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History of Changes for Study: NCT02064569
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
Latest version (submitted June 30, 2020) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 February 14, 2014 None (earliest Version on record)
2 February 24, 2015 Study Status and Study Design
3 November 17, 2015 Recruitment Status, Study Status and Contacts/Locations
4 April 22, 2016 Study Status
5 February 22, 2017 Study Status, IPDSharing and Study Design
6 April 5, 2018 Study Status
7 February 22, 2019 Study Status and Study Design
8 June 30, 2020 Recruitment Status and Study Status
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Study NCT02064569
Submitted Date:  February 14, 2014 (v1)

Open or close this module Study Identification
Unique Protocol ID: GS-LHON/CLIN/01
Brief Title: Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
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
Secondary IDs:
Open or close this module Study Status
Record Verification: February 2014
Overall Status: Recruiting
Study Start: February 2014
Primary Completion: December 2015 [Anticipated]
Study Completion:
First Submitted: February 13, 2014
First Submitted that
Met QC Criteria:
February 14, 2014
First Posted: February 17, 2014 [Estimate]
Last Update Submitted that
Met QC Criteria:
February 14, 2014
Last Update Posted: February 17, 2014 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: GenSight Biologics
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
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:
Open or close this module Conditions
Conditions: Leber Hereditary Optic Neuropathy
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Other
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 18 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: GS010 Genetic: GS010
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Incidence of local and general adverse events and Serious Adverse Events
[ Time Frame: Up to 48 weeks ]

Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

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

Open or close this module Contacts/Locations
Central Contact Person: Serge Fitoussi, MD
Telephone: +33 (6) 65 75 78 41
Email: sfitoussi@gensight-biologics.com
Study Officials: CATHERINE VIGNAL, MD
Principal Investigator
CIC CHNO DES QUINZE VINGTS
Locations: France
CIC du CHNO DES QUINZE-VINGTS
[Recruiting]
Paris, France, 75012
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links: Description: Sponsor web site
Available IPD/Information:

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