CompletedPhase 2

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

United States25 participantsStarted 2016-01-07

Plain-language 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.

Who can participate

Age range12 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Participants Experiencing Any of the Adjudicated Events

Timeframe: Within 72 hours post-infusion

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: Up to Month 12 post-infusion

Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12

Timeframe: 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

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12

Trial details

NCT IDNCT02485938

SponsorCapricor Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-09-14

Results submitted2024-02-29

View on ClinicalTrials.gov More Duchenne Muscular Dystrophy trials

Plain-language summary

Who can participate

Age range12 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Participants Experiencing Any of the Adjudicated Events

Timeframe: Within 72 hours post-infusion

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: Up to Month 12 post-infusion

Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12

Timeframe: 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

Timeframe: Baseline, Month 6 and Month 12

Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12

Timeframe: Baseline, Month 6 and Month 12