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Retinal Disease clinical trials at UCSF

3 in progress, 2 open to eligible people

Retinal diseases damage the eye's ability to process light and send signals to the brain. At UCSF, scientists are testing sepofarsen for LCA Type 10 and ultevursen for RP due to specific genetic mutations. These trials focus on drug safety and 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

  • Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

    open to eligible people ages 8 years and up

    The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.

    San Francisco, California and other locations

  • Global Patient Registry of Inherited Retinal Diseases

    Sorry, in progress, not accepting new patients

    The purpose of this study is to better understand the natural history of Inherited Retinal Disease (IRD) and help inform patient management.

    San Francisco, California and other locations

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