CAP-1002 for Duchenne Muscular Dystrophy
(HOPE-2-OLE Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Overseen byCraig McDonald, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Capricor Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of twenty intravenous administrations of deramiocel, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of deramiocel. Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, and 60. Safety and efficacy assessments will be conducted prior to deramiocel administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57 trial visits, unless otherwise indicated. All deramiocel infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, and 57. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.

Research Team

Craig McDonald, MD

Principal Investigator

UC Davis

Mark Awadalla

Principal Investigator

Capricor Inc.

Eligibility Criteria

This trial is for those who were in the HOPE-2 study, finished a year of follow-up, and can give consent. They must have good veins for infusions and be able to stick to the trial plan. People with planned major surgery, recent investigational drug use, substance abuse issues, or severe respiratory problems can't join.

Inclusion Criteria

Documented enrollment in the HOPE-2 trial and completion of trial follow-up through Month 12

I am willing and able to agree to participate in the trial, and if under 18, I have parental or guardian consent.

I am willing and able to follow the trial's requirements.

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Exclusion Criteria

I have a chronic lung condition not related to muscular dystrophy.

History, or current use, of drugs or alcohol that could impair ability to comply with participation in the trial

Treatment with an investigational product ≤ 6 months prior to first infusion

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Treatment Details

Interventions

CAP-1002 (Cell Therapy)

Trial OverviewThe safety and effectiveness of CAP-1002 are being tested through sixteen intravenous doses given every three months over two years. Participants will have regular check-ups before each dose to monitor their response to the treatment.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: DeramiocelExperimental Treatment1 Intervention

Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months for a total of 20 IV infusions