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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

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

NCT ID

NCT02064569

First Posted

February 13, 2014

Last Updated

June 30, 2020

Sponsor

GenSight Biologics

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

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

Learn more about this trial

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

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