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5 recruiting studies matching your filters

A Phase 1b/2a Open-Label, Dose-Exploration Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-RDH12 Administered in Participants With Leber Congenital Amaurosis With Autosomal-Recessive Retinol Dehydrogenase 12 Mutations

This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by m…

Leber Congenital AmaurosisLeber Congenital Amaurosis (LCA)

Estimated

~$10 – $8,750

Opus Genetics, IncNCT07681778

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

Estimated

~$10 – $7,500

Laboratoires TheaNCT06891443

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

Estimated

~$10 – $750

Foundation Fighting BlindnessNCT02435940

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

Estimated

~$10 – $8,750

HuidaGene Therapeutics Co., Ltd.NCT05906953

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