Myopathy clinical trials at UCSD
2 in progress, 1 open to eligible people
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
open to all eligible people
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
San Diego, California and other locations
Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Sorry, in progress, not accepting new patients
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.
La Jolla, California and other locations
Our lead scientists for Myopathy research studies include Richard Haas, MD PhD Robert Naviaux, MD.