Trials / NCT06627179
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
Underway, not enrolling Phase 2 RNA / antisense
- Sponsor
- Laboratoires Thea
- Treatment
- Intravitreal Injection of Ultevursen; No intervention, will not receive any active study intervention
- Gene
- USH2A
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
- Ages
- 8 Years to no upper limit
- Enrollment
- 81 participants
- Started
- 2024-12-11
- Main results expected
- 2028-07
- Results posted
- Not yet
- Record last updated
- 2026-07-09
What the study is testing
The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
Where it runs
Belgium (1)
- Ghent University Hospital, Ghent
Brazil (2)
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte, Belo Horizonte
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP), São Paulo
Canada (2)
- Hospital for Sick Children, Toronto, Ontario
- McGill University Health Centre for Innovative Medicine, Montreal, Quebec
Denmark (1)
- Rigshospitalet and University of Copenhagen, Glostrup Municipality
France (2)
- Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique, Montpellier
- Centre de maladies rares CHNO des Quinze Vingt, Paris
Germany (1)
- Universitätsklinikum Tübingen, Tübingen
Italy (1)
- ASST Santi Paolo e Carlo Hospital, University of Milan, Milan
Netherlands (3)
- Amsterdam University Medical Center - Locatie AMC, Amsterdam
- Radboud Universitair Medisch Centrum, Nijmegen
- Het Oogziekenhuis Rotterdam, Rotterdam
United Kingdom (3)
- Oxford Eye Hospital, Headington, Oxford
- University of Edinburgh / NHS Lothian, Edinburgh
- Moorfields Eye Hosptial, London
United States (10)
- The University of California, San Francisco, San Francisco, California
- Bascom Palmer Eye Institute/University of Miami, Miami, Florida
- Emory University, Atlanta, Georgia
- Massachusetts Eye and Ear, Boston, Massachusetts
- University of Michigan- Kellogg Eye Center, Ann Arbor, Michigan
- Casey Eye Institute, Oregon Health & Science University, Portland, Oregon
- University of Pennsylvania, Scheie Eye Institute, Philadelphia, Pennsylvania
- Retina Foundation of the Southwest, Dallas, Texas
- Baylor College of Medicine, Houston, Texas
- University of Wisconsin- Madison, Madison, Wisconsin