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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-02-13

Study Completion Date

2020-06-25

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152

Leber Hereditary Optic Neuropathy (LHON)

TERMINATED PHASE1/PHASE2

Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

NCT02362438

Giant Axonal Neuropathy Gene Transfer

ACTIVE_NOT_RECRUITING PHASE1

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

NCT05901987

SMA II

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

NCT02550691

Spinal Muscular Atrophy

TERMINATED NA

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

NCT06191354

Spinal Muscular Atrophy 1

ACTIVE_NOT_RECRUITING NA

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Leber Hereditary Optic Neuropathy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

GS010

Group Type EXPERIMENTAL

GS010

Intervention Type GENETIC

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

GS010

Intervention Type GENETIC

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No