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    Clinical Trial Results:
    Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year

    Summary
    EudraCT number
    		 2017-002187-40
    Trial protocol
    		 BE   FR   GB   ES   NL   IT  
    Global end of trial date
    23 Jul 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    05 Mar 2026
    First version publication date
    05 Mar 2026
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GS-LHON-CLIN-05
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03293524
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GenSight Biologics
    Sponsor organisation address
    74 Rue de Faubourg Saint Antoine, Paris, France, 75012
    Public contact
    VP Regulatory Affairs , GenSight Biologics, 33 176217233, schekroun@gensight-biologics.com
    Scientific contact
    Chief Medical Officer, GenSight Biologics, 33 176217233, mtaiel@gensight-biologics.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    06 Nov 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jul 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Jul 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess the efficacy of intravitreal GS010 compared to placebo intravitreal injection in second affected/not yet affected eyes, at 1.5-Year post-treatment, utilizing the change from baseline of the best-corrected visual acuity (BCVA) reported with the Log of the Minimal Angle of Resolution (LogMAR), in ND4 LHON subjects with vision loss up to one year.
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles established in the Declaration of Helsinki (7th revision, 2013) with the principles of Good Clinical Practice (GCP) according to the International Council for Harmonization (ICH) guideline (ICH E6 [R2]), as well as with applicable regulatory requirements.
    Background therapy
    		 There were restrictions on the use of concomitant medications during the trial. The following medications were not permitted: IVT drug delivery to any eye within 30 days prior to the Screening Visit, and use of idebenone that was not completely discontinued at least 7 days prior to Visit 2 (Inclusion Visit).
    Evidence for comparator
    		 No gene therapy product had been approved for the treatment of LHON at the time of the study conduct. This study included a treatment arm (Treatment Arm 2) in which each subject received IVT lenadogene nolparvovec in their first-affected eye and a placebo IVT injection in their second-affected/not-yet-affected eye
    Actual start date of recruitment
    12 Mar 2018
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 5 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Belgium: 9
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Taiwan: 15
    Country: Number of subjects enrolled
    United States: 56
    Country: Number of subjects enrolled
    France: 4
    Worldwide total number of subjects
    98
    EEA total number of subjects
    19
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    10
    Adults (18-64 years)
    86
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 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 subjects were screen failures.

    Pre-assignment
    Screening details
    		 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

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst
    Blinding implementation details
    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.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 GS010-GS010
    Arm description
    		 Patients received single IVT injection of GS010 in both their first-affected eye and their second-affected/not-yet-affected eye
    Arm type
    		 Experimental

    Investigational medicinal product name
    GS010/GS010
    Investigational medicinal product code
    GS010/GS010
    Other name
    Lenadogene nolparvovec
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravitreal use
    Dosage and administration details
    Patients received IVT lenadogene nolparvovec in both eyes at a droplet digital polymerase chain reaction (ddPCR) dose of 1.2/1.3E11 vg in 90 microL 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)

    Arm title
    		 GS010-Placebo
    Arm description
    		 Patients received single IVT injection of GS010 in their first-affected eye and placebo IVT injection in their second-affected/not-yet-affected eye
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravitreal use
    Dosage and administration details
    Patients received IVT lenadogene nolparvovec in their first-affected eye (ddPCR dose of 1.2/1.3E11 vg in a volume of 90 microL) and placebo IVT injection (volume of 90 microL) in their second-affected/not-yet-affected eye

    Number of subjects in period 1
    		GS010-GS010 GS010-Placebo
    Started
    48
    50
    Completed
    40
    36
    Not completed
    8
    14
         Email by patient 9 months after final visit
    -
    1
         Death
    -
    2
         Non-compliant to visits
    -
    1
         Lost to follow-up
    4
    4
         Withdrawal by subject
    4
    6

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    GS010-GS010
    Reporting group description
    		 Patients received single IVT injection of GS010 in both their first-affected eye and their second-affected/not-yet-affected eye

    Reporting group title
    GS010-Placebo
    Reporting group description
    		 Patients received single IVT injection of GS010 in their first-affected eye and placebo IVT injection in their second-affected/not-yet-affected eye

    Reporting group values
    		 GS010-GS010 GS010-Placebo Total
    Number of subjects
    		 48 50 98
    Age categorical
    Units: Subjects
        Adolescents (12-17 years)
    		 3 7 10
        Adults (18-64 years)
    		 44 42 86
        From 65-84 years
    		 1 1 2
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 32.4 ( 14.4 ) 31.8 ( 13.4 ) -
    Gender categorical
    Units: Subjects
        Female
    		 11 9 20
        Male
    		 37 41 78
    Affected eye status
    Units: Subjects
        Unilateral
    		 1 0 1
        Bilateral
    		 47 50 97
    Current alcohol use
    Units: Subjects
        <=1 drink/day or occasionally
    		 24 27 51
        >1 to 2 drinks/day
    		 5 5 10
        >2 drinks/day
    		 2 4 6
        No use
    		 16 13 29
        Missing
    		 1 1 2
    Age at onset of the disease
    Units: years
        arithmetic mean (standard deviation)
    		 31.7 ( 14.4 ) 31.2 ( 13.4 ) -
    Time interval of vision loss between 1st and 2nd-affected eye
    Units: days
        arithmetic mean (standard deviation)
    		 56.9 ( 66.3 ) 61.9 ( 54.1 ) -
    Durations of disease
    Units: months
        arithmetic mean (standard deviation)
    		 8.3 ( 3.4 ) 8.3 ( 3.1 ) -

    End points

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    End points reporting groups
    Reporting group title
    GS010-GS010
    Reporting group description
    		 Patients received single IVT injection of GS010 in both their first-affected eye and their second-affected/not-yet-affected eye

    Reporting group title
    GS010-Placebo
    Reporting group description
    		 Patients received single IVT injection of GS010 in their first-affected eye and placebo IVT injection in their second-affected/not-yet-affected eye

    Subject analysis set title
    Intent-to-treat population
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    The intent-to-treat analysis (ITT) population consisted of all randomized subjects. The analyses were based on the planned treatment (as randomized).

    Subject analysis set title
    Safety analysis set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The safety population was defined as those patients who received study drug (GS010 or Placebo) in at least one eye. Patients were classified according to treatment actually received.

    Primary: Change from baseline of the Best Corrected Visual Acuity (BCVA) reported with LogMAR at 1.5 years post-treatment

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    End point title
    		 Change from baseline of the Best Corrected Visual Acuity (BCVA) reported with LogMAR at 1.5 years post-treatment
    End point description
    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.
    End point type
    Primary
    End point timeframe
    Change from baseline of the Best Corrected Visual Acuity (BCVA) reported with LogMAR at 1.5 years post-treatment, in the second-affected/not-yet affected eyes
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: log units
        least squares mean (standard error)
    -0.09 ( 0.072 )
    -0.04 ( 0.071 )
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-GS010 v GS010-Placebo
    Number of subjects included in analysis
    98
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.608
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -0.05
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.25
         upper limit
    		 0.15
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.101

    Secondary: Change from baseline of the BCVA reported with LogMAR at 5 years post-treatment

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    End point title
    		 Change from baseline of the BCVA reported with LogMAR at 5 years post-treatment
    End point description
    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.
    End point type
    Secondary
    End point timeframe
    Change from baseline of the Best Corrected Visual Acuity (BCVA) reported with LogMAR at 5 years post-treatment, in the second-affected/not-yet affected eyes
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: LogMAR
        arithmetic mean (standard deviation)
    -0.13 ( 0.080 )
    -0.05 ( 0.079 )
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-GS010 v GS010-Placebo
    Number of subjects included in analysis
    98
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5086
    Method
    		 ANCOVA
    Parameter type
    		 LS Mean Difference
    Point estimate
    -0.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.3
         upper limit
    		 0.15
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.112

    Secondary: Proportion of patients who switched from off-chart eyes to on-chart eyes at 5 years post-treatment

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    End point title
    		 Proportion of patients who switched from off-chart eyes to on-chart eyes at 5 years post-treatment
    End point description
    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
    End point type
    Secondary
    End point timeframe
    From baseline to 5 years post-treatment
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: Proportion of patients
        number (not applicable)
    61.54
    33.33
    No statistical analyses for this end point

    Secondary: Responder analyses - Improvements from nadir (gainer eyes) at 5 years

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    End point title
    		 Responder analyses - Improvements from nadir (gainer eyes) at 5 years
    End point description
    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
    End point type
    Secondary
    End point timeframe
    From nadir to 5 years post-treatment
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: Percentage of patients
        number (not applicable)
    68.8
    66.0
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-GS010 v GS010-Placebo
    Number of subjects included in analysis
    98
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7605
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.5
         upper limit
    		 2.7

    Secondary: Responder analyses - Clinically Relevant Recovery from nadir at 5 years

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    End point title
    		 Responder analyses - Clinically Relevant Recovery from nadir at 5 years
    End point description
    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
    End point type
    Secondary
    End point timeframe
    From nadir to 5 years post-treatment
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: Percentage of patients
        number (not applicable)
    75.0
    60.0
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-Placebo v GS010-GS010
    Number of subjects included in analysis
    98
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1352
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.9
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.8
         upper limit
    		 4.6

    Secondary: Responder analyses- Clinically Relevant Benefit at 5 years

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    End point title
    		 Responder analyses- Clinically Relevant Benefit at 5 years
    End point description
    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
    End point type
    Secondary
    End point timeframe
    From baseline to 5 years post-treatment
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    48
    50
    Units: Proportion of patients
        number (not applicable)
    75.0
    62.0
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-GS010 v GS010-Placebo
    Number of subjects included in analysis
    98
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1995
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.8
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.7
         upper limit
    		 4.3

    Secondary: Quality of life questionnaire: VFQ-25 - Composite score

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    End point title
    		 Quality of life questionnaire: VFQ-25 - Composite score
    End point description
    End point type
    Secondary
    End point timeframe
    Change from baseline to 5 years follow up in the Visual Function Questionnaire (VFQ-25) composite score
    End point values
    		 GS010-GS010 GS010-Placebo
    Number of subjects analysed
    39
    36
    Units: points
        least squares mean (standard error)
    11.8 ( 2.48 )
    15.0 ( 2.51 )
    Statistical analysis title
    		 Treatment contrast
    Comparison groups
    GS010-GS010 v GS010-Placebo
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3605
    Method
    		 Mixed models analysis
    Confidence interval

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the time of the ICF was signed throughout the completion of the study follow-up (Year 5)
    Adverse event reporting additional description
    Patients were expected to volunteer information about adverse events that they experienced. In addition, the investigator or designee questioned the patient at each visit about adverse events and recorded these as well as other adverse events at the visit
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    GS010-GS010
    Reporting group description
    		 Patients received single IVT injection of GS010 in both their first-affected eye and their second-affected/not-yet-affected eye. This population took into account the study treatment actually received by the patients

    Reporting group title
    GS010-Placebo
    Reporting group description
    		 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 in this group took into account the study treatment actually received by the patients

    Serious adverse events
    		 GS010-GS010 GS010-Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 49 (10.20%)
    6 / 49 (12.24%)
         number of deaths (all causes)
    0
    2
         number of deaths resulting from adverse events
    0
    0
    Injury, poisoning and procedural complications
    Subdural haemorrhage
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Multiple sclerosis
         subjects affected / exposed
    1 / 49 (2.04%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Death
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Sudden cardiac death
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Gastrointestinal disorders
    Gastritis
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Psychotic disorder
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Muscle spasms
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    1 / 49 (2.04%)
    0 / 49 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Clostridium difficile infection
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypomagnesaemia
         subjects affected / exposed
    0 / 49 (0.00%)
    1 / 49 (2.04%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 GS010-GS010 GS010-Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    49 / 49 (100.00%)
    49 / 49 (100.00%)
    Investigations
    Gamma glutamyltransferase increased
         subjects affected / exposed
    1 / 49 (2.04%)
    5 / 49 (10.20%)
         occurrences all number
    1
    5
    Blood glucose increased
         subjects affected / exposed
    1 / 49 (2.04%)
    4 / 49 (8.16%)
         occurrences all number
    1
    5
    Nervous system disorders
    Headache
         subjects affected / exposed
    11 / 49 (22.45%)
    8 / 49 (16.33%)
         occurrences all number
    13
    8
    Eye disorders
    Dry eye
         subjects affected / exposed
    3 / 49 (6.12%)
    5 / 49 (10.20%)
         occurrences all number
    8
    9
    Conjunctival hyperaemia
         subjects affected / exposed
    6 / 49 (12.24%)
    2 / 49 (4.08%)
         occurrences all number
    10
    2
    Vitreous floaters
         subjects affected / exposed
    6 / 49 (12.24%)
    6 / 49 (12.24%)
         occurrences all number
    9
    10
    Iritis
         subjects affected / exposed
    7 / 49 (14.29%)
    11 / 49 (22.45%)
         occurrences all number
    13
    14
    Intraocular pressure increased
         subjects affected / exposed
    9 / 49 (18.37%)
    7 / 49 (14.29%)
         occurrences all number
    16
    12
    Eye pain
         subjects affected / exposed
    10 / 49 (20.41%)
    8 / 49 (16.33%)
         occurrences all number
    15
    9
    Uveitis
         subjects affected / exposed
    11 / 49 (22.45%)
    6 / 49 (12.24%)
         occurrences all number
    20
    7
    Vitritis
         subjects affected / exposed
    19 / 49 (38.78%)
    23 / 49 (46.94%)
         occurrences all number
    40
    26
    Keratic precipitates
         subjects affected / exposed
    15 / 49 (30.61%)
    14 / 49 (28.57%)
         occurrences all number
    31
    18
    Iridocyclitis
         subjects affected / exposed
    14 / 49 (28.57%)
    12 / 49 (24.49%)
         occurrences all number
    33
    18
    Punctate keratitis
         subjects affected / exposed
    12 / 49 (24.49%)
    7 / 49 (14.29%)
         occurrences all number
    25
    11
    Photophobia
         subjects affected / exposed
    3 / 49 (6.12%)
    2 / 49 (4.08%)
         occurrences all number
    5
    3
    Anterior chamber inflammation
         subjects affected / exposed
    1 / 49 (2.04%)
    3 / 49 (6.12%)
         occurrences all number
    2
    3
    Conjunctival haemorrhage
         subjects affected / exposed
    3 / 49 (6.12%)
    6 / 49 (12.24%)
         occurrences all number
    5
    10
    Ocular hypertension
         subjects affected / exposed
    1 / 49 (2.04%)
    3 / 49 (6.12%)
         occurrences all number
    2
    3
    Eye irritation
         subjects affected / exposed
    0 / 49 (0.00%)
    3 / 49 (6.12%)
         occurrences all number
    0
    5
    Iris transillumination defect
         subjects affected / exposed
    2 / 49 (4.08%)
    3 / 49 (6.12%)
         occurrences all number
    4
    3
    Photopsia
         subjects affected / exposed
    3 / 49 (6.12%)
    0 / 49 (0.00%)
         occurrences all number
    5
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    3 / 49 (6.12%)
    3 / 49 (6.12%)
         occurrences all number
    3
    4
    Anxiety
         subjects affected / exposed
    3 / 49 (6.12%)
    2 / 49 (4.08%)
         occurrences all number
    3
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 49 (6.12%)
    9 / 49 (18.37%)
         occurrences all number
    3
    11
    COVID-19
         subjects affected / exposed
    2 / 49 (4.08%)
    5 / 49 (10.20%)
         occurrences all number
    2
    5
    Influenza
         subjects affected / exposed
    2 / 49 (4.08%)
    3 / 49 (6.12%)
         occurrences all number
    2
    3

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    07 Aug 2017
    Amendment number 2, dated 25 September 2017 reiterates the changes made in amendment 1. Main change included the modification of the population used for the primary efficacy analysis from the modified Intent-to-Treat (mITT) to the ITT population requested by the FDA
    16 Jul 2019
    Main changes included the updated of the primary efficacy timepoint which was extended from Year 1 (Week 52) to Year 1.5 (Week 78) based on preliminary results of the study, and addition of quality of life assessments (VFQ-25 and SF-36v2) at Visit 12 (Year 1.5)
    23 Dec 2019
    Main change included the extension of the follow-up period from 2 years to 5 years

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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