NCT06891443

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

A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study 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 Gene

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 32 (estimated)
• Sponsor: Laboratoires Thea · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a double-masked, randomized, placebo-controlled, paired-eye study in which one eye of each subject will serve as a control. At the start of the study the two eyes of each subject will be randomized such that one eye receives sepofarsen and the other eye receives placebo for the first year. In the second year, for all subjects, the eye that was randomized to receive sepofarsen will continue to receive sepofarsen. For the eye that was randomized to placebo in the first year, treatment in the second year will be allocated, as follows: 50% of the eyes will continue to receive placebo, and 50% of the eyes will receive sepofarsen. Sepofarsen and placebo will be administered via intravitreal injection every 6 months.

Conditions

• Leber Congenital Amaurosis 10
• Blindness
• Leber Congenital Amaurosis
• Sensation Disorders
• Vision Disorder
• Neurological Manifestations
• Eye Diseases, Hereditary
• Eye Diseases
• Eye Disorders Congenital
• Retinal Disease

Interventions

Type	Name	Description
DRUG	sepofarsen	RNA antisense oligonucleotide for intravitreal injection
OTHER	Placebo IVT	Placebo with identical appearance to sepofarsen

Timeline

• Start date: 2025-06-04
• Primary completion: 2027-11-01
• Completion: 2028-10-01
• First posted: 2025-03-24
• Last updated: 2026-03-17

Locations

14 sites across 7 countries: United States, Belgium, Canada, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study