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Trial record 1 of 1 for:    LHON long term follow up
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RESCUE and REVERSE Long-term Follow-up (RESCUE/REVERSE)

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ClinicalTrials.gov Identifier: NCT03406104
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : April 4, 2018
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

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
Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber) Genetic: GS010 Other: Sham Intravitreal Injection

Study Type : Observational
Estimated Enrollment : 74 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Actual Study Start Date : January 9, 2018
Estimated Primary Completion Date : July 2022
Estimated Study Completion Date : August 2022



Intervention Details:
  • Genetic: GS010
    Different procedures will be performed in order to assess the safety and efficacy of GS010 up to 5 years post-treatment
  • Other: Sham Intravitreal Injection
    Different procedures will be performed in order to assess the safety and efficacy of GS010 up to 5 years post-treatment


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


Biospecimen Retention:   Samples Without DNA
Hematology, Serum chemistry and liver function tests


Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
The study population will include subjects who were treated with GS010 in either of the 2 Phase III trials—RESCUE and REVERSE
Criteria

Inclusion Criteria Subjects must meet all the following criteria at Inclusion period to be eligible for inclusion.

  • 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 Subjects meeting any of the following criteria at Inclusion period will be excluded from eligibility.

  • 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

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


Contacts
Contact: Barrett Katz, MD +1-646-831-3799 bkatz@gensight-biologics.com
Contact: Serge Fitoussi, MD +33 (0)6 65 75 78 41 sfitoussi@gensight-biologics.com

Locations
United States, California
Doheny Eye Center UCLA Recruiting
Pasadena, California, United States, 91105
Contact: Rosio Mendoza       r.mendoza@jsei.ucla.edu   
Principal Investigator: Alfredo Sadun, MD         
United States, Georgia
Emory University Hospital Recruiting
Atlanta, Georgia, United States, 30322
Contact: Lindy Dubois       ldubois@emory.edu   
Principal Investigator: Nancy Newman, MD         
United States, Pennsylvania
Wills Eye Institute Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Maria Massini       mmassini@willseye.org   
Principal Investigator: Mark Moster, MD         
France
CHNO Les Quinze Vingts Recruiting
Paris, France, 75012
Contact: Lise Plaine       lplaine@15-20.fr   
Principal Investigator: Catherine Vignal-Clermont, MD         
Germany
LMU Klinikum der Universität München / Friedrich-Baur-Institut Not yet recruiting
Munich, Germany, 80336
Contact: Claudia Catarino, MD       Claudia.catarino@med.uni-muenchen.de   
Principal Investigator: Thomas Klopstock, MD         
Italy
Ospedale Bellaria Not yet recruiting
Bologna, Italy, 40139
Contact: Chiara La Morgia, MD       chiaralamorgia@gmail.com   
Principal Investigator: Valerio Carelli, MD         
United Kingdom
Moorfields Eye Hospital Not yet recruiting
London, Greater London, United Kingdom, EC1V 2PD
Contact: Lauren Leitch-Devlin       lauren.leitch-devlin@moorfields.nhs.uk   
Principal Investigator: Patrick Yu Wai Man, MD         
Sponsors and Collaborators
GenSight Biologics

Additional Information:
Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT03406104     History of Changes
Other Study ID Numbers: GS-LHON-CLIN-06
2017-002153-11 ( EudraCT Number )
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: April 4, 2018
Last Verified: January 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by GenSight Biologics:
Leber Hereditary Optic Atrophy
Leber Hereditary Optic Neuropathy
LHON
Heredity Optic Atrophy
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:
Atrophy
Optic Nerve Diseases
Optic Neuritis
Optic Atrophy, Hereditary, Leber
Pathological Conditions, Anatomical
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