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

• ClinicalTrials.gov Identifier: NCT02064569
• Recruitment Status: Completed
• First Posted: February 17, 2014
• Last Update Posted: July 1, 2020
• Sponsor: GenSight Biologics
• Information provided by (Responsible Party): GenSight Biologics

Tracking Information

• First Submitted Date: February 13, 2014
• First Posted Date: February 17, 2014
• Last Update Posted Date: July 1, 2020
• Actual Study Start Date: February 13, 2014
• Actual Primary Completion Date: June 25, 2020 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 14, 2014): Incidence of local and general adverse events and Serious Adverse Events [ Time Frame: Up to 48 weeks ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Leber Hereditary Optic Neuropathy
• Intervention: Genetic: GS010

Study Arms

Experimental: GS010

Intervention: Genetic: GS010

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 22, 2019): 19
• Original Estimated Enrollment (submitted: February 14, 2014): 18
• Actual Study Completion Date: June 25, 2020
• Actual Primary Completion Date: June 25, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT02064569
• Other Study ID Numbers: GS-LHON/CLIN/01
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: GenSight Biologics
• Original Responsible Party: Same as current
• Current Study Sponsor: GenSight Biologics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: GenSight Biologics
• Verification Date: June 2020