Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)NCT03672968
EAP_GS010_single Patient
A study testing GS010 in people with Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber), run by GenSight Biologics.
Trial summary
Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
- What is being tested?
- GS010
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Presence of documented G11778A ND4 LHON-causing mutation
- Signature of informed consent and assent from the parent/guardian and the patient.
Who cannot join
- Contraindications to GS010 product or IVT procedures are to be checked prior to consent signature and treatment injection:
- Any known allergy or hypersensitivity to GS010 or its constituents.
- Contraindication to intravitreal injection in any eye.
- Intravitreal drug delivery to any eye within 30 days prior to the injection
- Previous vitrectomy in either eye.
- Narrow angle in any eye contra-indicating pupillary dilation.
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- Presence of disorders or diseases of the eye or adnexa, excluding LHON, which may interfere with visual or ocular assessments, including SD-OCT, during the study period.
- Presence of known/documented mutations, other than the G11778A ND4 LHON-causing mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system.
- Presence of systemic or ocular/vision diseases, disorders or pathologies, other than LHON, known to cause or be associated with vision loss, or whose associated treatment(s) or therapy(ies) is/are known to cause or be associated with vision loss.
- Presence of optic neuropathy from any cause except LHON.
- Presence of illness or disease that, in the opinion of the Investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the central nervous system, including Multiple Sclerosis (diagnosis of Multiple Sclerosis must be based on the 2010 Revisions to the McDonald Criteria [Polman 2011]).
- History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation.
- Previous treatment with ocular gene therapy in either eye.
- Subjects who have undergone ocular surgery of clinical relevance (per Investigator assessment) within 90 days prior to injection.
- Subjects who are unable to tolerate (e.g. the immune modulating regimen) or unable or unwilling to comply with all the protocol requirements.
About this study
Official title: EAP Single Patient: Safety of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected With G11778A ND4 Leber Hereditary Optic Neuropathy
Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy
- Being tested
- GS010
- Keywords
- Heredity Optic Atrophy, Leber Hereditary Optic Atrophy, Leber Hereditary Optic Neuropathy, LHON, Eye Diseases, Hereditary Eye Diseases, Inherited retinal dystrophies or degeneration, Inborn Genetic Disease, Gene Therapy, Intravitreal Injections, Mitochondrial Disease, AAV2 Vectors, Neurodegenerative Disease, Atrophy
- Medical subject headings
- Optic Atrophy, Hereditary, Leber, Atrophy, Eye Diseases, Eye Diseases, Hereditary, Genetic Diseases, Inborn, Mitochondrial Diseases, Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS010Genetic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- GenSight Biologics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03672968
- Sponsor's protocol ID
- EAP_GS010_001
- Status last verified
- Feb 2026
- First submitted
- 13 Sep 2018
- Last updated
- 2 Mar 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- EAP Single Patient: Safety of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected With G11778A ND4 Leber Hereditary Optic Neuropathy
- ClinicalTrials.gov ID
- NCT03672968
- Acronym / sponsor ID
- EAP_GS010_001
- Keywords
- Heredity Optic Atrophy, Leber Hereditary Optic Atrophy, Leber Hereditary Optic Neuropathy, LHON, Eye Diseases, Hereditary Eye Diseases, Inherited retinal dystrophies or degeneration, Inborn Genetic Disease
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- GenSight BiologicsIndustry (pharma, biotech, devices)
- First posted
- 17 Sep 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Expanded Access Use for a single patient of Bilateral Intravitreal Injection of GS010 in a Single Subject Affected with G11778A ND4 Leber Hereditary Optic Neuropathy
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Presence of documented G11778A ND4 LHON-causing mutation
- Signature of informed consent and assent from the parent/guardian and the patient.
Key exclusion criteria (15 listed)
- Contraindications to GS010 product or IVT procedures are to be checked prior to consent signature and treatment injection:
- Any known allergy or hypersensitivity to GS010 or its constituents.
- Contraindication to intravitreal injection in any eye.
- Intravitreal drug delivery to any eye within 30 days prior to the injection
- Previous vitrectomy in either eye.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GS010 | Genetic | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing GS010 in people with Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber), run by GenSight Biologics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Mar 2026.