A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)

• Status: Not yet recruiting
• Trial end date: 2024-06-30
• Target enrollment: 0
• Participant gender: Male

Summary

HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Capricor Inc.

Criteria

Inclusion Criteria:

1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial and diagnosed with DMD as confirmed by the Investigator

2. Genetically confirmed DMD

3. Reduced ability to walk/run (if ambulatory)

4. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for weight-based or toxicity-related adjustments

5. Current and up-to-date immunizations

Exclusion Criteria:

1. Left ventricular ejection fraction (LVEF) < 35%

2. Elbow-flexion contractures > 30° in both extremities

3. Body mass index (BMI) > 45

4. Percent predicted forced vital capacity (FVC%) < 35%