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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02064569
Recruitment Status : Active, not recruiting
First Posted : February 17, 2014
Last Update Posted : April 6, 2018
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics

February 13, 2014
February 17, 2014
April 6, 2018
February 2014
June 2020   (Final data collection date for primary outcome measure)
Incidence of local and general adverse events and Serious Adverse Events [ Time Frame: Up to 48 weeks ]
Same as current
Complete list of historical versions of study NCT02064569 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
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.
Not Provided
Interventional
Phase 1
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Leber Hereditary Optic Neuropathy
Genetic: GS010
Experimental: GS010
Intervention: Genetic: GS010
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
21
18
June 2020
June 2020   (Final data collection date for primary outcome measure)

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

Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
France
 
 
NCT02064569
GS-LHON/CLIN/01
Yes
Not Provided
Plan to Share IPD: Undecided
GenSight Biologics
GenSight Biologics
Not Provided
Principal Investigator: CATHERINE VIGNAL, MD CIC CHNO DES QUINZE VINGTS
GenSight Biologics
February 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP