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
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
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
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
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Primary Completion Date
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Study Completion Date
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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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EAP_GS010_single Patient
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