Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

Plain-language summary

This study evaluated the safety and tolerability profile of ascending doses of GS010 in patients with Leber Hereditary Optic Neuropathy (LHON). The Phase 1/2 trial enrolled participants aged 18 or older with a documented diagnosis confirmed by genetic testing showing the G11778A mutation in the mitochondrial ND4 gene, and visual acuity of 1/10 or worse in the less functional eye.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

GenSight Biologics

Intervention

GS010

Start date

2/13/2014

Estimated completion

6/25/2020

Countries

France

Locations

• Paris, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4

Age 18 years old or older at the time of study entry (informed consent signature)

Visual acuity ≤ 1/10 of the less functional eye

Exclusion Criteria:

Any known allergy or hypersensibility to one of the product used during the trial

Contraindication to IVT surgery (anaemia Hb \<8g/dl, severe cardiovascular disease, severe coagulopathy…)

Disorder of the ocular humors and of the internal retina involving visual disability

Glaucoma

Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion

Narrow angle contra-indicating pupillary dilation

Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)

Patients presenting known mutation of other genes implicated in pathological retinal conditions