LHON Gene Therapy Clinical Trial Seeking Participants

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A gene therapy clinical trial for the treatment of Leber hereditary optic neuropathy (LHON) due to the ND4 mutation is currently recruiting participants. The trial is based in Moorfields Eye Hospital in London and open to participants from the Republic of Ireland.

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

Each participant will have one eye randomly selected to receive a single dose of GS010 via intravitreal injection. The other eye will receive a sham or placebo injection. The primary outcome measure is improvement in visual acuity measured 48 weeks after injection.

If you are interested in becoming a participant in this study, please contact your ophthalmologist.

Selection and inclusion criteria are outlined below. For more information about the clinical trial and full details of exclusion criteria, please visit https://clinicaltrials.gov/ct2/show/NCT02652767?term=GenSight+Biologics&rank=2

Selection Criteria:

Participants must meet all the following criteria at the Screening Visit (Visit 1) in order to be included into the study.

  1. Age 18 years or older.
  2. Onset of vision loss based on medically documented history or subject 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.
  3. Each eye of the subject maintaining visual ability to allow at least for counting of the examiner’s fingers at any distance.
  4. Female subjects (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after IVT injection and male subjects must agree to use condoms for up to 6 months after IVT injection.
  5. Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing.
  6. Signed written informed consent.

Inclusion Criteria:

Participants included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).

  1. Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject’s mitochondrial DNA.
  2. Review of all selection criteria to ensure continued compliance.
  3. Have a negative test for infection with human immunodeficiency virus (HIV).
  4. Have a negative pregnancy test for women of childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential).

Study title: Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

Lead investigator: Dr Patrick Yu-Wai-Man

Sponsor: GenSight Biologics