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Phase 1
A Phase 1/2, Open-label, Multi-national, Multiple-cohort, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of HG004 Gene Therapy in Subjects with RPE65-associated Leber Congenital Amaurosis Type 2 (LCA2)
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutation…
Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd.NCT05906953
Foundation Fighting Blindness My Retina Tracker Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative …
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting BlindnessNCT02435940
Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, …
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Phase 3
A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study 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 Gene
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subje…
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
Laboratoires TheaNCT06891443