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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02064569
Recruitment Status : Completed
First Posted : February 17, 2014
Last Update Posted : July 1, 2020
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
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 : February 13, 2014
Actual Primary Completion Date : June 25, 2020
Actual Study Completion Date : June 25, 2020

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Arms and Interventions
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Arm Intervention/treatment
Experimental: GS010 Genetic: GS010


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of local and general adverse events and Serious Adverse Events [ Time Frame: Up to 48 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02064569


Locations
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France
CIC du CHNO DES QUINZE-VINGTS
Paris, France, 75012
Sponsors and Collaborators
GenSight Biologics
Investigators
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Principal Investigator: CATHERINE VIGNAL, MD CIC CHNO DES QUINZE VINGTS
More Information
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Additional Information:

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Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT02064569    
Other Study ID Numbers: GS-LHON/CLIN/01
First Posted: February 17, 2014    Key Record Dates
Last Update Posted: July 1, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
 Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases