Leber Congenital Amaurosis clinical trials at UCSF

1 research study open to eligible people

Leber congenital amaurosis is a genetic disorder that causes very poor vision from birth. UCSF is running a study that gives an experimental treatment to one eye and a placebo to the other, with random assignment and masking. UCSF is tracking vision and safety during planned clinic visits.

Showing trials for

  • Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

    open to eligible people ages 6 years and up

    The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290.

    San Francisco, California and other locations

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