Clinical Trial

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Study acronym: HYPERION
Recruiting Phase 3
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Summary
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.
Protocol Amendment History 9 amendments
This ClinicalTrials.gov record has been amended 9 times since 2025-03-21; most recent amendment 2026-06-24.
Status change: Not Yet Recruiting → Recruiting 2025-06-11
Trial Details
NCT Number NCT06891443
Lead Sponsor Laboratoires Thea
Collaborators: Sepul Bio
Conditions Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases +2 more
Enrollment 32 participants
Start Date 2025-06-04
Primary Completion 2027-11 (estimated)
Study Completion 2028-10 (estimated)
Updated on ClinicalTrials.gov 2026-06-25