sepofarsen: RNA antisense oligonucleotide for intravitreal injection
Placebo IVT: Placebo with identical appearance to sepofarsen
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.
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Wayne and Gladys Valley Center for Vision | San Francisco | California | Recruiting |
| University of Miami - Bascom Palmer Eye Institute | Miami | Florida | Recruiting |
| University of Iowa | Iowa City | Iowa | Recruiting |
| University of Minnesota Medical School | Minneapolis | Minnesota | Recruiting |
| University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics | Philadelphia | Pennsylvania | Recruiting |
| Universitair Ziekenhuis Gent (UZ) | Ghent | Belgium | Recruiting |
| INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte | Belo Horizonte | Minas Gerais | Recruiting |
| Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) | São Paulo | São Paulo | Recruiting |
| University of Alberta | Edmonton | Alberta | Recruiting |
| The Hospital for Sick Children - SickKids | Toronto | Ontario | Recruiting |
| Centre de maladies rares CHNO des Quinze Vingt | Paris | France | Recruiting |
| Justus-Liebig Universität - Department of Ophthalmology | Giessen | Germany | Recruiting |
| Klinikum der Ludwig-Maximilian Universität München | München | Germany | Recruiting |
| University of Tuebingen - Inst. for Ophthalmic Research | Tübingen | Germany | Recruiting |
| Radboud Universitair Medisch Centrum | Nijmegen | Netherlands | Recruiting |
| Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital) | Barcelona | Spain | Recruiting |
| Moorfields Eye Hospital NHS Foundation Trust | London | United Kingdom | Recruiting |
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06891443 on ClinicalTrials.gov ↗ ← All trials in the UK