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Completed
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Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
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Phase 1/Phase 2 |
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Completed
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Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
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N/A |
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Completed
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Safety Study in Subjects With Leber Congenital Amaurosis
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Phase 1 |
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Active, not recruiting
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NCT03920007 -
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
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Phase 1/Phase 2 |
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Recruiting
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Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
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Phase 1/Phase 2 |
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Active, not recruiting
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NCT00999609 -
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
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Phase 3 |
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Active, not recruiting
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NCT01208389 -
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
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Phase 1/Phase 2 |
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Terminated
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NCT03913130 -
Extension Study to Study PQ-110-001 (NCT03140969)
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Phase 1/Phase 2 |
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Recruiting
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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.
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Phase 2/Phase 3 |
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Recruiting
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NCT02435940 -
Inherited Retinal Degenerative Disease Registry
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Completed
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NCT00749957 -
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
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Phase 1/Phase 2 |
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Recruiting
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Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Not yet recruiting
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NCT04731883 -
RPE65 Gene Therapy (LX101)for Leber's Congenital Amaurosis
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N/A |
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Recruiting
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Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
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Phase 1/Phase 2 |
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Recruiting
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NCT06064565 -
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)
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Early Phase 1 |
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Recruiting
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NCT06088992 -
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
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Early Phase 1 |
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Completed
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NCT00821340 -
Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
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Phase 1 |
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Active, not recruiting
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NCT03913143 -
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
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Phase 2/Phase 3 |
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Completed
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NCT02781480 -
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
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Phase 1/Phase 2 |