NCT03913130
Extension Study to Study PQ-110-001 (NCT03140969) treatment 1 / 2 terminated NCT05203939
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis treatment 1 / 2 active_not_recruiting NCT05906953
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) treatment 1 / 2 recruiting NCT06088992
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) No drug interventions treatment 0 active_not_recruiting NCT04855045
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. treatment 2 / 3 unknown_status NCT03920007
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D treatment 1 / 2 active_not_recruiting NCT00999609
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis treatment 3 active_not_recruiting NCT02714816
Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 No drug interventions Not Available Not Available completed NCT00749957
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis No drug interventions treatment 1 / 2 completed NCT02970266
Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. No drug interventions Not Available Not Available completed NCT01496040
Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 No drug interventions Not Available 1 / 2 completed NCT00821340
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations No drug interventions treatment 1 completed NCT00516477
Safety Study in Subjects With Leber Congenital Amaurosis treatment 1 completed NCT02781480
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) No drug interventions treatment 1 / 2 completed NCT01208389
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 treatment 1 / 2 active_not_recruiting NCT02435940
Inherited Retinal Degenerative Disease Registry No drug interventions Not Available Not Available recruiting NCT01793168
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford No drug interventions Not Available Not Available recruiting NCT03913143
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) treatment 2 / 3 active_not_recruiting NCT03140969
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene treatment 1 / 2 completed NCT02946879
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) No drug interventions Not Available Not Available completed NCT02575430
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT No drug interventions Not Available Not Available completed NCT01014052
Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations treatment 1 completed NCT01521793
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) treatment 1 completed