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

• Sponsor: GenSight Biologics (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02064569
• Collaborator: (none)
• Enrollment: 19
• Location: 1
• Arm: 1
• Actual Duration (Months): 76.4
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Leber Hereditary Optic Neuropathy	Genetic: GS010	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Feb 13, 2014
• Actual Primary Completion Date: Jun 25, 2020
• Actual Study Completion Date: Jun 25, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: GS010	Genetic: GS010

Outcome Measures

Primary Outcome Measures

1. Incidence of local and general adverse events and Serious Adverse Events [Up to 48 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4

Age 18 years old or older at the time of study entry (informed consent signature)

Visual acuity ≤ 1/10 of the less functional eye

Exclusion Criteria:

Any known allergy or hypersensibility to one of the product used during the trial

Contraindication to IVT surgery (anaemia Hb <8g/dl, severe cardiovascular disease, severe coagulopathy…)

Disorder of the ocular humors and of the internal retina involving visual disability

Glaucoma

Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion

Narrow angle contra-indicating pupillary dilation

Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)

Patients presenting known mutation of other genes implicated in pathological retinal conditions

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	CIC du CHNO DES QUINZE-VINGTS	Paris		France	75012

Sponsors and Collaborators

• GenSight Biologics

Investigators

• Principal Investigator: CATHERINE VIGNAL, MD, CIC CHNO DES QUINZE VINGTS

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Sponsor web site

Publications

None provided.

• Responsible Party: GenSight Biologics
• ClinicalTrials.gov Identifier: NCT02064569
• Other Study ID Numbers: GS-LHON/CLIN/01
• First Posted: Feb 17, 2014
• Last Update Posted: Jul 1, 2020
• Last Verified: Jun 1, 2020

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Additional relevant MeSH terms: Optic Nerve Diseases; Optic Atrophy, Hereditary, Leber

Study Results

No Results Posted as of Jul 1, 2020