricle just before the heart contracts. Percent change in LVEDV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100%.	Baseline, Month 6 and Month 12
Absolute Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12	LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. LVESV was assessed by magnetic resonance imaging. Absolute change was calculated as: post-baseline value-Baseline value.	Baseline, Month 6 and Month 12
Percent Change From Baseline in Left Ventricular End Systolic Volume (LVESV) at Month 6 and 12	LVESV is the amount of blood remaining in the ventricle at the end of systole, after the heart has contracted. Percent change in LVESV was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100%.	Baseline, Month 6 and Month 12
Absolute Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12	Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. LV LGE scar tissue mass in grams was assessed by cardiac MRI. Absolute change was calculated as: post-baseline value-Baseline value.	Baseline, Month 6 and Month 12
Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) Scar Tissue Mass at Month 6 and 12	Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Percent change in LV LGE scar tissue mass was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100.	Baseline, Month 6 and Month 12
Absolute Change From Baseline in Left Ventricular (LV) Late Gadolinium Enhancement (LGE) at Month 6 and 12	Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Improvement in infarct size as a percent of LV mass was assessed by magnetic resonance imaging and reported in this outcome measure.	Baseline, Month 6 and 12
Percent Change From Baseline in LV Late Gadolinium Enhancement (LGE) at Month 6 and 12	Late gadolinium enhancement is a technique used in cardiac MRI for cardiac tissue characterization, in particular, the assessment of myocardial scar formation and regional myocardial fibrosis. Percent improvement in infarct size defined by scar as a percent of LV mass was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100%.	Baseline, Month 6 and 12
Absolute Change From Baseline in Circumferential Strain at Month 6 and 12	Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis. Improvement in Circumferential Strain was expressed in percentage and was assessed by cardiac MRI.	Baseline, Month 6 and Month 12
Percent Change From Baseline in Circumferential Strain at Month 6 and 12	Circumferential strain represents the change in length (shortening in systole, represented as a negative strain value) of the myocardium along the circumferential axis of the LV as viewed in the short axis; and was assessed by cardiac MRI. Percent change from Baseline was calculated as: Percent change = (post-baseline value-Baseline value)/Baseline value *100%.	Baseline, Month 6 and Month 12
Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12	The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as change from baseline was assessed by magnetic resonance imaging. Change was calculated as: post-baseline value - baseline value.	Baseline, Month 6 and Month 12
Percent Change From Baseline in Function of the Region Receiving CAP-1002 Therapy at Month 6 and 12	The regions assessed of the heart were: Anterior, Lateral, Inferior and Septal. Tissue mass recovery in the function of region receiving therapy expressed as percent change from baseline was assessed by magnetic resonance imaging. Percent change from Baseline was calculated as: Percent change = (post-baseline value - Baseline value)/Baseline value *100%.	Baseline, Month 6 and Month 12
Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Excluding Shoulder) at Month 6 and 12	The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (excluding the shoulder dimension) and ranged from 0 to 58. Higher scores indicated improvement and lower scores indicated severity. Change from Baseline in PUL overall score (excluding shoulder) at Month 6 and 12 is reported in this outcome measure.	Baseline, Month 6 and 12
Change From Baseline in Performance of the Upper Limb (PUL) Overall Score (Including Shoulder) at Month 6 and 12	The PUL Scale consists of an entry item to define the starting functional level and 21 items subdivided into shoulder level (4 items,), middle level (9 items), and distal level (8 items) dimensions. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for middle level, and 24 for distal level. The total score is calculated by the sum of all the scores of the three subscales (including the shoulder dimension) and ranged from 0 to 74. Higher scores over time indicate improvement and lower scores indicated severity. Change from Baseline in PUL overall score (including shoulder) at Month 6 and 12 is reported in this outcome measure.	Baseline, Month 6 and 12
Change From Baseline in Peak Expiratory Flow (PEF) at Month 6 and 12	The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter.	Baseline, Month 6 and 12
Change From Baseline in Percent Predicted Peak Expiratory Flow at Month 6 and 12	Percent predicted PEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function.	Baseline, Month 6 and 12
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 6 and 12	FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.	Baseline, Month 6 and 12
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second at Month 6 and 12	FEV1 was the volume of air exhaled in the first second of a forced expiration as measured by a spirometer.	Baseline, Month 6 and 12
Change From Baseline in Percent Predicted Forced Vital Capacity Level at Month 6 and 12	FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.	Baseline, Month 6 and 12
Change From Baseline in Forced Vital Capacity (FVC) Levels at Month 6 and 12	FVC was the total amount of air exhaled from the lungs during the lung function test measured by spirometer.	Baseline, Month 6 and 12
Change From Baseline in Forced Expiratory Flow at 25-75% of FVC (FEF25-75%) at Month 6 and 12	Forced Expiratory Flow (FEF) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test).	Baseline, Month 6 and 12
Change From Baseline in Percent Predicted Forced Expiratory Flow at 25-75% of FVC at Month 6 and 12	FEF is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF25-75% is defined as the mean forced expiratory flow between the 25% and 75% of the FVC (total amount of air exhaled from the lungs during the lung function test).	Baseline, Month 6 and 12
Change From Baseline in Forced Expiratory Time (FET) at Month 6 and 12	FET is defined as the time taken for an individual to complete a forceful exhalation after maximal inspiration. FET is measured by asking the subject to take a deep breath and blow it all out as fast as possible.	Baseline, Month 6 and 12
Change From Baseline in Six-Minute Walk Test at Month 6 and 12	The six-minute walk test measures the distance a participant is able to walk over a total of six minutes on a hard, flat surface. The goal is for the participant to walk as far as possible in six minutes. The participant is allowed to self-pace and rest as needed as they traverse back and forth along a marked walkway. The total distance walked, in meters, was recorded for each participant. Longer distances indicate better outcomes.	Baseline, Month 6 and 12
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Total Summary Score - Participant Responses at Month 6 and 12	The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- participant responses at Month 6 and 12 was reported in this outcome measure.	Baseline, Month 6 and 12
Change From Baseline in Pediatric Quality of Life Inventory Total Summary Score - Parent Responses at Month 6 and 12	The PedsQL-Duchenne muscular dystrophy (DMD) module consists of 18 items and comprised of 4 domains: Daily Activities, Treatment Barriers, Worry, Communication. Items are reverse scored and linearly transformed to a 0-100 scale. The overall range for total PedsQL score (18 items; mean of all items) was 0 (improvement) to 100 (severity). Decreasing scores over time indicate improvement. Change From Baseline in PedsQL total summary score- parent responses at Month 6 and 12 was reported in this outcome measure.	Baseline, Month 6 and 12
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Parent Responses at Month 6 and 12	The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of "0" represents a poor outcome/worse health, while "100" is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning).	Baseline, Month 6 and 12
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Score - Participant Responses at Month 6 and 12	The PODCI consists of 83 items and 5 core scales: Upper Extremity and Physical Function, Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort, and Happiness; a Global Functioning Scale. The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale). The Global Functioning Scale and each of the core scales were standardized so that a score of "0" represents a poor outcome/worse health, while "100" is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning).	Baseline, Month 6 and 12
Change From Baseline in Duchenne Muscular Dystrophy Biomarkers at Month 6 and 12	Osteopontin, Cardiac stress biomarker (ST2) and Galectin-3 were DMD biomarkers assessed through serum samples.	Baseline, Month 6 and Month 12

Collaborators and Investigators

• Sponsor: Capricor Inc.
• Investigators: Study Director: Mark Awadalla, Capricor Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 7, 2016
• Primary Completion (Actual): September 14, 2017
• Study Completion (Actual): September 14, 2017

Study Registration Dates

• First Submitted: June 19, 2015
• First Submitted That Met QC Criteria: June 26, 2015
• First Posted (Estimated): June 30, 2015

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 6, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Duchenne Muscular Dystrophy; Cardiomyopathy
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Cardiovascular Diseases; Muscular Diseases; Heart Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Cardiomyopathies; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: CAP-1002-DMD-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No