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    Clinical Trial Results:
    EXPLORE: A Phase 2, outcomes assessor-masked, multicentre, randomised study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration.

    Summary
    EudraCT number
    		 2019-003421-22
    Trial protocol
    		 GB   FR   DE   ES   IE   NL  
    Global end of trial date
    05 Apr 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    30 Mar 2025
    First version publication date
    30 Mar 2025
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GT005-02
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04437368
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 Sponsor Code II: CPPY988A12202
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.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
    05 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    05 Apr 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To evaluate the effect of GT005 on the progression of GA in subjects with GA due to AMD. Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.nov for complete trial results.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 Apr 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 4
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    United States: 68
    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)
    0
    Adults (18-64 years)
    8
    From 65 to 84 years
    79
    85 years and over
    11

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Subjects were enrolled at 32 centers in 6 countries: 1 center in Australia, 3 centers in France, 2 centers in Germany, 5 centers in Spain, 3 centers in United Kingdom, and 18 centers in United States. A total of 98 subjects were enrolled into the study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 GT005 Low Dose [2E10 vg]
    Arm description
    		 GT005 Low Dose [2E10 vg]
    Arm type
    		 Experimental

    Investigational medicinal product name
    GT005
    Investigational medicinal product code
    PPY988
    Other name
    PPY988
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Ophthalmic use
    Dosage and administration details
    single time subretinal injection of GT005 [5E10 vg]

    Arm title
    		 GT005 High Dose [2E11 vg]
    Arm description
    		 GT005 High Dose [2E11 vg]
    Arm type
    		 Experimental

    Investigational medicinal product name
    GT005
    Investigational medicinal product code
    PPY988
    Other name
    PPY988
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Ophthalmic use
    Dosage and administration details
    single time subretinal injection of GT005 [2E11 vg]

    Arm title
    		 Untreated control
    Arm description
    		 Subjects allocated to the untreated control group did not receive any treatment.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Started
    52
    9
    37
    Randomized Part 1
    10
    9
    14
    Randomized Part 2
    42
    0 [1]
    23
    Randomized and treated Part 1
    9
    9
    0 [2]
    Randomized and treated Part 2
    18
    0 [3]
    0 [4]
    Completed
    6
    8
    7
    Not completed
    46
    1
    30
         Adverse event, serious fatal
    3
    -
    2
         Consent withdrawn by subject
    4
    -
    3
         Study terminated by sponsor
    16
    1
    25
         Sponsor instructions
    23
    -
    -
    Notes
    [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The disposition is broken down to greater detail: Randomized Part 1 , Randomized Part 2, Randomized and treated Part 1, and Randomized and treated Part 2.
    [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The disposition is broken down to greater detail: Randomized Part 1 , Randomized Part 2, Randomized and treated Part 1, and Randomized and treated Part 2.
    [3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The disposition is broken down to greater detail: Randomized Part 1 , Randomized Part 2, Randomized and treated Part 1, and Randomized and treated Part 2.
    [4] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
    Justification: The disposition is broken down to greater detail: Randomized Part 1 , Randomized Part 2, Randomized and treated Part 1, and Randomized and treated Part 2.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    GT005 Low Dose [2E10 vg]
    Reporting group description
    		 GT005 Low Dose [2E10 vg]

    Reporting group title
    GT005 High Dose [2E11 vg]
    Reporting group description
    		 GT005 High Dose [2E11 vg]

    Reporting group title
    Untreated control
    Reporting group description
    		 Subjects allocated to the untreated control group did not receive any treatment.

    Reporting group values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control Total
    Number of subjects
    		 52 9 37 98
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 0 0 0 0
        Adults (18-64 years)
    		 2 1 5 8
        From 65-84 years
    		 42 7 30 79
        85 years and over
    		 8 1 2 11
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 76.8 ( 7.33 ) 72.7 ( 8.96 ) 75.2 ( 7.07 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 34 5 18 57
        Male
    		 18 4 19 41
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 1 0 0 1
        Asian
    		 0 0 0 0
        Native Hawaiian or Other Pacific Islander
    		 0 0 0 0
        Black or African American
    		 0 0 0 0
        White
    		 51 7 35 93
        More than one race
    		 0 0 0 0
        Unknown or Not Reported
    		 0 2 2 4

    End points

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    End points reporting groups
    Reporting group title
    GT005 Low Dose [2E10 vg]
    Reporting group description
    		 GT005 Low Dose [2E10 vg]

    Reporting group title
    GT005 High Dose [2E11 vg]
    Reporting group description
    		 GT005 High Dose [2E11 vg]

    Reporting group title
    Untreated control
    Reporting group description
    		 Subjects allocated to the untreated control group did not receive any treatment.

    Primary: The change from baseline to Week 48 in geographic atrophy (GA) - Part 1

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    End point title
    		 The change from baseline to Week 48 in geographic atrophy (GA) - Part 1
    End point description
    The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)
    End point type
    Primary
    End point timeframe
    Baseline, Weeks 12, 24, 36, and 48
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    8
    12
    Units: mm2
    least squares mean (standard error)
        Part 1 Week 12 (n=9,8,12)
    0.773 ( 0.2151 )
    0.764 ( 0.2159 )
    0.482 ( 0.1848 )
        Part 1 Week 24 (n=9,7,9)
    1.338 ( 0.2763 )
    1.519 ( 0.2845 )
    0.680 ( 0.2500 )
        Part 1 Week 36 (n=6,7,10)
    1.800 ( 0.3740 )
    2.044 ( 0.3669 )
    1.132 ( 0.3229 )
        Part 1 Week 48 (n=5,8,10)
    2.120 ( 0.3726 )
    2.378 ( 0.3414 )
    1.144 ( 0.3040 )
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 12
    Comparison groups
    Untreated control v GT005 Low Dose [2E10 vg]
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.291
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.195
         upper limit
    		 0.777
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2838
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 12
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.282
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.206
         upper limit
    		 0.769
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.2843
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 24
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.658
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.017
         upper limit
    		 1.299
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.373
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 24
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.84
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.189
         upper limit
    		 1.49
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.3791
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 36
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.912
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.078
         upper limit
    		 1.746
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.4887
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 36
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.668
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.177
         upper limit
    		 1.512
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.4954
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 48
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    0.976
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.15
         upper limit
    		 1.803
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.4813
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 48
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.233
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 0.445
         upper limit
    		 2.022
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.4573

    Secondary: The change from baseline in geographic atrophy (GA) at Week 72 and Week 96 - Part 1

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    End point title
    		 The change from baseline in geographic atrophy (GA) at Week 72 and Week 96 - Part 1
    End point description
    The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    6
    8
    9
    Units: mm2
    least squares mean (standard error)
        Part 1 Week 72 (n=5,7,9)
    3.643 ( 0.9725 )
    3.149 ( 0.9150 )
    1.875 ( 0.8273 )
        Part 1 Week 96 (n=6,8,7)
    4.837 ( 1.0712 )
    4.074 ( 1.0305 )
    2.796 ( 0.9240 )
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 72
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.769
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.441
         upper limit
    		 3.979
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.2808
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 72
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.274
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.863
         upper limit
    		 3.412
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.2349
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 96
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    15
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    2.041
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -0.386
         upper limit
    		 4.468
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.4177
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 96
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    17
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean
    Point estimate
    1.278
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -1.099
         upper limit
    		 3.655
    Variability estimate
    Standard error of the mean
    Dispersion value
    1.3857

    Secondary: Summary of Adverse Events

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    End point title
    		 Summary of Adverse Events
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject. A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs. AE = adverse event SAE = serious adverse event Rel = related Trt = study treatment Proc. = procedure Disc. = discontinuation AESI = AE of special interest Surg. = surgical
    End point type
    Secondary
    End point timeframe
    Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    52
    9
    37
    Units: Participants
        Subjects with at least 1 ocular AE - study eye
    18
    9
    2
        Subjects with at least 1ocular AE - fellow eye
    6
    3
    5
        Subjects with at least one non-ocular AE
    13
    8
    14
        n >= 1 ocular AE rel. to trt. - study eye
    3
    4
    0
        n with at least 1 non-ocular AE rel. to trt.
    0
    0
    0
        n >= 1 ocular AE rel. to surg proc - study eye
    12
    7
    0
        n >= 1 non-ocular AE rel. to surg. proc.
    0
    0
    0
        n >= 1 ocular AE rel.to proc.- study eye
    1
    1
    0
        n >= 1 non-ocular AE rel. to study proc.
    1
    0
    0
        n >= 1 ocular AE leading to disc.- study eye
    0
    0
    0
        n >= 1 non-ocular AE leading to disc.
    3
    0
    2
        n >= 1 ocular AESI for the study eye
    4
    7
    0
        n with at least 1 ocular AESI for the fellow eye
    0
    0
    1
        n with at least 1 ocular SAE for the study eye
    1
    0
    0
        n with at least 1 ocular SAE for the fellow eye
    0
    0
    0
        Subjects with at least 1 non-ocular SAE
    5
    2
    4
        n >= 1 ocular SAE rel. to study trt.- study eye
    0
    0
    0
        n >= 1 non-ocular SAE rel.to study trt.
    0
    0
    0
        n >= 1 ocular SAE rel. to surg. proc. - study eye
    0
    0
    0
        n >= 1 non-ocular SAE rel.to surg. proc.
    0
    0
    0
        n >= 1 ocular SAE rel. to study proc. - study eye
    0
    0
    0
        n >= 1 non-ocular SAE rel. to study proc.
    0
    0
    0
        n >= 1 ocular SAE leading to disc.- study eye
    0
    0
    0
        n >= 1 non-ocular SAE leading to study disc.
    3
    0
    2
        Deaths
    3
    0
    2
    No statistical analyses for this end point

    Secondary: Ocular adverse events by primary system organ class and preferred term for the study eye

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    End point title
    		 Ocular adverse events by primary system organ class and preferred term for the study eye
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject. A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs. System organ classes are sorted alphabetically, and preferred terms are sorted by decreasing overall frequency within system organ class.
    End point type
    Secondary
    End point timeframe
    Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    52
    9
    37
    Units: Participants
        Subjects with at least one event
    18
    9
    2
        Eye disorders
    15
    9
    2
        -Retinal pigmentation
    3
    6
    0
        -Conjunctival haemorrhage
    6
    1
    0
        -Cataract
    3
    2
    1
        -Cataract nuclear
    2
    1
    0
        -Eye pain
    1
    2
    0
        -Retinal haemorrhage
    2
    1
    0
        -Anterior chamber cell
    1
    1
    0
        -Blepharitis
    2
    0
    0
        -Conjunctivitis allergic
    1
    1
    0
        -Anterior chamber flare
    0
    1
    0
        -Choroidal detachment
    0
    1
    0
        -Conjunctival hyperaemia
    1
    0
    0
        -Dry eye
    1
    0
    0
        -Eye pruritus
    0
    0
    1
        -Hypotony maculopathy
    0
    1
    0
        -Iridocyclitis
    0
    1
    0
        -Iritis
    1
    0
    0
        -Keratitis
    1
    0
    0
        -Lacrimation increased
    0
    1
    0
        -Macular hole
    1
    0
    0
        -Meibomian gland dysfunction
    1
    0
    0
        -Metamorphopsia
    1
    0
    0
        -Ocular hypertension
    1
    0
    0
        -Open angle glaucoma
    0
    1
    0
        -Photophobia
    0
    1
    0
        -Photopsia
    1
    0
    0
        -Posterior capsule opacification
    0
    0
    1
        -Punctate keratitis
    1
    0
    0
        -Retinal depigmentation
    1
    0
    0
        -Visual acuity reduced
    1
    0
    0
        -Visual impairment
    1
    0
    0
        -Visual snow syndrome
    1
    0
    0
        -Vitreous floaters
    1
    0
    0
        -Vitreous haemorrhage
    1
    0
    0
        Injury, poisoning and procedural complications
    4
    1
    0
        -Post procedural discomfort
    2
    0
    0
        -Procedural pain
    1
    1
    0
        -Suture related complication
    1
    1
    0
        Investigations
    4
    3
    0
        -Intraocular pressure increased
    4
    3
    0
        Skin and subcutaneous tissue disorders
    1
    0
    0
        -Telangiectasia
    1
    0
    0
    No statistical analyses for this end point

    Secondary: Non-ocular adverse events - summary

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    End point title
    		 Non-ocular adverse events - summary
    End point description
    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject. A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs.
    End point type
    Secondary
    End point timeframe
    Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    52
    9
    37
    Units: Participants
    13
    8
    14
    No statistical analyses for this end point

    Secondary: Change in GA morphology from Baseline to Week 96 on colour fundus photography (CFP) - Number of participants with increase in Fundus autofluorescence - Part 1

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    End point title
    		 Change in GA morphology from Baseline to Week 96 on colour fundus photography (CFP) - Number of participants with increase in Fundus autofluorescence - Part 1
    End point description
    Change in GA morphology on multimodal imaging through Week 96
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5,12,24,36,48,72,96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    8
    12
    Units: Participants
        Part 1 Week 5 (n= 0,2,0)
    999
    2
    999
        Part 1 Week 12 (n=9,8,12)
    8
    8
    11
        Part 1 Week 24 (n=9,7,10)
    8
    7
    10
        Part 1 Week 36 (n=6,7,10)
    5
    7
    10
        Part 1 Week 48 (n=5,8,10)
    5
    8
    10
        Part 1 Week 72 (n=5,8,9)
    5
    8
    9
        Part 1 Week 96 (n=6,8,7)
    6
    8
    7
    No statistical analyses for this end point

    Secondary: Change from Baseline to Week 48 in GA morphology on colour fundus photography (CFP) - Number of participants with increase in Fundus autofluorescence - Part 2

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    End point title
    		 Change from Baseline to Week 48 in GA morphology on colour fundus photography (CFP) - Number of participants with increase in Fundus autofluorescence - Part 2
    End point description
    Change in GA morphology on multimodal imaging through Week 48
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5,12,24,36,48
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    17
    0 [1]
    20
    Units: Participants
        Part 2 Week 5 (n= 16,0, 0)
    16
    999
        Part 2 Week 12 (n=17,0,20)
    17
    20
        Part 2 Week 24 (n=17,0,11)
    16
    11
        Part 2 Week 36 (n=14,0,0)
    13
    999
        Part 2 Week 48 (n=5,0,0)
    5
    999
    Notes
    [1] - Not applicable for Part 2
    No statistical analyses for this end point

    Secondary: Change in Best corrected visual acuity (BCVA) Score from Baseline through Week 96 via the early treatment for diabetic retinopathy (ETDRS) chart - Part 1

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    End point title
    		 Change in Best corrected visual acuity (BCVA) Score from Baseline through Week 96 via the early treatment for diabetic retinopathy (ETDRS) chart - Part 1
    End point description
    BCVA was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts. Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 1, 5, 8, 12, 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    9
    12
    Units: Letters read
    least squares mean (standard error)
        Part 1 - Week 1 (n=9,9,0)
    -4.7 ( 4.04 )
    -5.2 ( 4.27 )
    999 ( 999 )
        Part 1 - Week 5 (n=8,9,0)
    -1.7 ( 4.08 )
    -7.6 ( 4.26 )
    999 ( 999 )
        Part 1 - Week 8 (n=9,9,0)
    -1.6 ( 4.03 )
    -4.5 ( 4.23 )
    999 ( 999 )
        Part 1 - Week 12 (N=9,9,12)
    -2.3 ( 4.03 )
    -7.5 ( 4.18 )
    -1.2 ( 3.81 )
        Part 1 - Week 24 (n=9,9,10)
    -5.6 ( 4.03 )
    -12.0 ( 4.18 )
    -0.4 ( 3.87 )
        Part 1 - Week 36 (n=8,8,10)
    -10.0 ( 4.10 )
    -12.9 ( 4.28 )
    -1.0 ( 3.89 )
        Part 1 - Week 48 (n=5,8,10)
    -8.6 ( 4.52 )
    -13.3 ( 4.32 )
    -5.7 ( 3.91 )
        Part 1 - Week 72 (n=5,8,10)
    -6.3 ( 4.56 )
    -9.6 ( 4.34 )
    -8.9 ( 3.94 )
        Part 1 - Week 96 (n=6,8,7)
    -6.5 ( 4.50 )
    -11.0 ( 4.33 )
    -7.9 ( 4.30 )
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 12
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -1.1
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -10.3
         upper limit
    		 8.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.49
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 12
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -6.3
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -16.3
         upper limit
    		 3.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.96
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 24
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -5.2
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -14.5
         upper limit
    		 4.1
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.54
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 24
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -11.7
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -21.7
         upper limit
    		 -1.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    6
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 36
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -9
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -18.4
         upper limit
    		 0.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.63
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 36
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -11.9
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -22.1
         upper limit
    		 -1.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.08
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 48
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -2.9
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -12.7
         upper limit
    		 6.9
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.88
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 48
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -7.6
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -17.9
         upper limit
    		 2.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.14
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 72
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    2.6
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -7.4
         upper limit
    		 12.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.96
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 72
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -0.7
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -11.1
         upper limit
    		 9.6
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.18
    Statistical analysis title
    		 GT005 Low Dose [2E10 vg] v Untreated control
    Statistical analysis description
    Week 96
    Comparison groups
    GT005 Low Dose [2E10 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    1.4
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -8.7
         upper limit
    		 11.5
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.07
    Statistical analysis title
    		 GT005 High Dose [2E11 vg] v Untreated control
    Statistical analysis description
    Week 96
    Comparison groups
    GT005 High Dose [2E11 vg] v Untreated control
    Number of subjects included in analysis
    21
    Analysis specification
    Pre-specified
    Analysis type
    Method
    		 mixed model repeated measures
    Parameter type
    		 LS Mean Difference
    Point estimate
    -3
    Confidence interval
         level
    		 90%
         sides
    2-sided
         lower limit
    		 -13.8
         upper limit
    		 7.8
    Variability estimate
    Standard error of the mean
    Dispersion value
    6.47

    Secondary: Change in Low luminance difference (LLD) letter count from Baseline at Weeks 12, 24, 36, 48, 72 and 96, via early treatment for diabetic retinopathy (ETDRS) chart - part 1

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    End point title
    		 Change in Low luminance difference (LLD) letter count from Baseline at Weeks 12, 24, 36, 48, 72 and 96, via early treatment for diabetic retinopathy (ETDRS) chart - part 1
    End point description
    LLD was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts. The test was to be performed after BCVA testing, prior to pupil dilation, and distance refraction was to be carried out before Low Luminance Visual Acuity (LLVA) was measured. LLVA was to be measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. The LLD was calculated as the difference between BCVA and LLVA. Initially, letters were to be read at a distance of 4 metres from the chart. If <20 letters were read at 4 metres, testing at 1 metre should have been performed. LLD was to be reported as number of letters read correctly by the subject. Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    9
    12
    Units: Letters read
    arithmetic mean (standard deviation)
        Part 1 - Week 12 (N=9,9,12)
    2.0 ( 9.06 )
    2.4 ( 22.49 )
    -2.0 ( 7.21 )
        Part 1 - Week 24 (n=9,9,10)
    2.1 ( 8.40 )
    2.9 ( 24.81 )
    -2.9 ( 7.68 )
        Part 1 - Week 36 (n=8,8,10)
    2.3 ( 15.65 )
    -7.5 ( 19.41 )
    -4.0 ( 12.51 )
        Part 1 - Week 48 (n=5,8,10)
    3.0 ( 6.96 )
    1.8 ( 26.25 )
    -6.2 ( 19.99 )
        Part 1 - Week 72 (n=5,8,10)
    4.4 ( 7.57 )
    3.1 ( 32.24 )
    -6.6 ( 21.15 )
        Part 1 - Week 96 (n=6,8,6)
    5.2 ( 8.47 )
    1.5 ( 29.17 )
    -10.5 ( 27.41 )
    No statistical analyses for this end point

    Secondary: Change in Low luminance difference (LLD) letter count from Baseline at Weeks 12, 24, 36, and 48, via early treatment for diabetic retinopathy (ETDRS) chart - part 2

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    End point title
    		 Change in Low luminance difference (LLD) letter count from Baseline at Weeks 12, 24, 36, and 48, via early treatment for diabetic retinopathy (ETDRS) chart - part 2
    End point description
    LLD was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts. The test was to be performed after BCVA testing, prior to pupil dilation, and distance refraction was to be carried out before Low Luminance Visual Acuity (LLVA) was measured. LLVA was to be measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. The LLD was calculated as the difference between BCVA and LLVA. Initially, letters were to be read at a distance of 4 metres from the chart. If <20 letters were read at 4 metres, testing at 1 metre should have been performed. LLD was to be reported as number of letters read correctly by the subject. Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 12, 24, 36, and 48
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    18
    0 [2]
    20
    Units: Letters read
    arithmetic mean (standard deviation)
        Part 2 - Week 12 (N=18,0,20)
    0.7 ( 10.83 )
    ( )
    -1.4 ( 11.76 )
        Part 2 - Week 24 (n=17,0,10)
    2.6 ( 9.98 )
    ( )
    -1.6 ( 20.13 )
        Part 2 - Week 36 (n=14,0,0)
    4.6 ( 11.77 )
    ( )
    999 ( 999 )
        Part 2 - Week 48 (n=5,0,0)
    -2.8 ( 4.32 )
    ( )
    999 ( 999 )
    Notes
    [2] - Not applicable for Part 2
    No statistical analyses for this end point

    Secondary: Change from Baseline at Weeks 24, 36, 48, 72 and 96 in Reading performance, measured as the maximum reading speed (words per minute), as assessed by Minnesota low-vision reading test (MNRead) chart - part 1

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    End point title
    		 Change from Baseline at Weeks 24, 36, 48, 72 and 96 in Reading performance, measured as the maximum reading speed (words per minute), as assessed by Minnesota low-vision reading test (MNRead) chart - part 1
    End point description
    The maximum reading speed (MRS) represents the highest reading speed an individual can achieve when print size is not a limiting factor. Essentially, it measures how quickly a person can read text when the print is large enough to be easily readable. A higher count represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    8
    9
    Units: Words read per minute
    arithmetic mean (standard deviation)
        Part 1 - Week 24 (n=9,8,9)
    20.384 ( 74.7769 )
    -36.154 ( 40.3322 )
    -7.837 ( 21.8918 )
        Part 1 - Week 36 (n=7,7,8)
    -15.048 ( 33.0479 )
    -34.653 ( 27.2693 )
    -15.245 ( 22.0983 )
        Part 1 - Week 48 (n=4,7,9)
    10.397 ( 11.8698 )
    -36.285 ( 40.0938 )
    -6.837 ( 31.6162 )
        Part 1 - Week 72 (n=5,7,9)
    -20.796 ( 42.2571 )
    -44.913 ( 46.7594 )
    -15.429 ( 30.1012 )
        Part 1 - Week 96 (n=6,5,5)
    10.802 ( 32.1161 )
    -32.266 ( 36.9172 )
    -21.602 ( 26.0643 )
    No statistical analyses for this end point

    Secondary: Change from Baseline at Weeks 24, 36 and 48 in Reading performance, measured as the maximum reading speed (words per minute), as assessed by Minnesota low-vision reading test (MNRead) chart - part 2

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    End point title
    		 Change from Baseline at Weeks 24, 36 and 48 in Reading performance, measured as the maximum reading speed (words per minute), as assessed by Minnesota low-vision reading test (MNRead) chart - part 2
    End point description
    A higher count represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36 and 48
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    16
    0 [3]
    10
    Units: Words read per minute
    arithmetic mean (standard deviation)
        Part 2 - Week 24 (n=16,0,10)
    15.581 ( 78.2848 )
    ( )
    -30.707 ( 27.3225 )
        Part 2 - Week 36 (n=14,0,0)
    9.812 ( 94.4255 )
    ( )
    999 ( 999 )
        Part 2 - Week 48 (n=5,0,0)
    -28.111 ( 38.6209 )
    ( )
    999 ( 999 )
    Notes
    [3] - Not applicable for Part 2
    No statistical analyses for this end point

    Secondary: Change from Baseline at Weeks 24, 36, 48, 72 and 96 in Functional reading independence (FRI) index - part 1

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    End point title
    		 Change from Baseline at Weeks 24, 36, 48, 72 and 96 in Functional reading independence (FRI) index - part 1
    End point description
    The FRI index is a patient-reported outcome measure developed specifically for use in GA patients. The FRI index evaluates the level of independence subjects have in performing everyday activities that require reading, such as writing a cheque or reading a prescription. Scores derived from the index range from 1 (unable to do) to 4 (total independence). A higher score represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    8
    10
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Part 1 - Week 24 (n=9,8,10)
    0.7 ( 3.57 )
    -1.3 ( 2.55 )
    -0.3 ( 4.06 )
        Part 1 - Week 36 (n=8,5,10)
    -1.0 ( 4.24 )
    2.6 ( 4.10 )
    -0.6 ( 4.72 )
        Part 1 - Week 48 (n=6,7,10)
    0.5 ( 2.43 )
    -0.1 ( 7.69 )
    -3.4 ( 4.38 )
        Part 1 - Week 72 (n=5,7,10)
    -3.8 ( 4.60 )
    0.4 ( 3.41 )
    -1.3 ( 4.35 )
        Part 1 - Week 96 (n=6,6,7)
    -2.7 ( 5.79 )
    -2.0 ( 3.29 )
    -0.7 ( 5.53 )
    No statistical analyses for this end point

    Secondary: Change from Baseline at Weeks 24, 36 and 48 in Functional reading independence (FRI) index - part 2

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    End point title
    		 Change from Baseline at Weeks 24, 36 and 48 in Functional reading independence (FRI) index - part 2
    End point description
    The FRI index is a patient-reported outcome measure developed specifically for use in GA patients. The FRI index evaluates the level of independence subjects have in performing everyday activities that require reading, such as writing a cheque or reading a prescription. Scores derived from the index range from 1 (unable to do) to 4 (total independence). A higher score represents better visual functioning.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36 and 48
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    17
    0 [4]
    9
    Units: Scores on a scale
    arithmetic mean (standard deviation)
        Part 2 - Week 24 (n=17,0,9)
    -0.4 ( 2.83 )
    ( )
    0.4 ( 6.86 )
        Part 2 - Week 36 (n=14,0,0)
    -1.7 ( 3.83 )
    ( )
    999 ( 999 )
        Part 2 - Week 48 (n=5,0,0)
    0.4 ( 5.13 )
    ( )
    999 ( 999 )
    Notes
    [4] - Not applicable for Part 2
    No statistical analyses for this end point

    Secondary: Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1

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    End point title
    		 Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1
    End point description
    The National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) measures the influence of visual disability and visual symptoms on general health domains. The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better visual functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A composite score is derived based on the average of the 11 subscales.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    9
    9
    10
    Units: Scores on a Scale
    arithmetic mean (standard deviation)
        Part 1 - Week 24 (n=9,9,10)
    -2.312 ( 5.7079 )
    -3.755 ( 7.4470 )
    -7.304 ( 12.5136 )
        Part 1 - Week 36 (n=8,6,10)
    -4.400 ( 11.1307 )
    1.960 ( 9.0113 )
    -4.706 ( 11.8617 )
        Part 1 - Week 48 (n=6,8,10)
    -6.439 ( 24.2128 )
    -6.810 ( 11.5980 )
    -4.091 ( 15.3598 )
        Part 1 - Week 72 (n=5,8,10)
    -8.698 ( 22.1289 )
    -3.332 ( 8.1470 )
    -3.826 ( 11.0029 )
        Part 1 - Week 96 (n=6,7,7)
    -10.352 ( 23.9470 )
    0.171 ( 11.3543 )
    -10.700 ( 11.6900 )
    No statistical analyses for this end point

    Secondary: Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2

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    End point title
    		 Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2
    End point description
    The National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) measures the influence of visual disability and visual symptoms on general health domains. The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better visual functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A composite score is derived based on the average of the 11 subscales.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 24, 36, 48, 72 and 96
    End point values
    		 GT005 Low Dose [2E10 vg] GT005 High Dose [2E11 vg] Untreated control
    Number of subjects analysed
    17
    0 [5]
    8
    Units: Scores on a Scale
    arithmetic mean (standard deviation)
        Part 2 - Week 24 (n=17,0,8)
    -4.034 ( 6.8881 )
    ( )
    -3.070 ( 7.4251 )
        Part 2 - Week 36 (n=14,0,0)
    -2.733 ( 7.9219 )
    ( )
    999 ( 999 )
        Part 2 - Week 48 (n=5,0,0)
    -0.530 ( 6.9722 )
    ( )
    999 ( 999 )
    Notes
    [5] - Not applicable for Part 2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    GT005@High dose@[2E11 vg]
    Reporting group description
    		 GT005@High dose@[2E11 vg]

    Reporting group title
    GT005@Low dose@[2E10 vg]
    Reporting group description
    		 GT005@Low dose@[2E10 vg]

    Reporting group title
    Overall
    Reporting group description
    		 Overall

    Reporting group title
    Untreated control
    Reporting group description
    		 Untreated control

    Serious adverse events
    		 GT005@High dose@[2E11 vg] GT005@Low dose@[2E10 vg] Overall Untreated control
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 9 (22.22%)
    5 / 52 (9.62%)
    11 / 98 (11.22%)
    4 / 37 (10.81%)
         number of deaths (all causes)
    0
    3
    5
    2
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer metastatic
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hepatic cancer
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    Lung neoplasm malignant
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    Lung adenocarcinoma stage IV
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hip fracture
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Rib fracture
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Amyotrophic lateral sclerosis
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cerebrovascular accident
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Encephalopathy
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    Sciatica
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Seizure
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 4
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Visual acuity reduced - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pleural effusion
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Hypoxia
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    Choking
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    Pneumothorax
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
    0 / 0
    Staphylococcal infection
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
    Urinary tract infection
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 GT005@High dose@[2E11 vg] GT005@Low dose@[2E10 vg] Overall Untreated control
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    9 / 9 (100.00%)
    19 / 52 (36.54%)
    44 / 98 (44.90%)
    16 / 37 (43.24%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer recurrent
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Meningioma
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    1
    0
    2
    1
    Orthostatic hypotension
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Peripheral ischaemia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Drug intolerance
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Reproductive system and breast disorders
    Vaginal haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Psychiatric disorders
    Sleep disorder
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Investigations
    Blood potassium decreased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Intraocular pressure increased - Study eye
         subjects affected / exposed
    3 / 9 (33.33%)
    4 / 52 (7.69%)
    7 / 98 (7.14%)
    0 / 37 (0.00%)
         occurrences all number
    4
    5
    9
    0
    C-reactive protein increased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    2
    1
    Blood pressure increased
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Blood pressure decreased
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Lower limb fracture
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Joint dislocation
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Hand fracture
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Fall
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    3 / 98 (3.06%)
    1 / 37 (2.70%)
         occurrences all number
    1
    2
    4
    1
    Ligament sprain
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Suture related complication - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Tendon rupture
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Meniscus injury
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Muscle strain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Post procedural discomfort - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 52 (3.85%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Procedural pain - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Skin abrasion
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Skin laceration
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Upper limb fracture
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Cardiac disorders
    Cardiomyopathy
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Coronary artery disease
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Nervous system disorders
    Carpal tunnel syndrome
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Cognitive disorder
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Tension headache
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Seizure
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Hemiparesis
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Headache
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    2
    1
    Facial paralysis
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Dizziness
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Transient ischaemic attack
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Blood and lymphatic system disorders
    Hypochromic anaemia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Anaemia
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    1
    0
    2
    1
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Eye disorders
    Anterior chamber flare - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Anterior chamber cell - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Cataract - Fellow eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    3 / 98 (3.06%)
    2 / 37 (5.41%)
         occurrences all number
    1
    0
    3
    2
    Blepharitis - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 52 (3.85%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Blepharitis - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    2 / 52 (3.85%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Cataract - Study eye
         subjects affected / exposed
    2 / 9 (22.22%)
    3 / 52 (5.77%)
    6 / 98 (6.12%)
    1 / 37 (2.70%)
         occurrences all number
    2
    3
    6
    1
    Cataract nuclear - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    2 / 52 (3.85%)
    3 / 98 (3.06%)
    0 / 37 (0.00%)
         occurrences all number
    2
    2
    4
    0
    Choroidal detachment - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Choroidal neovascularisation - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Conjunctival haemorrhage - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    6 / 52 (11.54%)
    7 / 98 (7.14%)
    0 / 37 (0.00%)
         occurrences all number
    1
    6
    7
    0
    Conjunctivitis allergic - Fellow eye
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Conjunctival hyperaemia - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Conjunctivitis allergic - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Iridocyclitis - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Hypotony maculopathy - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Eye pruritus - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Eye pruritus - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Eye pain - Study eye
         subjects affected / exposed
    2 / 9 (22.22%)
    1 / 52 (1.92%)
    3 / 98 (3.06%)
    0 / 37 (0.00%)
         occurrences all number
    2
    1
    3
    0
    Dry eye - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Dry eye - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Iritis - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Meibomian gland dysfunction - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Macular hole - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Lacrimation increased - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Keratitis - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Meibomian gland dysfunction - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Metamorphopsia - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Ocular hypertension - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Open angle glaucoma - Fellow eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Photopsia - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Photopsia - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Photophobia - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Open angle glaucoma - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Posterior capsule opacification - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Retinal degeneration - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Punctate keratitis - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Posterior capsule opacification - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Retinal depigmentation - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Retinal haemorrhage - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Retinal haemorrhage - Study eye
         subjects affected / exposed
    1 / 9 (11.11%)
    2 / 52 (3.85%)
    3 / 98 (3.06%)
    0 / 37 (0.00%)
         occurrences all number
    1
    2
    3
    0
    Retinal oedema - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Visual snow syndrome - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Visual impairment - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Visual impairment - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Retinal tear - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Retinal pigmentation - Study eye
         subjects affected / exposed
    6 / 9 (66.67%)
    3 / 52 (5.77%)
    9 / 98 (9.18%)
    0 / 37 (0.00%)
         occurrences all number
    6
    3
    9
    0
    Vitreous haemorrhage - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Vitreous floaters - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Vitreous floaters - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Vitreous detachment - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Gastrointestinal disorders
    Hiatus hernia
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    2 / 98 (2.04%)
    2 / 37 (5.41%)
         occurrences all number
    0
    0
    2
    2
    Constipation
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    2 / 98 (2.04%)
    2 / 37 (5.41%)
         occurrences all number
    0
    0
    2
    2
    Diverticulum intestinal
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Dysphagia
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Abdominal pain upper
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Umbilical hernia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Oesophageal achalasia
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Loose tooth
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Inguinal hernia
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Rosacea
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Telangiectasia - Study eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Urticaria
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Telangiectasia - Fellow eye
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Renal and urinary disorders
    Urethral polyp
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Urethral haemorrhage
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Dysuria
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Renal impairment
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Spinal osteoarthritis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    0
    1
    2
    1
    Rotator cuff syndrome
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Plantar fasciitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Pain in extremity
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Osteoarthritis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Muscle contracture
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Temporomandibular joint syndrome
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Infections and infestations
    Cystitis
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    COVID-19
         subjects affected / exposed
    2 / 9 (22.22%)
    3 / 52 (5.77%)
    6 / 98 (6.12%)
    1 / 37 (2.70%)
         occurrences all number
    2
    3
    6
    1
    Bronchitis
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Ear infection
         subjects affected / exposed
    1 / 9 (11.11%)
    1 / 52 (1.92%)
    2 / 98 (2.04%)
    0 / 37 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Herpes zoster
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Sinusitis
         subjects affected / exposed
    1 / 9 (11.11%)
    0 / 52 (0.00%)
    2 / 98 (2.04%)
    1 / 37 (2.70%)
         occurrences all number
    1
    0
    2
    1
    Metabolism and nutrition disorders
    Vitamin D deficiency
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Obesity
         subjects affected / exposed
    0 / 9 (0.00%)
    0 / 52 (0.00%)
    1 / 98 (1.02%)
    1 / 37 (2.70%)
         occurrences all number
    0
    0
    1
    1
    Dyslipidaemia
         subjects affected / exposed
    0 / 9 (0.00%)
    1 / 52 (1.92%)
    1 / 98 (1.02%)
    0 / 37 (0.00%)
         occurrences all number
    0
    1
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    31 Oct 2019
    The protocol was updated to extricate that the primary endpoint was masked to the assessor and the number of subjects had been increased to include a projected 10% withdrawal rate
    13 May 2020
    The protocol was updated to change the corticosteroid regimen required for GT005-treated subjects (from systemic to topical administration) given the COVID-19 risk, and also due to the absence of clinically meaningful inflammation in all dose escalation cohorts of the ongoing Phase I/II study, FOCUS. An additional in-clinic visit added at Week 8 (Visit 5) was included. The number of study subjects increased to adequately power for a statistically significant effect assuming the true underlying change in GA area was inhibited by a 40% treatment effect. Inclusion/Exclusion criteria were updated to more accurately reflect the anticipated age group of subjects with GA, to cap the number of subjects with GA lesion size >10 mm2, to clarify the serum CFI inclusion and to further specify the excluded confounding comorbidities. Given the low prevalence of CFI mutations and desired to minimize subject study activities, genotyping and serum CFI results received to confirm eligibility prior to other screening activities commencing. CNV was added to the list of adverse events of special interest.
    13 Nov 2020
    Subject participation period has been extended to a total of 96 weeks with the addition of 2 extra visits at Week 72 and Week 96. Key changes to the eligibility criteria included: • Subjects that have dry AMD and/or GA, secondary to AMD in study eye as determined by the Investigator, and a diagnosis of AMD in the contralateral eye (except if the subject is monocular) lowering of the BCVA score from 34 or better to 24 letters or better, using ETDRS charts. • included an evidence-based approach to enrolment of subjects into the study. • clarified the required duration of prior intraocular surgery before screening cataract surgery. • broadened the malignancies/cancer types excluded from the study. The addition of an aqueous sample for biomarker analysis at Visit 2 to facilitate understanding of long-term transgene expression. All OCT-bleb scans to be read by a dose-masked reader at the CRC. Inclusion of new imaging assessments: FA and OCT-A. The differing OCT modalities (OCT-A, OCT bleb and OCT macula) have been clarified, all OCT scans were sent to the CRC and that OCT bleb scans are to be read by a dose-masked reader at the CRC. A separate genotyping ICF was +created to facilitate ease of genotyping for screening eligibility. Randomisation to either study eye if both are eligible has been changed to select the worse eye (BVCA score). Removal of the requirement to identify the PRL prior to surgery. The location of the bleb has been relocated to outside the vascular arcades. Safety reporting requirements updated to collect all AEs from date of consent.
    03 Jun 2021
    Change in eligibility criteria to allow inclusion of subjects with CNV/wet AMD in fellow eye. Subjects with fellow eye CNV were stratified across all three treatment groups. Clarification of Screening CNV assessment with OCT-A and FA imaging requirements. Clarification of Microperimetry imaging requirements. Removal of text stating OCT-A certification was not required. Viral shedding statement updated to be consistent with core study documentation and text included to confirm viral shedding was monitored in the Phase I/II FOCUS study. Correction of text related to causality assessment of adverse events associated with surgical procedures rather than study procedures.
    06 Dec 2022
    The study design was amended to include two parts: Part 1 provides an evaluation of CFI rare variant GA subjects with the low and high dose of GT005 compared with untreated control, randomized in a 2:1 ratio; Part 2 evaluates the broad genetic GA population (including CFI rare variant) with the low dose of GT005 compared with untreated control randomized in a 2:1 ratio. The dosing and administration section was amended such that subretinal GT005 dosing occurred via one or more GT005 administration bleb(s). Prohibited Medications section: subjects should not receive systemic and/or locally in the study eye approved and investigational GA treatment up to Week 48 for the untreated group and until the end of study for the treated group. The overall study sample size increased to approximately 202 and the number investigational sites increased from 40 to approximately 60. Statistical Methods have been updated in line with the change in sample size and study design. Secondary and exploratory study endpoints have also been revised. RPE changes following GT005 treatment to be captured as an AESI. Additionally, information regarding recommendations for further safety-related functional assessments follow-up for affected subjects have been added to the protocol to align with the Central Imaging Manual.

    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.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.nov for complete trial results.
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