Leber Congenital Amaurosis Inherited Blindness of Gene Therapy

Updated 05/03/2024

Status Recruiting

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 mutationsin RPE65 gene.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06088992
Study type	Interventional
Source	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Contact	Director Study
Phone	+862125076143
Email	HG00401@huidagene.com
Status	Recruiting
Phase	Early Phase 1
Start date	January 10, 2023
Completion date	December 30, 2024

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	`NCT06064565` - 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	`NCT02781480` - Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)	Phase 1/Phase 2
	Completed	`NCT02714816` - Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65