Phase 2
N=25
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Duchenne Muscular Dystrophy · Cardiomyopathy
Bottom Line
View on ClinicalTrials.gov: NCT02485938 ↗Enrolled (actual)
25
Serious AEs
16.0%
Results posted
Jan 2025
Primary outcome: Primary: Number of Participants Experiencing Any of the Adjudicated Events — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Cardiosphere-Derived Cells (CAP-1002) (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Capricor Inc.
- Primary completion
- Sep 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Any of the Adjudicated Events |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
10; 12; 1; 3 | — |
| PRIMARY Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 |
100.9; 102.7; -0.4; -0.8; 0.2; -0.9 | — |
| PRIMARY Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 |
4.48; 3.87; -0.09; 0.36; -0.02; 0.32 | — |
| PRIMARY Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 |
87.1; 89.5; 2.1; 12.3; -4.1; 6.8 | — |
| PRIMARY Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 |
357.9; 318.6; -24.5; 40.4; -26.0; 24.0 | — |
| PRIMARY Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 |
14.48; 13.76; -0.05; 0.05; -0.13; 0.29 | — |
| PRIMARY Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 |
5.01; 4.68; -0.05; 0.20; -0.07; 0.23 | — |
| PRIMARY Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 |
111.3; 113.7; 2.3; -2.1; -0.3; 2.8 | — |
| PRIMARY Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 |
90.8; 87.1; -3.0; 4.8; -0.2; 4.4 | — |
| PRIMARY Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 |
17.3; 16.3; -0.5; 0.3; 0.0; 0.8 | — |
| PRIMARY Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 |
36.79; 36.68; -0.13; 0.18; -0.09; 0.05 | — |
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 |
0; 0; 0; 0; 1; 1 | — |
| PRIMARY Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 |
116.2; 129.2; 8.7; -2.8; 8.3; 1.0 | — |
| PRIMARY Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 |
87.8; 89.8; 0.8; 1.2; -1.8; 2.2 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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.
- Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.
- 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.
- 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.
- Participants must be taking systemic glucocorticoids for at least six months prior to screening.
- Participants must be 12 years of age or older at time of screening
- 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
- Therapy with intravenous inotropic or vasoactive medications at the time of screening.
- Inability to undergo cardiac catheterization and/or MRI without general anesthesia.
- Immunologic incompatibility with all available Master Cell Banks (MCBs) by single-antigen bead (SAB) serum antibody profiling.
- Planned or likely major surgery in the next 12 months after planned randomization.
- Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of acquiring a LVAD.
- Contraindication to cardiac MRI.
- Known hypersensitivity to contrast agents.
- Estimated glomerular filtration rate (GFR) 10 times the upper reference range) and/or abnormal hematology (hematocrit 9.0).
- Inability to comply with protocol-related procedures, including required study visits.
- Any condition or other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for the study.
- 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
Data sourced from ClinicalTrials.gov (NCT02485938). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.