An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
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