A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
Running, not enrolling · Phase 2/Phase 3 · Has a placebo group
Conditions studied: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
In brief
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
Key facts
- Study ID
- NCT03913143
- Run by
- ProQR Therapeutics
- People needed
- 36
- Starts
- 2019-04-04
- Expected to finish
- 2023-03-01
- Last updated by the study team
- 2022-03-17
Who can join
Age: 8 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of Iowa — Iowa City, Iowa, United States
- Universitair Ziekenhuis Gent (UZ) — Ghent, Belgium
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte — Belo Horizonte, Minas Gerais, Brazil
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) — São Paulo, São Paulo, Brazil
- The Hospital for Sick Children - SickKids — Toronto, Ontario, Canada
- McGill University Health Centre - Centre for Innovative Medicine — Montreal, Quebec, Canada
- Centre de maladies rares CHNO des Quinze Vingt — Paris, France
- Hospital Civil de Strasbourg — Strasbourg, France
- Justus-Liebig Universität - Department of Ophthalmology — Giessen, Germany
- University of Tuebingen - Inst. for Ophthalmic Research — Tübingen, Germany
- Eye Clinic University of Campania Luigi Vanvitelli — Naples, Italy
- Amsterdam University Medica Center - Locatie AMC — Amsterdam, Netherlands
- Het Oogziekenhuis Rotterdam — Rotterdam, Netherlands
- Moorfields Eye Hospital - NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
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