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

Primary Purpose

Leber Hereditary Optic Neuropathy

Status
Completed
Phase
Phase 1
Locations
France
Study Type
Interventional
Intervention
GS010
Sponsored by
GenSight Biologics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leber Hereditary Optic Neuropathy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • CIC du CHNO DES QUINZE-VINGTS

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

GS010

Arm Description

Outcomes

Primary Outcome Measures

Incidence of local and general adverse events and Serious Adverse Events

Secondary Outcome Measures

Full Information

First Posted
February 13, 2014
Last Updated
June 30, 2020
Sponsor
GenSight Biologics
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1. Study Identification

Unique Protocol Identification Number
NCT02064569
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2020
Overall Recruitment Status
Completed
Study Start Date
February 13, 2014 (Actual)
Primary Completion Date
June 25, 2020 (Actual)
Study Completion Date
June 25, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GenSight Biologics

4. Oversight

Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber Hereditary Optic Neuropathy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GS010
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
GS010
Primary Outcome Measure Information:
Title
Incidence of local and general adverse events and Serious Adverse Events
Time Frame
Up to 48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
CATHERINE VIGNAL, MD
Organizational Affiliation
CIC CHNO DES QUINZE VINGTS
Official's Role
Principal Investigator
Facility Information:
Facility Name
CIC du CHNO DES QUINZE-VINGTS
City
Paris
ZIP/Postal Code
75012
Country
France

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Links:
URL
http://www.gensight-biologics.com/
Description
Sponsor web site

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

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