Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
NCT ID: NCT03293524
Last Updated: 2025-08-26
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
98 participants
INTERVENTIONAL
2018-03-12
2024-07-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Treatment Arm 1
Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 1 will receive intravitreal GS010 in both eyes.
GS010
GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.
Treatment Arm 2
Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 2 will receive GS010 in one eye and placebo intravitreal injection in the other eye.
GS010
GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.
Placebo
The placebo is a BSS, sterile, apyrogenic solution and used for ocular surgery. The placebo will be administered via intravitreal injection in a volume of 90 μL.
Interventions
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GS010
GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.
Placebo
The placebo is a BSS, sterile, apyrogenic solution and used for ocular surgery. The placebo will be administered via intravitreal injection in a volume of 90 μL.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Contraindication to intravitreal injection in any eye.
* Subjects refusing to discontinue idebenone.
* Previous vitrectomy in either eye.
* Narrow angle in any eye contra-indicating pupillary dilation.
* Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system.
* History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.
* Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion Visit (Visit 2).
* Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as defined by the study's SOP for visual acuity testing.
* Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA.
Exclusion Criteria
* Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye.
* Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding nicotine).
15 Years
ALL
No
Sponsors
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GenSight Biologics
INDUSTRY
Responsible Party
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Principal Investigators
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Nancy Newman, MD
Role: PRINCIPAL_INVESTIGATOR
Emory University Hospital Atlanta, Georgia, United States, 30322
Locations
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Doheny Eye Center UCLA Pasadena
Pasadena, California, United States
University of Colorado Health Eye Center
Aurora, Colorado, United States
Emory Healthcare - The Emory Clinic
Atlanta, Georgia, United States
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Wills Eye Institute - Ocular Oncology Service
Philadelphia, Pennsylvania, United States
Vanderbilt Eye Institute
Nashville, Tennessee, United States
Universitair Ziekenhuis Gent
Ghent, , Belgium
CHNO Les Quinze Vingts
Paris, , France
IRCCS Istituto delle Scienze Neurologiche di Bologna UOC Clinica Neurologica
Bologna, , Italy
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Taipei Veterans General Hospital
Taipei, , Taiwan
Moorfields Eye Hospital
London, Greater London, United Kingdom
Countries
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References
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Carelli V, Newman NJ, Yu-Wai-Man P, Biousse V, Moster ML, Subramanian PS, Vignal-Clermont C, Wang AG, Donahue SP, Leroy BP, Sergott RC, Klopstock T, Sadun AA, Rebolleda Fernandez G, Chwalisz BK, Banik R, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA; the LHON Study Group. Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation. Ophthalmol Ther. 2023 Feb;12(1):401-429. doi: 10.1007/s40123-022-00611-x. Epub 2022 Nov 30.
Related Links
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GenSight Biologics website
Other Identifiers
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2017-002187-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
GS-LHON-CLIN-05
Identifier Type: -
Identifier Source: org_study_id
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