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EAP_GS010_single Patient

Primary Purpose

Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
GS010
Sponsored by
GenSight Biologics
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber) focused on measuring Heredity Optic Atrophy, Leber Hereditary Optic Atrophy, Leber Hereditary Optic Neuropathy, LHON, Eye Diseases, Hereditary Eye Diseases, Inherited retinal dystrophies or degeneration, Inborn Genetic Disease, Gene Therapy, Intravitreal Injections, Mitochondrial Disease, AAV2 Vectors, Neurodegenerative Disease, Atrophy

Eligibility Criteria

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The EAP applies to patients not eligible to ongoing GS010 clinical trials.

Inclusion Criteria:

  • Presence of documented G11778A ND4 LHON-causing mutation
  • Signature of informed consent and assent from the parent/guardian and the patient.

Exclusion Criteria:

Contraindications to GS010 product or IVT procedures are to be checked prior to consent signature and treatment injection:

  • Any known allergy or hypersensitivity to GS010 or its constituents.
  • Contraindication to intravitreal injection in any eye.
  • Intravitreal drug delivery to any eye within 30 days prior to the injection
  • Previous vitrectomy in either eye.
  • Narrow angle in any eye contra-indicating pupillary dilation.
  • Presence of disorders or diseases of the eye or adnexa, excluding LHON, which may interfere with visual or ocular assessments, including SD-OCT, during the study period.
  • 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.
  • Presence of systemic or ocular/vision diseases, disorders or pathologies, other than LHON, known to cause or be associated with vision loss, or whose associated treatment(s) or therapy(ies) is/are known to cause or be associated with vision loss.
  • Presence of optic neuropathy from any cause except LHON.
  • Presence of illness or disease that, in the opinion of the Investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system, including Multiple Sclerosis (diagnosis of Multiple Sclerosis must be based on the 2010 Revisions to the McDonald Criteria [Polman 2011]).
  • History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.
  • Previous treatment with ocular gene therapy in either eye.
  • Subjects who have undergone ocular surgery of clinical relevance (per Investigator assessment) within 90 days prior to injection.
  • Subjects who are unable to tolerate (e.g. the immune modulating regimen) or unable or unwilling to comply with all the protocol requirements.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    September 13, 2018
    Last Updated
    August 2, 2021
    Sponsor
    GenSight Biologics
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03672968
    Brief Title
    EAP_GS010_single Patient
    Official Title
    EAP Single Patient: Safety of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected With G11778A ND4 Leber Hereditary Optic Neuropathy
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2021
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    GenSight Biologics

    4. Oversight

    5. Study Description

    Brief Summary
    Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
    Keywords
    Heredity Optic Atrophy, Leber Hereditary Optic Atrophy, Leber Hereditary Optic Neuropathy, LHON, Eye Diseases, Hereditary Eye Diseases, Inherited retinal dystrophies or degeneration, Inborn Genetic Disease, Gene Therapy, Intravitreal Injections, Mitochondrial Disease, AAV2 Vectors, Neurodegenerative Disease, Atrophy

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Genetic
    Intervention Name(s)
    GS010

    10. Eligibility

    Sex
    All
    Eligibility Criteria
    The EAP applies to patients not eligible to ongoing GS010 clinical trials. Inclusion Criteria: Presence of documented G11778A ND4 LHON-causing mutation Signature of informed consent and assent from the parent/guardian and the patient. Exclusion Criteria: Contraindications to GS010 product or IVT procedures are to be checked prior to consent signature and treatment injection: Any known allergy or hypersensitivity to GS010 or its constituents. Contraindication to intravitreal injection in any eye. Intravitreal drug delivery to any eye within 30 days prior to the injection Previous vitrectomy in either eye. Narrow angle in any eye contra-indicating pupillary dilation. Presence of disorders or diseases of the eye or adnexa, excluding LHON, which may interfere with visual or ocular assessments, including SD-OCT, during the study period. 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. Presence of systemic or ocular/vision diseases, disorders or pathologies, other than LHON, known to cause or be associated with vision loss, or whose associated treatment(s) or therapy(ies) is/are known to cause or be associated with vision loss. Presence of optic neuropathy from any cause except LHON. Presence of illness or disease that, in the opinion of the Investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system, including Multiple Sclerosis (diagnosis of Multiple Sclerosis must be based on the 2010 Revisions to the McDonald Criteria [Polman 2011]). History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation. Previous treatment with ocular gene therapy in either eye. Subjects who have undergone ocular surgery of clinical relevance (per Investigator assessment) within 90 days prior to injection. Subjects who are unable to tolerate (e.g. the immune modulating regimen) or unable or unwilling to comply with all the protocol requirements.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Magali Taiel, MD
    Phone
    +33.1762172 26
    Email
    mtaiel@gensight-biologics.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    Michel Roux, MD
    Phone
    +33.69464783
    Email
    mroux@gensight-biologics.com

    12. IPD Sharing Statement

    Links:
    URL
    http://www.gensight-biologics.com
    Description
    Related Info

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