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NCT05126758 · ClinicalTrials.gov registry record · Phase 3
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
A Phase 3 study of Nervous System Diseases and Neuromuscular Diseases, sponsored by Capricor.
- Active
- Registry status
- Phase 3
- Development phase
- 106
- Enrollment target
- 20
- Study locations
NCT05126758: Active Phase 3 study of Nervous System Diseases and Neuromuscular Diseases, sponsored by Capricor.
NCT05126758 is a Phase 3 study of Nervous System Diseases and Neuromuscular Diseases that is active but no longer recruiting, run by Capricor. The registered enrollment target is 106 participants, below the 319-participant average among 23 other Nervous System Diseases trials with a reported enrollment target (67% lower). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05126758, a Phase 3 study of Nervous System Diseases and Neuromuscular Diseases, is active but no longer recruiting, sponsored by Capricor.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 106 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
Mean percent change from baseline in Performance of the Upper Limb test, version 2 (PUL 2.0) Total Score. Items are scored on a three-point scale: 0=unable to perform the item, 1=impaired or performs with compensation, 2=performs task without compensation. Percent change from baseline is calculated as change from baseline divided by baseline score at the subject level.
Conditions Studied
Interventions
- BIOLOGICAL Placebo
- BIOLOGICAL Deramiocel (CAP-1002)
Study Locations (20)
California
- UCSD Altman Clinical and Translational Research Institute - La Jolla
- Children's Hospital of Los Angeles, Division of Neurology - Los Angeles
- University of California, Davis - Sacramento
Missouri
- University of Missouri Health Care - Columbia
- Saint Louis Children's Hospital - St Louis
Ohio
- Akron Children's Hospital - Akron
- Cincinnati Children's Hospital Medical Center - Cincinnati
Arizona
- Phoenix Children's Hospital - Phoenix
Arkansas
- Arkansas Children's Hospital - Little Rock
Colorado
- Children's Hospital Colorado - Aurora
Georgia
- Rare Disease Research, LLC - Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 106 participants |
| Start Date | 2022-06-22 |
| Est. Completion | 2026-12 |
| Phase | Phase 3 |
What the registry record for NCT05126758 still lists
NCT05126758 is an interventional study that assigns participants to a tested intervention. The registered 106 participants enrollment target is mid-sized for trials with a published cap, below the 319-participant average among 23 other Nervous System Diseases trials with a reported enrollment target (67% lower).
The record links to 8 conditions, with Nervous System Diseases appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05126758 names 20 study sites across 16 states, led by California, Missouri, Ohio.
Frequently Asked Questions
What is clinical trial NCT05126758 about?
NCT05126758 is a clinical study titled "A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy". 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-ambulat...
What is the current status of trial NCT05126758?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 106 participants. The study started on 2022-06-22. Estimated completion is 2026-12.
What conditions does trial NCT05126758 study?
This clinical trial studies the following conditions: Nervous System Diseases, Neuromuscular Diseases, Muscular Dystrophies, Muscular Diseases, Genetic Diseases, Inborn.
What interventions are being tested in trial NCT05126758?
The interventions under investigation include: Placebo (BIOLOGICAL), Deramiocel (CAP-1002) (BIOLOGICAL).
Who is sponsoring clinical trial NCT05126758?
This trial is sponsored by Capricor, which has 9 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05126758 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, Colorado, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Where NCT05126758's enrollment target sits among peer trials
106 10th of 23 higher than 14 of 23 other Nervous System Diseases trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Nervous System Diseases trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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