Status: Recruiting
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
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.
Participants needed: 32
Trial details
Phase: Phase 3Age: 6+Biological sex: AllType: InterventionalSponsor: Laboratoires TheaUpdated: Jun 25, 2026Locations: 17
Eligibility criteria
Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity... [+4]
Mutations in genes other than the CEP290 gene associated with other IRD diseases... [+4]