HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

• Register: ClinicalTrials.gov
• Last refreshed on: 27 January 2025
• Main ID: NCT02485938
• Date of registration: 19/06/2015
• Prospective Registration: Yes
• Primary sponsor: Capricor Inc.
• Public title: HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) HOPE
• Scientific title: A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy
• Date of first enrolment: January 7, 2016
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02485938
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Mark Awadalla
• Affiliation: Capricor Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. 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.

2. Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.

3. 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.

4. 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.

5. Participants must be taking systemic glucocorticoids for at least six months prior
to screening.

6. Participants must be 12 years of age or older at time of screening

7. 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:

1. Therapy with intravenous inotropic or vasoactive medications at the time of
screening.

2. Inability to undergo cardiac catheterization and/or MRI without general anesthesia.

3. Immunologic incompatibility with all available Master Cell Banks (MCBs) by
single-antigen bead (SAB) serum antibody profiling.

4. Planned or likely major surgery in the next 12 months after planned randomization.

5. Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of
acquiring a LVAD.

6. Contraindication to cardiac MRI.

7. Known hypersensitivity to contrast agents.

8. Estimated glomerular filtration rate (GFR) <60 mL/min, as calculated by the CKD-EPI
cystatin C equation (Inker, Schmid et al. 2012).

9. Active infection not responsive to treatment.

10. Active systemic allergic reaction(s), connective tissue disease or autoimmune
disorder(s).

11. History of cardiac tumor or cardiac tumor demonstrated on screening MRI.

12. History of previous stem cell therapy.

13. History of use of medications listed in Appendix 3 within 3 months prior to signing
the Inform Consent Form / Assent through completion of the study infusion.

14. Known moderate-to-severe aortic stenosis/insufficiency or severe mitral
stenosis/regurgitation.

15. Current active alcohol or drug abuse.

16. Known history of Human Immunodeficiency Virus (HIV) infection.

17. Known history of chronic viral hepatitis.

18. Abnormal liver function (alanine aminotransferase (ALT)/aspartate aminotransferase
(AST) >10 times the upper reference range) and/or abnormal hematology (hematocrit
<25%, White Blood Cells <3000 µl, platelets <100,000 µl) studies without a
reversible, identifiable cause.

19. Known hypersensitivity to bovine products.

20. Known hypersensitivity to dimethyl sulfoxide (DMSO).

21. Uncontrolled diabetes (HbA1c >9.0).

22. Inability to comply with protocol-related procedures, including required study
visits.

23. Any condition or other reason that, in the opinion of the Investigator or Medical
Monitor, would render the subject unsuitable for the study.

24. 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


Age minimum: 12 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Cardiomyopathy

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)

Primary Outcome(s)

Change From Baseline in Clinical Laboratory Parameters (Chloride, Potassium and Sodium) at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Vital Signs - Respiratory Rate at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Clinical Laboratory Parameter - Glucose at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Number of Participants Experiencing Any of the Adjudicated Events [Time Frame: Within 72 hours post-infusion]

Change From Baseline in Hematological Parameters (Platelets, White Blood Cells, Basophils, Eosinophils, Lymphocytes, Monocytes and Neutrophils) at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: Up to Month 12 post-infusion]

Number of Participants With Clinically Significant Change From Baseline in Cardiac Physical Examinations at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Clinical Laboratory Parameter - Albumin at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Hematological Parameter - Red Blood Cells at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Electrocardiogram Parameter - Ventricular Rate at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Hematological Parameter - Hemoglobin at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Vital Signs - Heart Rate at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Electrocardiogram (ECG) Parameters (QRS Duration, PR, QT, QTc and QT Interval) at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Vital Signs - Blood Pressure at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Change From Baseline in Vital Signs - Temperature at Month 6 and Month 12 [Time Frame: Baseline, Month 6 and Month 12]

Secondary ID(s)

CAP-1002-DMD-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 09/01/2025
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02485938