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

Leber Hereditary Optic Neuropathy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02064569
• Other study ID #: GS-LHON/CLIN/01
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: February 13, 2014
• Est. completion date: June 25, 2020

Study information

• Verified date: June 2020
• Source: GenSight Biologics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: June 25, 2020
• Est. primary completion date: June 25, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

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

Related Conditions & MeSH terms

• Leber Hereditary Optic Neuropathy
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases

Intervention

Genetic:
• GS010

Locations

Country	Name	City	State
France	CIC du CHNO DES QUINZE-VINGTS	Paris

Sponsors (1)

Lead Sponsor	Collaborator
GenSight Biologics

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of local and general adverse events and Serious Adverse Events		Up to 48 weeks

External Links

•   Sponsor web site