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

NCT06891443 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2026-03-17

No results posted yet for this study

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.

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

DRUG

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

OTHER

Placebo IVT

Placebo with identical appearance to sepofarsen

Sponsors & Collaborators

  • Sepul Bio

    collaborator INDUSTRY

  • Laboratoires Thea

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-04
Primary Completion
2027-11-30
Completion
2028-10-31
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • France
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06891443 on ClinicalTrials.gov