A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2020-03-10
• Target enrollment: 0
• Participant gender: Male

Summary

HOPE-2 is a double-blind 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 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Capricor Inc.

Criteria

Inclusion Criteria:

1. Genetically confirmed DMD

2. Reduced upper arm strength as measured by the Performance of Upper Limb

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 < 35%

2. BMI > 45

3. Ambulant if ≥ 18 years of age

4. Exon 44 skip-amenable mutation(s) in the DMD gene

5. Deletion mutation(s) encompassing exons 3-7 of the DMD gene

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

7. Chronic respiratory disease not related to DMD (for example, asthma, bronchitis, and tuberculosis)

8. History of diabetes requiring treatment with metformin or insulin within 3 months prior to randomization

9. Treatment with an FDA-approved exon skipping therapy for the treatment of DMD if on a stable dose for less than 24 months prior to randomization

10. Treatment with human growth hormone (HGH) within 3 months prior to randomization, unless on a stable dose for at least 24 months prior to randomization

11. Treatment with idebenone within 3 months prior to randomization

12. Treatment with a cell therapy product within 12 months prior to randomization

13. Treatment with an investigational product within 6 months prior to randomization