- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02064569
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
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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-
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Paris, France, 75012
- CIC du CHNO DES QUINZE-VINGTS
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: GS010
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Incidence of local and general adverse events and Serious Adverse Events
Time Frame: Up to 48 weeks
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Up to 48 weeks
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Collaborators and Investigators
Sponsor
Investigators
- Principal Investigator: CATHERINE VIGNAL, MD, CIC CHNO DES QUINZE VINGTS
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Nervous System Diseases
- Eye Diseases
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Eye Diseases, Hereditary
- Heredodegenerative Disorders, Nervous System
- Cranial Nerve Diseases
- Optic Atrophies, Hereditary
- Optic Atrophy
- Mitochondrial Diseases
- Optic Nerve Diseases
- Optic Atrophy, Hereditary, Leber
Other Study ID Numbers
- GS-LHON/CLIN/01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Leber Hereditary Optic Neuropathy
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GenSight BiologicsNo longer availableLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
-
Neurophth Therapeutics IncRecruitingLeber Hereditary Optic Neuropathy (LHON)China, United States
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University Hospital, AngersUnknownLeber Hereditary Optic NeuropathyFrance
-
Neurophth Therapeutics IncRecruitingLeber Hereditary Optic Neuropathy (LHON)United States
-
GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, United Kingdom, Germany
-
GenSight BiologicsActive, not recruitingLeber Hereditary Optic NeuropathyBelgium, Spain, United States, France, United Kingdom, Taiwan, Italy
-
GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, Spain, United Kingdom
-
Huazhong University of Science and TechnologyShiyan Taihe HospitalActive, not recruitingLeber Hereditary Optic NeuropathyChina
-
Santhera PharmaceuticalsEuropean Vision Institute Clinical Research NetworkCompletedLeber Hereditary Optic Neuropathy (LHON)Belgium, France, Denmark, Italy, Slovenia
-
Bin LiHuazhong University of Science and TechnologyCompletedLeber Hereditary Optic NeuropathyChina
Clinical Trials on GS010
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GenSight BiologicsNo longer availableLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
-
GenSight BiologicsActive, not recruitingLeber Hereditary Optic NeuropathyBelgium, Spain, United States, France, United Kingdom, Taiwan, Italy
-
GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, United Kingdom, Germany
-
GenSight BiologicsCompletedOptic, Atrophy, Hereditary, LeberUnited Kingdom, United States, Germany, Italy, France
-
GenSight BiologicsCompletedOptic, Atrophy, Hereditary, LeberUnited Kingdom, United States, France, Germany, Italy