Muscular Dystrophies clinical trials at UCLA

2 in progress, 1 open to eligible people

Showing trials for

  • SAT-3247 in Pediatric Ambulatory Patients

    open to eligible males ages 7-9

    Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial has two parts. In Part 1, the trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.

    Los Angeles, California and other locations

  • AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

    Sorry, in progress, not accepting new patients

    A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

    Los Angeles, California and other locations

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