A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene

GenSight Biologics7 sites in 5 countries39 target enrollmentFebruary 23, 2016

ConditionsOptic, Atrophy, Hereditary, Leber

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Optic, Atrophy, Hereditary, Leber
Sponsor: GenSight Biologics
Enrollment: 39
Locations: 7
Primary Endpoint: Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48
Status: Completed
Last Updated: 3 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.

Registry

clinicaltrials.gov

Start Date

February 23, 2016

End Date

July 4, 2019

Last Updated

3 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

GenSight Biologics

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Selection Criteria:
•Participants must meet all the following criteria at the Screening Visit (Visit 1) in order to be included into the study.
•Age 15 years or older.
•Onset of vision loss based on medically documented history or participant testimony, in at least one eye for ≤180 days in duration and if both eyes are affected the duration of vision loss in both eyes must be ≤180 days in duration.
•Each eye of the participant maintaining visual ability to allow at least for counting of the examiner's fingers at any distance.
•Female participants (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after intravitreal (IVT) injection and male participants must agree to use condoms for up to 6 months after IVT injection.
•Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.
•Signed written informed consent.
•Inclusion Criteria:
•Participants included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).

Exclusion Criteria

•Non-Selection Criteria:
•Participants who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study.
•Any known allergy or hypersensitivity to GS010 or its constituents.
•Contraindication to IVT injection.
•IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1).
•Previous vitrectomy in either eye.
•Narrow angle in either eye contra-indicating pupillary dilation.
•Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period.
•Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.
•Causes of optic neuropathy other than LHON and glaucoma.

Outcomes

Primary Outcomes

Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48

Time Frame: Baseline and Week 48

Visual acuity was derived from the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Visual acuity is measured in "logarithm of the minimal angle of resolution" (LogMAR), which was derived from the number of letters participants could read on the ETDRS chart. 1 ETDRS line = 5 letters 1 ETDRS line = 0.1 LogMAR A lower LogMAR score denotes better visual acuity. A positive change from baseline indicates a worsening in symptoms. Change = (Week 48 score - Baseline score).

Secondary Outcomes

Change From Baseline in GCL Macular Volume(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in ETDRS Visual Acuity (Quantitative Score)(Baseline; Week 72 and Week 96)
Number of Subject Responders to Treatment(Week 48; Week 72 and Week 96)
Visual Field Mean Deviation in Decibels of Sensitivity Obtained With HVF Analyzer II(Baseline; Week 48; Week 72 and Week 96)
Number of Eye Responders to Treatment(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in RNFL Temporal Quadrant Thickness(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in Color Vision(Baseline; Week 48 and Week 96)
Change From Baseline in RNFL Papillomacular Bundle Thickness(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in ETDRS Total Macular Volume(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in the Foveal Threshold Sensitivities Obtained With HVF Analyzer II(Baseline; Week 48; Week 72 and Week 96)
Change From Baseline in Contrast Sensitivity(Baseline; Week 48; Week 72 and Week 96)