Trials / NCT03913143
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
Underway, not enrolling Phase 2 / Phase 3 RNA / antisense
- Sponsor
- ProQR Therapeutics
- Treatment
- sepofarsen; Sham
- Gene
- CEP290
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
- Ages
- 8 Years to no upper limit
- Enrollment
- 36 participants
- Started
- 2019-04-04
- Main results expected
- 2022-01-31
- Results posted
- Not yet
- Record last updated
- 2022-03-17
What the study is testing
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment
Where it runs
Belgium (1)
- Universitair Ziekenhuis Gent (UZ), Ghent
Brazil (2)
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte, Belo Horizonte, Minas Gerais
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP), São Paulo, São Paulo
Canada (2)
- The Hospital for Sick Children - SickKids, Toronto, Ontario
- McGill University Health Centre - Centre for Innovative Medicine, Montreal, Quebec
France (2)
- Centre de maladies rares CHNO des Quinze Vingt, Paris
- Hospital Civil de Strasbourg, Strasbourg
Germany (2)
- Justus-Liebig Universität - Department of Ophthalmology, Giessen
- University of Tuebingen - Inst. for Ophthalmic Research, Tübingen
Italy (1)
- Eye Clinic University of Campania Luigi Vanvitelli, Naples
Netherlands (2)
- Amsterdam University Medica Center - Locatie AMC, Amsterdam
- Het Oogziekenhuis Rotterdam, Rotterdam
United Kingdom (1)
- Moorfields Eye Hospital - NHS Foundation Trust, London
United States (1)
- University of Iowa, Iowa City, Iowa