Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
- Conditions
- Leber Hereditary Optic Neuropathy
- Interventions
- Genetic: GS010
- Registration Number
- NCT02064569
- Lead Sponsor
- GenSight Biologics
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 19
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
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description GS010 GS010 -
- Primary Outcome Measures
Name Time Method Incidence of local and general adverse events and Serious Adverse Events Up to 48 weeks
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
CIC du CHNO DES QUINZE-VINGTS
🇫🇷Paris, France