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Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)

Leber Congenital Amaurosis Clinical Trial

Official title:
An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy for 2 Leber's Congenital Amaurosis With RPE65 Mutation (LCA2)

Clinical Trial Summary

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 mutations in RPE65 gene.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06064565
Study type Interventional
Source HuidaGene Therapeutics Co., Ltd.
Contact Director Study
Phone +86 021-25076143
Email HG00401@huidagene.com
Status Recruiting
Phase Early Phase 1
Start date January 10, 2023
Completion date December 30, 2024
View Details
See also
  Status Clinical Trial Phase
Completed NCT01496040 - Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 Phase 1/Phase 2
Completed NCT02970266 - Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. N/A
Completed NCT00516477 - Safety Study in Subjects With Leber Congenital Amaurosis Phase 1
Active, not recruiting NCT03920007 - Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D Phase 1/Phase 2
Recruiting NCT05906953 - Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) Phase 1/Phase 2
Active, not recruiting NCT00999609 - Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis Phase 3
Active, not recruiting NCT01208389 - Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 Phase 1/Phase 2
Terminated NCT03913130 - Extension Study to Study PQ-110-001 (NCT03140969) Phase 1/Phase 2
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. Phase 2/Phase 3
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT00749957 - Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Not yet recruiting NCT04731883 - RPE65 Gene Therapy （LX101）for Leber's Congenital Amaurosis N/A
Recruiting NCT05203939 - Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis Phase 1/Phase 2
Recruiting NCT06088992 - Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) Early Phase 1
Completed NCT00821340 - Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Phase 1
Active, not recruiting NCT03913143 - A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) Phase 2/Phase 3
Completed NCT02714816 - Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
Completed NCT02781480 - Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) Phase 1/Phase 2