A Phase 3 interventional study of GS010 and Sham Intravitreal Injection in Leber Hereditary Optic Neuropathy, sponsored by GenSight Biologics. Completed at 7 sites in 5 countries. Open to participants aged 15 Years and older. Per ClinicalTrials.gov, last updated 2026-03-02.
Sponsored by GenSight Biologics · Phase 3, Interventional, and Treatment
The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.
Seven investigational centers from the RESCUE and REVERSE studies also participated in the RESTORE study, located in the European union and in the USA.
Primary objectives: to assess the long term safety of intravitreal injection up to 5 years of post treatment in subjects who were treated in the RESCUE or REVERSE studies.
Secondary objectives: (1) to assess the long-term efficacy of intravitreal GS010 administration up to 5 years post-treatment in subjects who were treated in the RESCUE or REVERSE studies; and (2) to assess the quality of life (QoL) in subjects who were treated with GS010 in the RESCUE or REVERSE studies for up to 5 years post-treatment.
Methodology: This was a Phase III prospective long-term follow-up (LTFU) clinical study of subjects previously treated with GS010 and Sham during 2 Phase III studies-RESCUE and REVERSE. The LTFU study followed subjects for an additional 3 years, for a total of 5 years post-injection. The LTFU study included 5 visits at 2, 2.5, 3, 4, and 5 years after the investigational medicinal product (IMP) injection. Safety, efficacy, and QoL variables were assessed during each of the 5 LTFU visits, and descriptive summaries and statistical testing were used for the analysis of the data.
This report presents the final analysis of results at Year 5 after treatment administration in RESCUE and REVERSE.
Number of analysed subjects: all subjects completing RECUE and REVERSE studies who provided consent for the RESTORE study: 62 subjects.
44 studies on the registry are indexed under Optic Atrophy, Hereditary, Leber; 9 are open to participants now.
This study's enrollment of 62 is above the median of 36 across 31 interventional studies indexed under Optic Atrophy, Hereditary, Leber.
Browse Optic Atrophy, Hereditary, Leber studies →GenSight Biologics is the lead sponsor of 9 studies on the registry; 1 is open to participants now.
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Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Lenadogene nolparvovec Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive a single injection of GS010 and the other eye will receive a sham injection. GS010-treated Eyes: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). Participants will receive a single dose of GS010 in one of their randomly selected eyes, via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) plus 0.001% Pluronic F68®.
Genetic: GS010
Sham Intravitreal ocular unilateral Injection Each participant will have one eye randomly selected to receive GS010 and the other eye will receive a sham injection. Eyes receiving sham injection will undergo the same preparatory procedures as eyes receiving GS010 injection, including pupillary dilation, topical anti-infection and topical anesthetic procedures. Sham intravitreal injection will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.
Other: Sham Intravitreal Injection
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Also known as: Lenadogene Nolparvovec
Lenadogene nolparvovec Intravitreal ocular unilateral Injection
Ocular Adverse Events (AEs)
Number of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment
Time frame: from year 2 to year 5 post treatment
Visual Acuity
Change from Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment, expressed in LogMAR. On chart visual acuity: Visual acuity inferior or equal to LogMAR +1.6 Off chart visual acuity: Visual acuity superior to LogMar +1.7 Normal vision LogMar: 0 and less than 0
Time frame: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment
Responder Analysis: Clinically Relevant Recovery
CRR clinically relevant recovery from Nadir defined as i/ for eyes on chart at Nadir, an improvement of at least -0.2 LogMar from Nadir and ii/ for eyes off chart at Nadir eyes that became on chart. Off chart visual acuity expressed in LogMar: more than +1.7 On chart visual acuity expressed in LogMar: less than 1.6 Normal vision LogMar: 0 and less than 0
Time frame: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment
Eyes on Chart
Definition: Visual acuity inferior or equal to LogMAR +1.6 at 5 Years post treatment
Time frame: Year 5 post-treatment
| Milestone | All Patient Treated in RECUE and REVERSE Studies |
|---|---|
| Started | 62 |
| Completed | 55 |
| Not completed | 7 |
| Withdrew: Lost to follow-up | 2 |
| Withdrew: Withdrawal by subject | 2 |
| Withdrew: Due to covid patient did not want to travel | 1 |
| Withdrew: Death | 2 |
Number of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment
| eyes | GS010 Treated Eyes | Sham Treated Eyes |
|---|---|---|
| Ocular Adverse Events (AEs) | 5 | 1 |
Change from Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment, expressed in LogMAR. On chart visual acuity: Visual acuity inferior or equal to LogMAR +1.6 Off chart visual acuity: Visual acuity superior to LogMar +1.7 Normal vision LogMar: 0 and less than 0
| LogMar | GS010 Treated Eyes | Sham Treated Eyes |
|---|---|---|
| Visual Acuity | -0.44 ± 0.46 | -0.39 ± 0.36 |
CRR clinically relevant recovery from Nadir defined as i/ for eyes on chart at Nadir, an improvement of at least -0.2 LogMar from Nadir and ii/ for eyes off chart at Nadir eyes that became on chart. Off chart visual acuity expressed in LogMar: more than +1.7 On chart visual acuity expressed in LogMar: less than 1.6 Normal vision LogMar: 0 and less than 0
| percentage of eye | GS010 Treated Eyes | Sham Treated Eyes |
|---|---|---|
| Responder Analysis: Clinically Relevant Recovery | 60 | 66 |
Definition: Visual acuity inferior or equal to LogMAR +1.6 at 5 Years post treatment
| percentage of eyes | GS010-treated Eyes | Sham-treated Eyes |
|---|---|---|
| Eyes on Chart | 76 | 79 |
Collected over from 2 to 5 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Subjects | 2/62 (3.2%) | 8/62 (12.9%) | 19/62 (30.6%) |
| Event | Subjects |
|---|---|
| hip fractureInjury, poisoning and procedural complications | 2/62 |
| humerus fractureMusculoskeletal and connective tissue disorders | 1/62 |
| cardiac arrestCardiac disorders | 1/62 |
| ileusGastrointestinal disorders | 1/62 |
| lower limb fractureMusculoskeletal and connective tissue disorders | 1/62 |
| limb injuryInjury, poisoning and procedural complications | 1/62 |
| cerebral haemorrageNervous system disorders | 1/62 |
| glioblastoma multiformeNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/62 |
| appenditis perforatedInfections and infestations | 1/62 |
| pneumoniaInfections and infestations | 1/62 |
| Event | Subjects |
|---|---|
| CataractEye disorders | 6/62 |
| Gamma-Glutamyltransferase IncreasedInvestigations | 5/62 |
| Intraocular Pressure IncreasedEye disorders | 4/62 |
| Covid-19Infections and infestations | 4/62 |
| Age, Categorical(Participants) | Lenadogene Nolparvovec and Sham All Participants |
|---|---|
| <=18 years | 7 |
| Between 18 and 65 years | 55 |
| >=65 years | 0 |
| Age, Continuous(years) | Lenadogene Nolparvovec and Sham All Participants |
|---|---|
| Mean | 35.9 ± 15.3 |
| Sex: Female, Male(Participants) | Lenadogene Nolparvovec and Sham All Participants |
|---|---|
| Female | 13 |
| Male | 49 |
| Race and Ethnicity Not Collected(Participants) | Lenadogene Nolparvovec and Sham All Participants |
|---|
| Region of Enrollment(participants) | Lenadogene Nolparvovec and Sham All Participants |
|---|---|
| United States | 31 |
| Italy | 1 |
| United Kingdom | 5 |
| France | 16 |
| Germany | 9 |
| Best Corrected Visual Acuity(LogMAR) | Lenadogene Nolparvovec and Sham All Participants |
|---|---|
| Mean | 1.40 ± 0.50 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2018 to primary completion in Jul 2022, 53 months.
Results were first posted in Jan 2024, 1 year 6 months after primary completion.
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This study is completed, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
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