HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• 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 2016
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02485938
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Deborah Ascheim, MD
• Affiliation: Capricor Inc.

• Name: John L Jefferies, MD, MPH
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male subjects 18 years of age or older must be able to provide informed consent and
follow up with protocol procedures. Male subjects 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 subjects will be randomized into this
study.

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

3. Cardiomyopathy with left ventricular scar by 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. Subjects must be taking systemic glucocorticoids for at least six months prior to
screening.

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

7. Subjects 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 ICF / 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 (ALT/AST >10 times the upper reference range) and/or abnormal
hematology (hematocrit <25%, WBC <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)

Safety and tolerability composite of CAP-1002 will be established as described below. [Time Frame: 72 hours post infusion of allogeneic cardiosphere-derived cells]

Secondary Outcome(s)

Quality of Life composite assessed as: Change in PedsQL (Pediatric Quality of Life Inventory), including the cardiac module, and PODCI Adolescent Questionnaire. [Time Frame: 12 months post infusion of CAP-1002 or randomization to the usual care arm]

Biomarkers composite assessed as: Osteopontin, ST2, IL-10, Galectin-3, and exploratory biomarkers (if consented/provided assent). [Time Frame: 12 months post infusion of CAP-1002 or randomization to the usual care arm]

Functional composite assessed as: Serial change in mobility measurements and Performance of Upper Limb (PUL) scale, spirometry, and 6-minute walk test (6MWT) when deemed appropriate by the Investigator. [Time Frame: 12 months post infusion of CAP-1002 or randomization to the usual care arm]

Cardiac Structural composite assessed as: Absolute and relative change in parameters measured by cardiac MRI [Time Frame: 12 months post infusion of CAP-1002 or randomization to the usual care arm]

Secondary ID(s)

CAP-1002-DMD-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors