Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
62 participants
INTERVENTIONAL
2018-01-09
2022-07-04
Brief Summary
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Detailed Description
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Primary objectives: to assess the long term safety of intravitreal injection up to 5 years of post treatment in subjects who were treated in the RESCUE or REVERSE studies.
Secondary objectives: (1) to assess the long-term efficacy of intravitreal GS010 administration up to 5 years post-treatment in subjects who were treated in the RESCUE or REVERSE studies; and (2) to assess the quality of life (QoL) in subjects who were treated with GS010 in the RESCUE or REVERSE studies for up to 5 years post-treatment.
Methodology: This was a Phase III prospective long-term follow-up (LTFU) clinical study of subjects previously treated with GS010 and Sham during 2 Phase III studies-RESCUE and REVERSE. The LTFU study followed subjects for an additional 3 years, for a total of 5 years post-injection. The LTFU study included 5 visits at 2, 2.5, 3, 4, and 5 years after the investigational medicinal product (IMP) injection. Safety, efficacy, and QoL variables were assessed during each of the 5 LTFU visits, and descriptive summaries and statistical testing were used for the analysis of the data.
This report presents the final analysis of results at Year 5 after treatment administration in RESCUE and REVERSE.
Number of analysed subjects: all subjects completing RECUE and REVERSE studies who provided consent for the RESTORE study: 62 subjects.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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GS010-treated Eyes
Lenadogene nolparvovec Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive a single injection of GS010 and the other eye will receive a sham injection. GS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.
GS010
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Sham-treated Eyes
Sham Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive GS010 and the other eye will receive a sham injection. Eyes receiving sham injection will undergo the same preparatory procedures as eyes receiving GS010 injection, including pupillary dilation, topical anti-infection and topical anesthetic procedures. Sham intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.
Sham Intravitreal Injection
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Interventions
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GS010
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Sham Intravitreal Injection
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Subject of legal consent age has provided informed consent; subjects that are not of legal consent age have undergone their country-approved clinical trial enrollment consent process
Exclusion Criteria
* Subject has any medical or psychological condition that, in the opinion of the Investigator, may compromise his or her safe participation in the study
* Subject is taking or intending to take idebenone during the long-term follow-up study period
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, California, United States
Emory University Hospital
Atlanta, Georgia, United States
Wills Eye Institute
Philadelphia, Pennsylvania, United States
CHNO Les Quinze Vingts
Paris, , France
LMU Klinikum der Universität München / Friedrich-Baur-Institut
Munich, , Germany
Ospedale Bellaria
Bologna, , Italy
Moorfields Eye Hospital
London, Greater London, United Kingdom
Countries
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References
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Yu-Wai-Man P, Newman NJ, Biousse V, Carelli V, Moster ML, Vignal-Clermont C, Klopstock T, Sadun AA, Sergott RC, Hage R, Degli Esposti S, La Morgia C, Priglinger C, Karanja R, Taiel M, Sahel JA; LHON Study Group. Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. JAMA Ophthalmol. 2025 Feb 1;143(2):99-108. doi: 10.1001/jamaophthalmol.2024.5375.
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.
Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, 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. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2017-002153-11
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
NCT02652767 RESCUE
Identifier Type: OTHER
Identifier Source: secondary_id
NCT02652780 REVERSE
Identifier Type: OTHER
Identifier Source: secondary_id
GS-LHON-CLIN-06
Identifier Type: -
Identifier Source: org_study_id
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