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

  • 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?”

    open to eligible males ages 10 years and up

    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 UC Davis

  • 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

  • Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

    Sorry, in progress, not accepting new patients

    The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.

    at UCLA UC Davis

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