Muscular Disorders, Atrophic clinical trials at University of California Health
3 in progress, 1 open to eligible people
open to eligible males ages 10 years and up
HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called 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 CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period. All participants will be eligible to receive CAP-1002 for an additional 12 months as part of an open label extended safety assessment period.
at UC Davis
Sorry, accepting new patients by invitation only
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
Sorry, in progress, not accepting new patients
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
at UC Davis UCSF
Our lead scientists for Muscular Disorders, Atrophic research studies include Craig McDonald, MD.