Atrophic Muscular Disorders clinical trials at University of California Health
2 in progress, 0 open to eligible people
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.
at UC Davis UCSD
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Sorry, in progress, not accepting new patients
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
at UCLA
Our lead scientists for Atrophic Muscular Disorders research studies include Craig McDonald, MD.
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