A Phase 3 interventional study of Deramiocel (CAP-1002) and Placebo in Muscular Dystrophies, Muscular Dystrophy, Duchenne and Muscular Disorders, Atrophic, sponsored by Capricor Inc.. Active, not recruiting at 20 sites in United States. Open to male participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2026-08-28.
Sponsored by Capricor Inc. · Phase 3, Interventional, and Treatment
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.
Up to 102 eligible study subjects will participate in this two cohort study. Cohort A will enroll approximately 58 subjects randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort A will receive CAP-1002A (deramiocel manufactured at Capricor's manufacturing facility in Los Angeles, CA). Once Cohort A enrollment is completed, Cohort B enrollment will begin. Cohort B will enroll approximately 44 participants randomized in a 1:1 ratio (active:placebo). Subjects randomized to the active treatment group in Cohort B will receive CAP-1002B (deramiocel manufactured at Capricor's manufacturing facility in San Diego, CA). Both cohorts will include visits at Screening, Baseline/Day 1, Month 1, and Months 3, 6, 9, and 12. Subjects will receive IV infusions of deramiocel or placebo on Day 1 and Months 3, 6, and 9. All subjects will then be eligible to receive additional IV infusions of deramiocel at Month 12, 15, 18, and 21 as part of the open-label phase of the study. All subjects who complete the first open-label extension phase of the study will be eligible to receive additional IV infusions of deramicoel every 3 months in a Long Term Open-Label Extension phase until commercial availability of deramiocel, or until sponsor's decision to terminate the trial, or the participant withdraws consent.
A primary analysis of efficacy and safety will be performed on the double-blind placebo-controlled phase of the study for both Cohort A and Cohort B combined at Month 12 following 4 administrations of deramiocel or placebo.
The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by Performance of the Upper Limb test, version 2.0 [PUL 2.0] Total Score at the 12-month time point. Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging [cMRI], changes in hand-to-mouth function [eat 10-bites assessed by the Duchenne Video Assessment (DVA)], quality of life assessments, and biomarker analysis for creatine kinase MB isoenzyme (CK-MB).
Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, and clinical laboratory testing.
An analysis of extended safety and efficacy will be performed in the subsequent open-label phases of the study.
548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.
This study's enrollment of 106 is above the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.
Browse Muscular Dystrophies studies →Capricor Inc. is the lead sponsor of 11 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Cohort A: Approximatetly 29 subjects will receive deramiocel (CAP-1002A) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive deramiocel (CAP-1002B) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months Open-Label Extension: After completion of the double-blind, placebo-controlled phase, subjects will receive deramiocel (CAP-1002) active treatment consisting of 150 million cardiosphere-derived cells (CDCs) via intravenous infusion every 3 months for 12 months followed by the long-term open label extension phase which continues deramiocel administration every 3 months until sponsor decision.
Biological: Deramiocel (CAP-1002)
Cohort A: Approximately 29 subjects will receive a Placebo solution via intravenous infusion every 3 months Cohort B: Approximately 22 participants will receive a Placebo solution via intravenous infusion every 3 months
Biological: Placebo
Cohort A: CAP-1002A manufactured in Los Angeles, CA; Cohort B: CAP-1002B manufactured in San Diego, CA; Open-Label Extension: CAP-1002A or CAP-1002B
Also known as: Cardiosphere-Derived Cells (CDCs)
Placebo
Change in the upper limb function
Mean percent change from baseline in Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.
Time frame: At Month 12
Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction
Mean change from baseline in left ventricular ejection fraction as assessed by Cardiac Magnetic Resonance (cMRI) and assessed centrally
Time frame: At Month 12
Change in mid-level (elbow) upper limb function
Mean percent change from baseline in mid-level \[elbow\] upper limb function in Performance of the Upper Limb test, version 2 (PUL 2.0); items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.
Time frame: At Month 12
Change in Global Statistical Test (Total GST) combining upper limb function, cardiac muscle function, and patient reported measure of disease severity
Mean change from baseline in Total GST that combines PUL 2.0 score, LVEF assessment by cMRI, and the Patient Global Impression of Severity (PGI-S)
Time frame: At Month 12
Change in the number of segments of myocardial scarring (Cohort B only)
Change in the number of segments of myocardial scarring as observed by late gadolinium enhancement (LGE)
Time frame: at Month 12
Change in cardiac muscle function and structure by assessment of left ventricular end-systolic volume
Mean change from baseline in left ventricular indexed end-systolic volume as assessed by Cardiac Magnetic Resonance (cMRI)
Time frame: At Month 12
Change in cardiac muscle function and structure by assessment of left ventricular ejection fraction in subjects with documented cardiomyopathy
Mean change from baseline in left ventricular ejection fraction as assessed by Cardiac Magnetic Resonance (cMRI) in subjects with documented cardiomyopathy at baseline.
Time frame: At Month 12
Change in a global statistical test (Cardiac GST) combining assessments of cardiac muscle function and structure
Mean change from baseline in Cardiac GST that combines LVEF, LV end-systolic volume index, and LV end-diastolic volume index
Time frame: At Month 12
Change in percentage of myocardial scarring (Cohort B only)
Change in percentage of myocardial scarring as observed by late gadolinium enhancement (LGE)
Time frame: at Month 12
Change in hand-to-mouth function in the context of functional eating
Mean change from baseline in the functional ability of eating 10 bites evaluated by Duchenne Video Assessment (DVA) using a video record of patient performing the task at home. Physical therapists score the patient's quality of movement in the video using scorecards with pre-specified compensatory movement criteria.
Time frame: At Month 12
Changes in cardiac inflammation biomarker, creatine kinase MB isoenzyme [CK-MB]
Mean change from baseline in CK-MB blood levels - MB fraction (% of total CK).
Time frame: At Month 12
Plan to share: No
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Capricor Inc.