Skip to main content

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

  • A Study of Experimental Treatment With Metformin for Fragile X Syndrome

    “Learn if Metformin may be helpful for language, behavior problems, and obesity / excessive appetite in patients with Fragile X Syndrome.”

    open to eligible people ages 6-25

    This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.

    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

  • A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

    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

Last updated: