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RESCUE and REVERSE Long-term Follow-up (RESTORE)

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: NCT03406104
Recruitment Status : Completed
First Posted : January 23, 2018
Last Update Posted : July 27, 2022
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

Study Description
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Brief Summary:
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.

Condition or disease Intervention/treatment Phase
Leber Hereditary Optic Neuropathy Genetic: GS010 Other: Sham Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 61 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: intra patient comparaison
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials (RESTORE)
Actual Study Start Date : January 9, 2018
Actual Primary Completion Date : July 4, 2022
Actual Study Completion Date : July 4, 2022

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Arms and Interventions
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Arm Intervention/treatment
Experimental: GS010
Lenadogene nolparvovec Intravitreal occular unilateral Injection
 Genetic: GS010
Lenadogene nolparvovec Intravitreal occular unilateral Injection
Sham Comparator: Sham
Sham Intravitreal occular unilateral Injection
 Other: Sham
Lenadogene nolparvovec Intravitreal occular unilateral Injection


Outcome Measures
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Primary Outcome Measures :
  1. Adverse events (AEs) or serious adverse events (SAEs) (ocular or systemic) [ Time Frame: Up to 5-Year post-treatment ]
    AEs or SAEs (ocular or systemic) related to IMP or administration procedure, as judged by the Investigator, reported during the long-term follow-up visits (2, 2.5, 3, 4, and 5 years) from the period of 96 weeks up to 5 years post-treatment and summarized descriptively by type, frequency (number, percentage), severity, causal relationship, and seriousness


Secondary Outcome Measures :
  1. Best-Corrected Visual Acuity (BCVA) reported with LogMAR [ Time Frame: Up to 5-Year post-treatment ]
    Change in best BCVA reported with LogMAR

  2. HumphreyTM visual field (HVF) 30-2 parameters [ Time Frame: Up to 5-Year post-treatment ]
    Change of parameters measured with HVF 30-2

  3. Spectral domain optical coherence tomography (SD-OCT) parameters [ Time Frame: Up to 5-Year post-treatment ]
    Change of parameters measured with SD-OCT

  4. Responder Analysis [ Time Frame: Up to 5-Year post-treatment ]
    Response status of eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection (An improvement of at least 15 ETDRS letters/Eyes that lose less than the 15 ETDRS letters)

  5. Time course of the response [ Time Frame: Up to 5-Year post-treatment ]
    Time course of the response in eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, for the BCVA reported with LogMAR, for parameters measured with HVF 30-2 and SD-OCT

  6. Visual improvement [ Time Frame: Up to 5-Year post-treatment ]
    Visual improvement as measured by LogMAR by analysis of covariance (ANCOVA)

  7. Change of ganglion cell layer (GCL) thickness/volume and topographical map and other parameters measured by SD-OCT [ Time Frame: Up to 5-Year post-treatment ]
    Change of GCL thickness/volume and topographical map and other parameters measured by SD-OCT using a mixed model of ANCOVA

  8. Quality of Life: Visual Functioning Questionnaire 25 (VFQ-25) [ Time Frame: Up to 5-Year post-treatment ]
    QOL as measured with VFQ-25 subject-rated instrument

  9. Quality of Life: 36-Item Short Form Health Survey, version 2 (SF-36-v2) [ Time Frame: Up to 5-Year post-treatment ]
    QOL as measured with SF-36v2 subject-rated instrument


Eligibility Criteria
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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  • Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase III clinical studies
  • 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

Non-Inclusion Criteria

  • Subject is unwilling or unable to comply with the protocol requirements
  • 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
Contacts and Locations
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Information from the National Library of Medicine

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): NCT03406104


Locations
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United States, California
Doheny Eye Center UCLA
Pasadena, California, United States, 91105
United States, Georgia
Emory University Hospital
Atlanta, Georgia, United States, 30322
United States, Pennsylvania
Wills Eye Institute
Philadelphia, Pennsylvania, United States, 19107
France
CHNO Les Quinze Vingts
Paris, France, 75012
Germany
LMU Klinikum der Universität München / Friedrich-Baur-Institut
Munich, Germany, 80336
Italy
Ospedale Bellaria
Bologna, Italy, 40139
United Kingdom
Moorfields Eye Hospital
London, Greater London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
GenSight Biologics
Investigators
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Principal Investigator: Nancy Newman, MD Emory University Hospital Atlanta, Georgia, United States, 30322
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT03406104    
Other Study ID Numbers: GS-LHON-CLIN-06
2017-002153-11 ( EudraCT Number )
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: July 27, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by GenSight Biologics:
Heredity Optic Atrophy
Leber Hereditary Optic Atrophy
Leber Hereditary Optic Neuropathy
LHON
Eye Diseases
Hereditary Eye Diseases
Inborn Genetic Disease
 Gene Therapy
Intravitreal Injections
Mitochondrial Disease
AAV2 Vectors
Nervous System Diseases
Neurodegenerative Disease
Heredodegenerative Disorders of the Nervous System
Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
 Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases