Leber Congenital Amaurosis clinical trials at UCSF

1 research study open to eligible people

Leber congenital amaurosis is a genetic condition affecting vision from early childhood. UCSF is exploring whether sepofarsen can improve sight in affected individuals through a carefully controlled study. Researchers are testing the drug against a placebo in one eye to check for effectiveness.

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  • 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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