Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial

Who is this study for? Child to adult patients with Duchenne Muscular Dystrophy
What treatments are being studied? CAP-1002 Allogeneic Cardiosphere-Derived Cells
Status: Active_not_recruiting
Location: See all (5) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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, over a period of approximately 60 months. Following completion of the initial open-label phase (Month 60), subjects who have completed all Month 60 assessments will be eligible to continue into a long-term open label extension (LT-OLE) period and can continue to receive deramiocel once every 3 months until deramiocel is commercially available or the sponsor terminates the study, or the subject withdraws consent or study participation is terminated by the sponsor. 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, 60, and all LT-OLE visits. 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, 57, and at all LT-OLE 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, 57, with continued dosing in the LT-OLE visits. 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 10
Healthy Volunteers: f
View:

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

• Willing and able to provide informed consent to participate in the trial if ≥ 18 years of age, and assent with parental or guardian informed consent if \< 18 years of age

• Adequate venous access for intravenous deramiocel (CAP-1002) infusions in the judgement of the Investigator

• Assessed by the Investigator as willing and able to comply with the requirements of the trial

Locations
United States
California
University of California, Davis
Sacramento
Colorado
Children's Hospital Colorado
Aurora
Missouri
Washington University
St Louis
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Wisconsin
Children's Hospital Wisconsin
Milwaukee
Time Frame
Start Date: 2020-08-05
Completion Date: 2026-05
Participants
Target number of participants: 13
Treatments
Other: Deramiocel
Participants will receive an intravenous (IV) infusion of deramiocel (150 million Cardiosphere-Derived Cells (CDCs) per infusion) every 3 months
Sponsors
Leads: Capricor Inc.

This content was sourced from clinicaltrials.gov

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