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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002187-40 | EudraCT Number |
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The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss up to one year.
The REFLECT study is a Phase 3, international, multi-center, randomized, double-masked, placebo-controlled, clinical trial. The primary objective is to assess the efficacy of intravitreal (IVT) of GS010 compared to IVT of placebo in second-affected/not-yet-affected eyes at 1.5 years post-treatment, by analyzing the change from baseline of the visual acuity in ND4 LHON subjects with vision loss up to one year.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GS010-GS010 | Experimental | Patients received single IVT injection of GS010 in both their first-affected eye and their second-affected/not-yet-affected eye at a droplet digital polymerase chain reaction (ddPCR) dose of 1.2/1.3E11 vg in 90 μL for each eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
|
| GS010-Placebo | Placebo Comparator | Patients received single IVT injection of GS010 in their first-affected eye and placebo IVT injection in their second-affected/not-yet-affected eye. Patients received IVT lenadogene nolparvovec in their first-affected eye (ddPCR dose of 1.2/1.3E11 vg in a volume of 90 μL) and placebo IVT injection (volume of 90 μL) in their second-affected/not-yet-affected eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GS010 | Genetic | GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 was administrated via intravitreal injection containing 1.2/1.3E11 vg in 90 μL balanced sterile saline solution (BSSS). |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes | The primary efficacy endpoint was the change from baseline of BCVA reported with LogMAR at 1.5-year post-treatment, in the second-affected/not-yet-affected eyes of ND4 LHON patients with vision loss up to one year. LogMAR BCVA was used to represent BCVA. | at 1.5 years post-treatment, in the second-affected/not-yet affected eyes |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of the BCVA Reported With LogMAR at 5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes | Change from baseline of BCVA reported with LogMAR at 5 years post-treatment, in the second-affected/not-yet-affected eyes of ND4 LHON patients with vision loss up to one year. LogMAR BCVA was used to represent BCVA. | at 5 years post-treatment, in the second-affected/not-yet affected eyes |
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Main Selection Criteria:
Main Non-Selection Criteria:
Main Inclusion Criteria:
Main Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Nancy Newman, MD | Emory University Hospital Atlanta, Georgia, United States, 30322 | Principal Investigator |
| Patrick Yu-Wai-Man, PhD | John van Geest Centre for Brain Repair, University of Cambridge, Cambridge, CB2 0PY, United Kingdom | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Doheny Eye Center UCLA Pasadena | Pasadena | California | 91105 | United States | ||
| University of Colorado Health Eye Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36449262 | Derived | Carelli V, Newman NJ, Yu-Wai-Man P, Biousse V, Moster ML, Subramanian PS, Vignal-Clermont C, Wang AG, Donahue SP, Leroy BP, Sergott RC, Klopstock T, Sadun AA, Rebolleda Fernandez G, Chwalisz BK, Banik R, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA; the LHON Study Group. Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation. Ophthalmol Ther. 2023 Feb;12(1):401-429. doi: 10.1007/s40123-022-00611-x. Epub 2022 Nov 30. |
| Label | URL |
|---|---|
| GenSight Biologics website | View source |
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108 patients with the G11778A mitochondrial point mutation in the ND4 gene and vision loss of up to 1 year in one or both eyes were screened. Screening failures (10) including patients who did not meet inclusion criteria or met exclusion criteria.
108 patients (≥ 15 years old) were screened for eligibility in 13 sites (Belgium, France, Italy, Spain, Taiwan, United Kingdom: 1 site each, and the United States: 7 sites). Of these, 98 were randomized: 48 to treatment Arm 1 (GS010-GS010) and 50 to treatment Arm 2 (GS010-placebo). The remaining 10 participants were screen failures.
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| ID | Title | Description |
|---|---|---|
| FG000 | GS010-GS010 | All study participants received single IVT injection of GS010 in their first-affected eye (droplet digital polymerase chain reaction [ddPCR] dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received a single IVT injection of GS010 in their second-affected/not-yet-affected eye at a ddPCR dose of 1.2/1.3E11 vg in 90 μL. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 23, 2019 | Feb 26, 2026 |
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Each participant had an eye designated as first-affected eye, and a fellow eye designated as either second-affected eye or not-yet-affected eye at the Screening Visit based on non-equal vision loss duration. In participants reporting simultaneous onset of vision loss in both eyes, the second-affected eye was randomly selected between both eyes. All participants received GS010 in their first-affected eye and were randomized in a 1:1 ratio to receive either GS010 (GS010- GS010 treatment arm) or placebo (GS010-Placebo treatment arm) in the second-affected/not-yet-affected eye.
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Study treatments were masked and the allocation to treatment groups was not known to the investigator or other persons involved in the conduct of the study, except the site pharmacies personnel to allow for preparation of investigational products before administration, in cases of emergencies and the data safety monitoring board (DSMB). To ensure that the double-masking design of the study was maintained, GS010 and placebo were identical in appearance and storage conditions.
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| Placebo | Drug | The placebo is a balanced sterile saline solution (BSSS) used for IVT. The placebo was administered via intravitreal injection in a volume of 90 μL. |
|
| Proportion of Patients Who Switched From Off-chart Eyes to On-chart Eyes at 5 Years Post-treatment | Proportion of patients with both eyes off-chart, defined as those patients unable to read letter on the ETDRS chart, who had at least one eye on-chart, defined as those patients able to read letters on the ETDRS chart (at either 4 meters or 1 meter) at 5 years | From baseline to 5 years post-treatment |
| Responder Analyses - Improvements From Nadir (Gainer Eyes) at 5 Years | Proportion of patients with an improvement of at least -0.3 LogMAR (≥ +15 ETDRS letters) from nadir to year 5 in at least one eye. Nadir was defined for each eye of each subject as the worst value observed from baseline to year 5 | From nadir to 5 years post-treatment |
| Responder Analyses - Clinically Relevant Recovery From Nadir at 5 Years | Proportion of patients with clinically relevant recovery (CRR) from nadir that was defined as patient with a CRR in at least one eye - Patient with at least one eye which was on chart at nadir, and which had an improvement of at least -0.2 LogMAR from nadir, or which was off-chart at nadir but became on-chart | From nadir to 5 years post-treatment |
| Responder Analyses- Clinically Relevant Benefit at 5 Years | Proportion of patients who had clinically relevant stabilization (CRS), defined as eyes with LogMAR BCVA < 1 at baseline and at 5 years post-treatment or had a clinically relevant recovery (CRR) from nadir. The best response observed in either eye was considered. | From baseline nadir to 5 years post-treatment |
| Quality of Life Questionnaire: VFQ-25 - Composite Score | Change from baseline to 5 years follow up in the Visual Function Questionnaire (VFQ-25) composite score. The VFQ-25 is a patient-reported outcome instrument assessing vision-related quality of life and consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs plus an additional single-item general health rating question. Each item was converted to a 0-100 scale for scoring, where 100 represents the best possible score on the measure and 0 represents the worst. Items within each subscale were averaged to create 12 subscale scores (11 subscales related to vision + 1 subscale related to general health). The vision-related subscale scores (excluding the general health rating question) were then averaged to calculate the composite score. | From baseline to 5 years post-treatment |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Emory Healthcare - The Emory Clinic | Atlanta | Georgia | 30322 | United States |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | United States |
| Department of Ophthalmology, Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| Vanderbilt Eye Institute | Nashville | Tennessee | 37232 | United States |
| Universitair Ziekenhuis Gent | Ghent | 9000 | Belgium |
| CHNO Les Quinze Vingts | Paris | Paris | 75012 | France |
| IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica | Bologna | 40139 | Italy |
| Hospital Universitario Ramon y Cajal | Madrid | 28034 | Spain |
| Taipei Veterans General Hospital | Taipei | 11217 | Taiwan |
| Moorfields Eye Hospital | London | Greater London | EC1V 2PD | United Kingdom |
| FG001 | GS010-Placebo | All study participants received single IVT injection of GS010 in their first-affected eye (droplet digital polymerase chain reaction [ddPCR] dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received placebo IVT injection (volume of 90 μL) in their second-affected/not-yet-affected eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
| COMPLETED |
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| NOT COMPLETED |
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The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized).
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| ID | Title | Description |
|---|---|---|
| BG000 | GS010-GS010 | All study participants received single IVT injection of GS010 in their first-affected eye (droplet digital polymerase chain reaction [ddPCR] dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received a single IVT injection of GS010 in their second-affected/not-yet-affected eye at a ddPCR dose of 1.2/1.3E11 vg in 90 μL. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
| BG001 | GS010-Placebo | All study participants received single IVT injection of GS010 in their first-affected eye (droplet digital polymerase chain reaction [ddPCR] dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received placebo IVT injection (volume of 90 μL) in their second-affected/not-yet-affected eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Age group at screening | Number | participants |
| |||||||||||||||
| Sex/Gender, Customized | Number | participants |
| ||||||||||||||||
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
| |||||||||||||||
| Region of Enrollment | Number | Participants |
| ||||||||||||||||
| Age at onset of the disease | Mean | Standard Deviation | years |
| |||||||||||||||
| Current alcohol use | Number | Participants |
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| Affected eye status | Number | Participants |
| ||||||||||||||||
| Durations of disease | Mean | Standard Deviation | Months |
| |||||||||||||||
| Time interval of vision loss between 1st and 2nd-affected eye | Mean | Standard Deviation | days |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes | The primary efficacy endpoint was the change from baseline of BCVA reported with LogMAR at 1.5-year post-treatment, in the second-affected/not-yet-affected eyes of ND4 LHON patients with vision loss up to one year. LogMAR BCVA was used to represent BCVA. | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized) on the primary eye (i.e., second-affected/not-yet-affected eye), unless otherwise specified. | Posted | Least Squares Mean | Standard Error | logMAR | at 1.5 years post-treatment, in the second-affected/not-yet affected eyes |
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| Secondary | Change From Baseline of the BCVA Reported With LogMAR at 5 Years Post-treatment, in the Second Affected/Not-yet Affected Eyes | Change from baseline of BCVA reported with LogMAR at 5 years post-treatment, in the second-affected/not-yet-affected eyes of ND4 LHON patients with vision loss up to one year. LogMAR BCVA was used to represent BCVA. | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). | Posted | Least Squares Mean | Standard Error | LogMAR | at 5 years post-treatment, in the second-affected/not-yet affected eyes |
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| Secondary | Proportion of Patients Who Switched From Off-chart Eyes to On-chart Eyes at 5 Years Post-treatment | Proportion of patients with both eyes off-chart, defined as those patients unable to read letter on the ETDRS chart, who had at least one eye on-chart, defined as those patients able to read letters on the ETDRS chart (at either 4 meters or 1 meter) at 5 years | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). | Posted | Number | Percentage of participants | From baseline to 5 years post-treatment |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Responder Analyses - Improvements From Nadir (Gainer Eyes) at 5 Years | Proportion of patients with an improvement of at least -0.3 LogMAR (≥ +15 ETDRS letters) from nadir to year 5 in at least one eye. Nadir was defined for each eye of each subject as the worst value observed from baseline to year 5 | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). | Posted | Number | percentage of participants | From nadir to 5 years post-treatment |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Responder Analyses - Clinically Relevant Recovery From Nadir at 5 Years | Proportion of patients with clinically relevant recovery (CRR) from nadir that was defined as patient with a CRR in at least one eye - Patient with at least one eye which was on chart at nadir, and which had an improvement of at least -0.2 LogMAR from nadir, or which was off-chart at nadir but became on-chart | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). | Posted | Number | percentage of participants | From nadir to 5 years post-treatment |
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| Secondary | Responder Analyses- Clinically Relevant Benefit at 5 Years | Proportion of patients who had clinically relevant stabilization (CRS), defined as eyes with LogMAR BCVA < 1 at baseline and at 5 years post-treatment or had a clinically relevant recovery (CRR) from nadir. The best response observed in either eye was considered. | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). | Posted | Number | percentage of participants | From baseline nadir to 5 years post-treatment |
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| Secondary | Quality of Life Questionnaire: VFQ-25 - Composite Score | Change from baseline to 5 years follow up in the Visual Function Questionnaire (VFQ-25) composite score. The VFQ-25 is a patient-reported outcome instrument assessing vision-related quality of life and consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs plus an additional single-item general health rating question. Each item was converted to a 0-100 scale for scoring, where 100 represents the best possible score on the measure and 0 represents the worst. Items within each subscale were averaged to create 12 subscale scores (11 subscales related to vision + 1 subscale related to general health). The vision-related subscale scores (excluding the general health rating question) were then averaged to calculate the composite score. | The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized). Participants in the ITT population without a Year 5 assessment were not included in the analysis. | Posted | Least Squares Mean | Standard Error | Points | From baseline to 5 years post-treatment |
|
From the time of the ICF was signed throughout the completion of the study follow-up (Year 5)
Patients from the safety population were classified according to the study treatment actually received, leading to 49 patients in each treatment arm (1 patient randomized to GS010-Placebo had received the study treatment for GS010-GS010). Systemic adverse events are reported per participant based on total treatment exposure. Ocular adverse events are reported per participant but identified by the specific eye of occurrence.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GS010-GS010 | All study participants received single IVT injection of GS010 in their first-affected eye (ddPCR dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received a single IVT injection of GS010 in their second-affected/not-yet-affected eye at a ddPCR dose of 1.2/1.3E11 vg in 90 μL. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). | 0 | 49 | 5 | 49 | 49 | 49 |
| EG001 | GS010-Placebo | All study participants received single IVT injection of GS010 in their first-affected eye (ddPCR dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received placebo IVT injection (volume of 90 μL) in their second-affected/not-yet-affected eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). | 2 | 49 | 6 | 49 | 49 | 49 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastritis (Systemic) | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Death (Systemic) | General disorders | MedDRA 25.0 | Systematic Assessment | General disorders and administration site conditions |
|
| Sudden cardiac death (Systemic) | General disorders | MedDRA 25.0 | Systematic Assessment | General disorders and administration site conditions |
|
| Appendicitis (Systemic) | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Clostridium difficile infection (Systemic) | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Subdural haemorrhage (Systemic) | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
| |
| Hypomagnesaemia (Systemic) | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Muscle spasms (Systemic) | Musculoskeletal and connective tissue disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Multiple sclerosis (Systemic) | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Psychotic disorder (Systemic) | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Acute respiratory failure (Systemic) | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival hyperaemia (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Conjunctival hyperaemia (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dry Eye (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Dry eye (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Eye pain (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Eye pain (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Intraocular pressure increased (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Intraocular pressure increased (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Iridocyclitis (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Iridocyclitis (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Iritis (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Iritis (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Keratic precipitates (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Keratic precipitates (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Punctate keratitis (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Punctate keratitis (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Uveitis (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Uveitis (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
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| Vitreous floaters (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Vitreous floaters (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Vitritis (First-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Vitritis (Second-affected/not-Yet-Affected Eye) | Eye disorders | MedDRA 25.0 | Systematic Assessment |
| |
| COVID-19 (Systemic) | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Influenza (Systemic) | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Nasopharyngitis (Systemic) | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Blood glucose increased (Systemic) | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Gamma glutamyltransferase increased (Systemic) | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Headache (Systemic) | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Anxiety (Systemic) | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Insomnia (Systemic) | Psychiatric disorders | MedDRA 25.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | GenSight Biologics | 33 176217233 | mtaiel@gensight-biologics.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 10, 2024 | Feb 26, 2026 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D029242 | Optic Atrophy, Hereditary, Leber |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D028361 | Mitochondrial Diseases |
| D015418 | Optic Atrophies, Hereditary |
| ID | Term |
|---|---|
| D009896 | Optic Atrophy |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
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| Between 18 and 60 years |
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| ≥ 60 years |
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| Male |
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| Europe |
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| United States |
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| > 1 to 2 drinks/day : |
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| > 2 drinks/day : |
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| No use : |
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| Missing : |
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| Bilateral |
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| OG001 | GS010-Placebo | All study participants received single IVT injection of GS010 in their first-affected eye (droplet digital polymerase chain reaction [ddPCR] dose of 1.2/1.3E11 vg in 90 μL). Participants randomized to this treatment arm received placebo IVT injection (volume of 90 μL) in their second-affected/not-yet-affected eye. Treatment could be performed either on a single day (1 IVT injection in each eye on Day 0) or on 2 consecutive days (1st IVT injection on Day -1 and 2nd IVT injection on Day 0). |
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