Neuromuscular Diseases clinical trials at UC Health
3 in progress, 2 open to new patients

  • AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

    open to eligible people ages 18-80

    The CENTAUR trial will be a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.

    at UC Irvine

  • Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    open to eligible males ages 5 years and up

    This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

    at UCSF UC Davis

  • A Study of Experimental Cell Therapy (CAP-1002) in Patients With Duchenne Muscular Dystrophy (DMD)

    “Would the study drug (CAP-1002) help improve weakened skeletal and heart muscle caused by DMD?”

    Sorry, in progress, not accepting new patients

    HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). 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.

    at UCLA UC Davis

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