NCT02064569

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Feb 2014

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 13, 2014

Completed
Same day until next milestone

Study Start

First participant enrolled

February 13, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 17, 2014

Completed
6.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 25, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 25, 2020

Completed
Last Updated

March 2, 2026

Status Verified

February 1, 2026

Enrollment Period

6.4 years

First QC Date

February 13, 2014

Last Update Submit

February 27, 2026

Conditions

Leber Hereditary Optic Neuropathy

Outcome Measures

Primary Outcomes (1)

  • Incidence of local and general adverse events and Serious Adverse Events

    Up to 48 weeks

Study Arms (1)

GS010

EXPERIMENTAL
Genetic: GS010

Interventions

GS010GENETIC
GS010

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CIC du CHNO DES QUINZE-VINGTS

Paris, 75012, France

Location

Related Links

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Condition Hierarchy (Ancestors)

Optic Atrophies, HereditaryOptic AtrophyOptic Nerve DiseasesCranial Nerve DiseasesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesEye Diseases, HereditaryEye DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • CATHERINE J. VIGNAL, MD

    CIC CHNO DES QUINZE VINGTS

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 13, 2014

First Posted

February 17, 2014

Study Start

February 13, 2014

Primary Completion

June 25, 2020

Study Completion

June 25, 2020

Last Updated

March 2, 2026

Record last verified: 2026-02

Locations

FR(1)