Leber Congenital Amaurosis Clinical Trials

27 clinical trials for Leber Congenital Amaurosis across international registries, including 5 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) — RECRUITING · PHASE3
• Inherited Retinal Degenerative Disease Registry — RECRUITING
• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford — RECRUITING
• 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 — RECRUITING
• Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) — RECRUITING · PHASE1
• Safety and Efficacy Study of Gene Therapy for RPE65-mediated Inherited Retinal Disease — OTHER · PHASE3
• Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D — ACTIVE_NOT_RECRUITING · PHASE1
• Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis — ACTIVE_NOT_RECRUITING · PHASE1
• Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD) — NOT_YET_RECRUITING · PHASE1
• Psychotherapy Group for Parents of Children With LCA — ACTIVE_NOT_RECRUITING · NA
• 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 — TERMINATED · PHASE3
• Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 — ACTIVE_NOT_RECRUITING · PHASE1
• Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis — ACTIVE_NOT_RECRUITING · PHASE3
• Extension Study to Study PQ-110-001 (NCT03140969) — TERMINATED · PHASE1
• Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) — ACTIVE_NOT_RECRUITING · PHASE1
• Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) — COMPLETED
• Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 — COMPLETED
• 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. — OTHER · PHASE2
• A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) — ACTIVE_NOT_RECRUITING · PHASE2
• AN OPEN-LABEL, DOSE ESCALATION AND DOUBLE-MASKED, RANDOMIZED, CONTROLLED STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF SEPOFARSEN IN PEDIATRIC SUBJECTS <8 YEARS OF AGE WITH LEBER CONGENITAL AMAUROSIS TYPE 10(LCA10) DUE TO THE P.CYS998X) MUTATION — NOT_YET_RECRUITING
• Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) — COMPLETED · PHASE1
• Safety Study in Subjects With Leber Congenital Amaurosis — COMPLETED · PHASE1
• Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations — COMPLETED · PHASE1
• Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis — COMPLETED · PHASE1
• Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. — COMPLETED
• Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT — COMPLETED
• Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 — COMPLETED · PHASE1

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