Back to Trials
NCT06891443PHASE3Recruiting

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

Laboratoires Thea

Start Date

6/4/2025

Completion Date

10/1/2028

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.

Eligibility Criteria

Age Range: 6 years to No maximum

Inclusion Criteria: 1. Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290. 2. Adults: \>=18 years / Minors: 6 to \<18 years. 3. 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. 4. Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline. 5. Detectable ONL in the macular area as determined by the CRC at Screening. Exclusion Criteria: 1. Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes. 2. Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible. 3. 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). 4. Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale. 5. Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.

Interventions

DRUG

sepofarsen

OTHER

Placebo IVT

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Leber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisSensation DisordersVision DisorderNeurological ManifestationsEye Diseases, HereditaryEye DiseasesEye Disorders CongenitalRetinal Disease

Locations

UCSF Wayne and Gladys Valley Center for Vision

San Francisco, California 94158

United States

University of Miami - Bascom Palmer Eye Institute

Miami, Florida 33156

United States

University of Iowa

Iowa City, Iowa 52242

United States

University of Minnesota Medical School

Minneapolis, Minnesota 55455

United States

University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics

Philadelphia, Pennsylvania 19104

United States

Universitair Ziekenhuis Gent (UZ)

Ghent, 9000

Belgium

INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, Minas Gerais 30150270

Brazil

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

São Paulo, São Paulo 04023-062

Brazil

University of Alberta

Edmonton, Alberta T6G 2C8

Canada

The Hospital for Sick Children - SickKids

Toronto, Ontario M5G 2L3

Canada

Centre de maladies rares CHNO des Quinze Vingt

Paris, 75012

France

Justus-Liebig Universität - Department of Ophthalmology

Giessen, 35392

Germany

Klinikum der Ludwig-Maximilian Universität München

München, 81377

Germany

University of Tuebingen - Inst. for Ophthalmic Research

Tübingen, 72076

Germany

Radboud Universitair Medisch Centrum

Nijmegen, 6525 GA

Netherlands

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

Barcelona, 08950

Spain

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD

United Kingdom