RecruitingPhase 3NCT06891443

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

Sponsor

Laboratoires Thea

Enrollment

32 participants

Start Date

Jun 4, 2025

Study Type

INTERVENTIONAL

Conditions

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

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.

Eligibility

Min Age: 6 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying Placebo IVT and a drug called sepofarsen for people with blindness, eye diseases, and other related conditions. The study is currently recruiting participants at 16 locations. People eligible for this study include aged 6 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGsepofarsen

RNA antisense oligonucleotide for intravitreal injection

OTHERPlacebo IVT

Placebo with identical appearance to sepofarsen

Locations(17)

UCSF Wayne and Gladys Valley Center for Vision

San Francisco, California, United States

University of Miami - Bascom Palmer Eye Institute

Miami, Florida, United States

University of Iowa

Iowa City, Iowa, United States

University of Minnesota Medical School

Minneapolis, Minnesota, United States

University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics

Philadelphia, Pennsylvania, United States

Universitair Ziekenhuis Gent (UZ)

Ghent, Belgium

INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, Minas Gerais, Brazil

Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP)

São Paulo, São Paulo, Brazil

University of Alberta

Edmonton, Alberta, Canada

The Hospital for Sick Children - SickKids

Toronto, Ontario, Canada

Centre de maladies rares CHNO des Quinze Vingt

Paris, France

Justus-Liebig Universität - Department of Ophthalmology

Giessen, Germany

Klinikum der Ludwig-Maximilian Universität München

München, Germany

University of Tuebingen - Inst. for Ophthalmic Research

Tübingen, Germany

Radboud Universitair Medisch Centrum

Nijmegen, Netherlands

Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital)

Barcelona, Spain

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06891443

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