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EAP_GS010_single Patient

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03672968
Expanded Access Status : Available
First Posted : September 17, 2018
Last Update Posted : August 9, 2021
Information provided by (Responsible Party):
GenSight Biologics

Tracking Information
First Submitted Date September 13, 2018
First Posted Date September 17, 2018
Last Update Posted Date August 9, 2021
Descriptive Information
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
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
Detailed Description Not Provided
Study Type Expanded Access
Expanded Access Type Individual Patients
Condition Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
Intervention Genetic: GS010
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
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.
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Contact: Magali Taiel, MD +33.1762172 26 mtaiel@gensight-biologics.com
Contact: Michel Roux, MD +33.69464783 mroux@gensight-biologics.com
Listed Location Countries Not Provided
Removed Location Countries  
Administrative Information
NCT Number NCT03672968
Other Study ID Numbers EAP_GS010_001
Current Responsible Party GenSight Biologics
Original Responsible Party Same as current
Current Study Sponsor GenSight Biologics
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators Not Provided
PRS Account GenSight Biologics
Verification Date August 2021