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EAP_GS010_single Patient

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ClinicalTrials.gov Identifier: NCT03672968
Expanded Access Status : Available
First Posted : September 17, 2018
Last Update Posted : February 25, 2019
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

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

Condition or disease Intervention/treatment
Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber) Genetic: GS010

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: EAP Single Patient: Safety of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected With G11778A ND4 Leber Hereditary Optic Neuropathy




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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.

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): NCT03672968


Contacts
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Contact: Barrett Katz, MD +16468313799 bkatz@gensight-biologics.com

Sponsors and Collaborators
GenSight Biologics

Additional Information:
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Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT03672968     History of Changes
Other Study ID Numbers: EAP_GS010_001
First Posted: September 17, 2018    Key Record Dates
Last Update Posted: February 25, 2019
Last Verified: September 2018

Keywords provided by GenSight Biologics:
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

Additional relevant MeSH terms:
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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