Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

Leber Hereditary Optic Neuropathy Clinical Trial

Official title:
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene

Status Completed

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Leber Hereditary Optic Neuropathy
Optic Atrophy, Hereditary, Leber
Optic Nerve Diseases

Clinical Trial Details

NCT number	NCT02064569
Study type	Interventional
Source	GenSight Biologics
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	February 13, 2014
Completion date	June 25, 2020

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT03475173` - New Non-invasive Modalities for Assessing Retinal Structure and Function	N/A
Active, not recruiting	`NCT03293524` - Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year	Phase 3
Recruiting	`NCT02176733` - Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy	Phase 2
Completed	`NCT03406104` - RESCUE and REVERSE Long-term Follow-up	Phase 3
Completed	`NCT03295071` - REALITY LHON Registry
Recruiting	`NCT03011541` - Stem Cell Ophthalmology Treatment Study II	N/A
Active, not recruiting	`NCT03153293` - A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy	Phase 2/Phase 3
Completed	`NCT01267422` - Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)	N/A