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.
Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A>G mutation in CEP290.
Adults: >=18 years / Minors: 6 to <18 years.
BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.
Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).
Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
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Treatment groupsSee each treatment group below.
Laboratoires Thea
Lead sponsor
Sepul Bio
Collaborator