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

NCT05126758 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 106

Last updated 2025-12-17

No results posted yet for this study

Summary

HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.

Conditions

  • Muscular Dystrophies
  • Muscular Dystrophy, Duchenne
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Neuromuscular Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
  • Nervous System Diseases

Interventions

BIOLOGICAL

Deramiocel (CAP-1002)

Cohort A: CAP-1002A manufactured in Los Angeles, CA; Cohort B: CAP-1002B manufactured in San Diego, CA

BIOLOGICAL

Placebo

Placebo

Sponsors & Collaborators

  • Capricor Inc.

    lead INDUSTRY

Principal Investigators

  • Craig McDonald, MD · University of California, Davis

  • Mark Awadalla · Capricor Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
10 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-22
Primary Completion
2025-06-18
Completion
2026-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05126758 on ClinicalTrials.gov