Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
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
Interventions
sepofarsen
Placebo IVT
Conditions
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