Leber Congenital Amaurosis clinical trials at University of California Health

2 in progress, 1 open to eligible people

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.

    at UCSF

  • OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

    Sorry, in progress, not accepting new patients

    This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101). To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study (OCU400-104)i This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study.

    at UCSD

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