Trials / NCT06891443
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Recruiting now Phase 3 RNA / antisense
- Sponsor
- Laboratoires Thea
- Treatment
- sepofarsen; Placebo IVT
- Gene
- 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
- Ages
- 6 Years to no upper limit
- Enrollment
- 32 participants
- Started
- 2025-06-04
- Main results expected
- 2027-11
- Results posted
- Not yet
- Record last updated
- 2026-10-21
What the study is testing
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.
Where it runs
Australia (1)
- Sydney Eye Hospital, Sydney, New South Wales (recruiting now)
Belgium (1)
- Universitair Ziekenhuis Gent (UZ), Ghent (recruiting now)
Brazil (2)
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte, Belo Horizonte, Minas Gerais (recruiting now)
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP), São Paulo, São Paulo (recruiting now)
Canada (2)
- University of Alberta, Edmonton, Alberta (recruiting now)
- The Hospital for Sick Children - SickKids, Toronto, Ontario (recruiting now)
France (1)
- Centre de maladies rares CHNO des Quinze Vingt, Paris (recruiting now)
Germany (3)
- Justus-Liebig Universität - Department of Ophthalmology, Giessen (recruiting now)
- Klinikum der Ludwig-Maximilian Universität München, München (recruiting now)
- University of Tuebingen - Inst. for Ophthalmic Research, Tübingen (recruiting now)
Netherlands (1)
- Radboud Universitair Medisch Centrum, Nijmegen (recruiting now)
Spain (1)
- Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital), Barcelona (recruiting now)
United Kingdom (1)
- Moorfields Eye Hospital NHS Foundation Trust, London (recruiting now)
United States (5)
- UCSF Wayne and Gladys Valley Center for Vision, San Francisco, California (recruiting now)
- University of Miami - Bascom Palmer Eye Institute, Miami, Florida (recruiting now)
- University of Iowa, Iowa City, Iowa (recruiting now)
- University of Minnesota Medical School, Minneapolis, Minnesota (withdrawn)
- University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics, Philadelphia, Pennsylvania (recruiting now)