Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy

Updated 06/21/2024

Status Completed

Clinical Trial Summary

The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.

Clinical Trial Description

n/a

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01496040
Study type	Interventional
Source	Nantes University Hospital
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	September 2011
Completion date	August 2014

See also
	Status	Clinical Trial	Phase
	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
	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	`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