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

PHASE3RecruitingINTERVENTIONAL
Enrollment

32

Participants

Timeline

Start Date

June 4, 2025

Primary Completion Date

November 30, 2027

Study Completion Date

October 31, 2028

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

sepofarsen

RNA antisense oligonucleotide for intravitreal injection

OTHER

Placebo IVT

Placebo with identical appearance to sepofarsen

Trial Locations (12)

9000

RECRUITING

Universitair Ziekenhuis Gent (UZ), Ghent

19104

RECRUITING

University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics, Philadelphia

33156

RECRUITING

University of Miami - Bascom Palmer Eye Institute, Miami

35392

RECRUITING

Justus-Liebig Universität - Department of Ophthalmology, Giessen

52242

RECRUITING

University of Iowa, Iowa City

55455

RECRUITING

University of Minnesota Medical School, Minneapolis

72076

RECRUITING

University of Tuebingen - Inst. for Ophthalmic Research, Tübingen

75012

RECRUITING

Centre de maladies rares CHNO des Quinze Vingt, Paris

94158

RECRUITING

UCSF Wayne and Gladys Valley Center for Vision, San Francisco

T6G 2C8

RECRUITING

University of Alberta, Edmonton

6525 GA

RECRUITING

Radboud Universitair Medisch Centrum, Nijmegen

EC1V 2PD

RECRUITING

Moorfields Eye Hospital NHS Foundation Trust, London

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Sepul Bio

INDUSTRY

lead

Laboratoires Thea

INDUSTRY