Leber Hereditary Optic DiseasePhase 2REVISENCT07303296
Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients
A Phase 2 study testing GS010 High dose and GS010 Low dose in people with Leber Hereditary Optic Disease, run by GenSight Biologics at 1 site in France.
Trial summary
The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON) Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Leber Hereditary Optic Disease
- What is being tested?
- GS010 High dose and GS010 Low dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteFrance
- Study dates
- 22 Jan 2026to Jun 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 15 years or older at the time of vision loss onset
- Clinically manifested vision loss due to ND4 LHON in both eyes
- BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes
- Documented results of genotyping showing the presence of pathogenic LHON-associated mutation(s) in the ND4 mitochondrial gene and the absence of other pathogenic LHON-associated mutations in the patient's mitochondrial DNA; absence of pathogenic mutations, other than the ND4 LHON-causing mutation(s), which are known to cause pathology of the optic nerve, retina or afferent visual system. Historic results of genetic analysis are acceptable upon Sponsor approval.
- Vision loss duration from 6 months to 1.5 years in the first affected eye at the Inclusion visit (Day 1)
- No limitations to OCT image collection that would prevent high quality, reliable images from being obtained in both eyes, as determined by the reading center
Show all 11Show fewer
- Clear ocular media and adequate pupillary dilation to permit thorough ocular examination, as assessed by the Investigator
- Human immunodeficiency virus (HIV) negative serology
- Female patients of childbearing potential must agree to use effective methods of birth control for up to 6 months after Treatment visit (Day 0). Male patients must agree to use condoms with their female partners for up to 6 months after Treatment visit (Day 0).
- Willing and able to comply with the protocol, follow study instructions, attend study visits as required and complete all study assessments
- Patient - and parent/legal guardian if the patient is under 18 years of age - has provided signed, written informed consent
Who cannot join
- 1. Any known allergy or hypersensitivity to GS010 or any of its constituents 2. Contraindication to IVT in any eye according to the Investigator's clinical judgment and international guidelines (Avery, 2014) 3. Prior intraocular surgery or procedure or IVT, which occurred 6 months prior to inclusion and is considered of clinical relevance per Investigator assessment, or planned intraocular surgery or procedure 4. Presence of optic neuropathy from any cause except LHON, as determined by the Investigator 5. History of recurrent uveitis (idiopathic or immune-related) or active intraocular inflammation, as determined by the Investigator 6. Indication of a unilateral treatment with GS010, as determined by the Investigator:
- Unilateral or asymmetric ND4 LHON disease: only one eye affected with a vision loss due to ND4 LHON, and the fellow eye with preserved BCVA (LogMAR 0 or close to 0);
- Amblyopia: unilateral treatment of the functional eye with a vision loss due to ND4 LHON;
- Patient's desire for unilateral treatment 7. Ingestion of idebenone less than 7 days prior to the Inclusion visit (Day -1) 8. Inability to tolerate the anti-inflammatory regimen 9. Presence of ocular disease (excluding LHON), or systemic disease, or condition (including medications and laboratory test abnormalities) that could compromise patient safety or interfere with assessment of efficacy and safety, as determined by the Investigator 10. Use of any investigational drug, or device, within 90 days, or 5 half-lives, preceding the Inclusion visit (Day-1), whichever is longer, or plans to participate in another study of an investigational drug or device during the study period 11. Previous treatment with ocular gene therapy in either eye. 12. Pregnancy or breast-feeding
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
GenSight Biologics contact
France1 site, 1 recruiting
- Recruiting
Hopital national des quinze-vingts
Paris, 75012
Catherine Vignal-Clermont, Dr, cvignal@15-20.fr
catherine vignal, Dr
About this study
Official title: A Dose-ranging Randomized, Open-label Study Evaluating the Effect of Bilateral Intravitreal Injection of GS010 at Two Dose Levels on Visual Acuity and Retinal Mitochondrial Activity in Patients Affected With ND4 Leber Hereditary Optic Neuropathy - The REVISE Study
The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON)
- Conditions
- Leber Hereditary Optic Disease
- Being tested
- GS010 High dose, GS010 Low dose
- Keywords
- Heredity Optic atrophy, leber hereditary optic atrophy, LHON, Eye diseases, gene therapy, AAV2 vectors, Inborn genetic disease, Mitochondrial disease, nervous system disease, Intravitreal injection, neurodegenerative disease, leber disease
- Medical subject headings
- Optic Atrophy, Hereditary, Leber, Eye Diseases, Genetic Diseases, Inborn, Mitochondrial Diseases, Nervous System Diseases, Neurodegenerative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS010 High doseGenetic
Also called Lumevoq, GS010, lenadogene nolparvovec
For patients allocated dose H GS010 will be administered at a dose of 3.9E11 VG/eye in a final volume of 90 μL (ARM H).
GS010 Low doseGenetic
Also called GS010, Lenadogene nolparvovec, Lumevoq
For patients allocated dose L, GS010 will be administered at a dose of 1.3E11 VGVG/eye in a final volume of 30 μL (ARM L).
| Study group | Role | What they receive |
|---|---|---|
| GS010 High dose | Active comparator | GS010, at a dose of 3.9E11 VG/eye (High Dose) in ARM H Receives: Genetic: GS010 High dose |
| GS010 Low dose | Active comparator | GS010, at a dose of 1.3E11 VG/eye (Low Dose) in ARM L. Receives: Genetic: GS010 Low dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint will be the BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Measured: from baseline to 1.5 years post-treatment
Secondary outcomes (3)
BCVA responder rates from baseline to 1.5 years post-treatment in the study eyes, defined as an improvement of at least -0.2 LogMAR.
Measured: from baseline to 1.5 years post-treatment
BCVA change from baseline to 1.5 years post-treatment in both eyes.
Measured: from baseline to 1.5 years post-treatment
Difference between ARM H and ARM L in BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Measured: from baseline to 1.5 years post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2025
- Study start22 Jan 2026
- Primary completion15 May 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber Hereditary Optic DiseaseStudies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The objective of the Phase 2 study REVISE is to assess the efficacy, retinal mitochondrial activity and safety of two dose levels (high and low doses) of bilateral GS010 IVT measured by ddPCR in ND4 LHON patients, in order to determine the optimal dose for future development.
- Masking
- None (open label)
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GenSight Biologics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07303296
- Sponsor's protocol ID
- GS-LHON-CLIN-08
- Acronym
- REVISE
- Status last verified
- Feb 2026
- First submitted
- 11 Dec 2025
- Last updated
- 2 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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
- A Dose-ranging Randomized, Open-label Study Evaluating the Effect of Bilateral Intravitreal Injection of GS010 at Two Dose Levels on Visual Acuity and Retinal Mitochondrial Activity in Patients Affected With ND4 Leber Hereditary Optic Neuropathy - The REVISE Study
- ClinicalTrials.gov ID
- NCT07303296
- Acronym / sponsor ID
- REVISE / GS-LHON-CLIN-08
- Conditions
- Leber Hereditary Optic Disease
- Keywords
- Heredity Optic atrophy, leber hereditary optic atrophy, LHON, Eye diseases, gene therapy, AAV2 vectors, Inborn genetic disease, Mitochondrial disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- GenSight BiologicsIndustry (pharma, biotech, devices)
- Study start
- 22 Jan 2026 (actual)
- Primary completion
- 15 May 2028 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 24 Dec 2025 (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.
The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients affected with ND4 Leber Hereditary Optic Neuropathy (LHON)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The objective of the Phase 2 study REVISE is to assess the efficacy, retinal mitochondrial activity and safety of two dose levels (high and low doses) of bilateral GS010 IVT measured by ddPCR in ND4 LHON patients, in order to determine the optimal dose for future development.
| Arm | Type | Receives |
|---|---|---|
| GS010 High dose | Active comparator | Genetic: GS010 High doseGS010, at a dose of 3.9E11 VG/eye (High Dose) in ARM H |
| GS010 Low dose | Active comparator | Genetic: GS010 Low doseGS010, at a dose of 1.3E11 VG/eye (Low Dose) in ARM L. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Aged 15 years or older at the time of vision loss onset
- Clinically manifested vision loss due to ND4 LHON in both eyes
- BCVA of at least LogMAR +2.39, measured at the FrACT scale, in both eyes
- Documented results of genotyping showing the presence of pathogenic LHON-associated mutation(s) in the ND4 mitochondrial gene and the absence of other pathogenic LHON-associated mutations in the patient's mitochondrial DNA; absence of pathogenic mutations, other than the ND4 LHON-causing mutation(s), which are known to cause pathology of the optic nerve, retina or afferent visual system. Historic results of genetic analysis are acceptable upon Sponsor approval.
- Vision loss duration from 6 months to 1.5 years in the first affected eye at the Inclusion visit (Day 1)
Key exclusion criteria (4 listed)
- 1. Any known allergy or hypersensitivity to GS010 or any of its constituents 2. Contraindication to IVT in any eye according to the Investigator's clinical judgment and international guidelines (Avery, 2014) 3. Prior intraocular surgery or procedure or IVT, which occurred 6 months prior to inclusion and is considered of clinical relevance per Investigator assessment, or planned intraocular surgery or procedure 4. Presence of optic neuropathy from any cause except LHON, as determined by the Investigator 5. History of recurrent uveitis (idiopathic or immune-related) or active intraocular inflammation, as determined by the Investigator 6. Indication of a unilateral treatment with GS010, as determined by the Investigator:
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 High doseAlso: Lumevoq, GS010, lenadogene nolparvovecFor patients allocated dose H GS010 will be administered at a dose of 3.9E11 VG/eye in a final volume of 90 μL (ARM H). | Genetic | GS010 High dose |
| GS010 Low doseAlso: GS010, Lenadogene nolparvovec, LumevoqFor patients allocated dose L, GS010 will be administered at a dose of 1.3E11 VGVG/eye in a final volume of 30 μL (ARM L). | Genetic | GS010 Low dose |
Primary outcome
The primary endpoint will be the BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Time frame: from baseline to 1.5 years post-treatment
3 secondary outcomes
BCVA responder rates from baseline to 1.5 years post-treatment in the study eyes, defined as an improvement of at least -0.2 LogMAR.
Time frame: from baseline to 1.5 years post-treatment
BCVA change from baseline to 1.5 years post-treatment in both eyes.
Time frame: from baseline to 1.5 years post-treatment
Difference between ARM H and ARM L in BCVA change from baseline to 1.5 years post-treatment in the study eyes.
Time frame: from baseline to 1.5 years post-treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Mar 2026.
- GenSight Biologics websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Leber Hereditary Optic Disease
RecruitingPhase 1/2
Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 3
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Leber Congenital Amaurosis 10, Blindness
Recruiting
New Non-invasive Modalities for Assessing Retinal Structure and Function
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion
Active, not recruitingObservational study
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Inherited Retinal Dystrophy Due to RPE65 Mutations
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.
GenSight Biologics contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GS010 High dose and GS010 Low dose in people with Leber Hereditary Optic Disease, run by GenSight Biologics at 1 site in France.
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 11 inclusion and 4 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: looking for participants now, 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.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact GenSight Biologics (clinics@gensight-biologics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary endpoint will be the BCVA change from baseline to 1.5 years post-treatment in the study eyes. (time frame: from baseline to 1.5 years post-treatment). It is what the study was designed to measure, not a finding.
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.