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    Clinical Trial Results:
    A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]

    Summary
    EudraCT number
    		 2016-002109-20
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    06 Apr 2015

    Results information
    Results version number
    		 v2(current)
    This version publication date
    15 Feb 2017
    First version publication date
    05 Jan 2017
    Other versions
    		 v1
    Version creation reason
    • Changes to summary attachments
    This update includes a revised synopsis of the clinical study report AAV2-hRPE65v2-301, 13 December 2016. The Amendment 1 has been mainly prepared in order to clarify the study completion date, i.e., the last patient last visit date, 6 April 2015, vs. the final data collection date for primary outcome measure, i.e., the data cut-off date, 16 July 2015. The updates ensured that the date of study completion is aligned with the individual case report forms (CRF).
    Summary report(s)
    		 Clinical Study Report Synopsis

    Trial information

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    Trial identification
    Sponsor protocol code
    AAV2-hRPE65v2-301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT00999609
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Spark Therapeutics, Inc.
    Sponsor organisation address
    3737 Market St, Suite 1300, Philadelphia, PA, United States, 19104
    Public contact
    Head of Clinical Research and Development, Spark Therapeutics, Inc., 001 888-772-7560, clinicaltrials@sparktx.com
    Scientific contact
    Head of Clinical Research and Development, Spark Therapeutics, Inc., 001 888-772-7560, clinicaltrials@sparktx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001684-PIP01-14
    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
    09 Sep 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    06 Apr 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Apr 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to determine whether non-simultaneous, bilateral, subretinal administration of AAV2-hRPE65v2 improves the ability to navigate as measured by mobility testing in adults and children, three years of age or older. Mobility test performance one year following vector administrations was compared to subjects’ pre-administration, baseline mobility test performance; independent, masked reviewers were trained to assess ability to navigate.
    Protection of trial subjects
    Reviewed trial documents and approved initiation of trial, evaluated progress of trial, made recommendations (as appropriate) to Sponsor, Investigators, IRB, medical monitor regarding continuation/termination of trial based on observed beneficial or adverse effects of the intervention and data review. The Committee included 5 members with expertise in the clinical area and/or clinical trial methodology. Met every 6 months while actively enrolling and then schedule based on safety events and study milestones. All Adverse Events reports and changes to protocol were reviewed by the Committee.
    Background therapy
    		 None
    Evidence for comparator
    		 None; no currently available treatment.
    Actual start date of recruitment
    15 Nov 2012
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety
    Long term follow-up duration
    		 15 Years
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 31
    Worldwide total number of subjects
    31
    EEA total number of subjects
    0
    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)
    17
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    11
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Potential subjects were recruited from inherited retinal disease centres at two university-based sites (Children’s Hospital of Philadelphia and University of Iowa). Subjects were to be aged 3 years or older and have confirmed RPE65 mutations.

    Pre-assignment
    Screening details
    		 Screening included informed consent process, medical and visual history, prior medications, screening for HIV, screening of RPE65 mutations (if adequate records were not available), urine pregnancy test for females of reproductive age, ophthalmic exams with optical coherence tomography (OCT), mobility testing, visual acuity, visual field testing.

    Pre-assignment period milestones
    Number of subjects started
    		 36 [1]
    Intermediate milestone: Number of subjects
    Screened: 36
    Intermediate milestone: Number of subjects
    Randomized: 31
    Number of subjects completed
    		 31

    Pre-assignment subject non-completion reasons
    Reason: Number of subjects
    		 Screen failure: 5
    Notes
    [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Out of 36 subjects screened five subjects were considered to be ineligible and were not included in the study.
    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded
    Blinding implementation details
    Masked, independent reviewers graded subjects’ mobility testing videos, without access to randomization information or any other retinal/visual function test results. The sequence of videos assessments, performed at multiple visits, was also be masked so that the graders did not know whether the video they graded was a baseline evaluation or a follow-up evaluation for any given subject.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Intervention
    Arm description
    		 AAV2-hRPE65v2 injected to each eye separately
    Arm type
    		 Experimental

    Investigational medicinal product name
    Adeno-associated viral vector serotype 2 containing the human RPE65 gene
    Investigational medicinal product code
    AAV2-hRPE65v2
    Other name
    voretigene neparvovec (INN)
    Pharmaceutical forms
    Concentrate and solvent for solution for injection
    Routes of administration
    Subretinal use
    Dosage and administration details
    1.5E11 vector genomes in a volume of 0.3 mL delivered by subretinal injection to each eye sequentially, no more than 18 days apart

    Arm title
    		 Control
    Arm description
    		 No intervention, no sham; uninjected control group.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Intervention Control
    Started
    21
    10
    Screened
    21
    10
    Randomized
    21
    10
    Completed 1 year assessments
    20
    9
    Completed
    20
    9
    Not completed
    1
    1
         Physician decision
    1
    -
         Consent withdrawn by subject
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Intervention
    Reporting group description
    		 AAV2-hRPE65v2 injected to each eye separately

    Reporting group title
    Control
    Reporting group description
    		 No intervention, no sham; uninjected control group.

    Reporting group values
    		 Intervention Control Total
    Number of subjects
    		 21 10 31
    Age categorical
    Subject age at randomization
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 12 5 17
        Adolescents (12-17 years)
    		 3 0 3
        Adults (18-64 years)
    		 6 5 11
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14.7 ( 11.8 ) 15.9 ( 9.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 12 6 18
        Male
    		 9 4 13
    Race
    Units: Subjects
        White
    		 14 7 21
        Asian
    		 3 2 5
        American Indian or Alaska Native
    		 2 1 3
        Black or African American
    		 2 0 2
    Subject analysis sets

    Subject analysis set title
    Efficacy Analysis
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    All randomized subjects

    Subject analysis sets values
    		 Efficacy Analysis
    Number of subjects
    31
    Age categorical
    Subject age at randomization
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    17
        Adolescents (12-17 years)
    3
        Adults (18-64 years)
    11
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    15.1 ( 10.9 )
    Gender categorical
    Units: Subjects
        Female
    18
        Male
    13
    Race
    Units: Subjects
        White
    21
        Asian
    5
        American Indian or Alaska Native
    3
        Black or African American
    2

    End points

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    End points reporting groups
    Reporting group title
    Intervention
    Reporting group description
    		 AAV2-hRPE65v2 injected to each eye separately

    Reporting group title
    Control
    Reporting group description
    		 No intervention, no sham; uninjected control group.

    Subject analysis set title
    Efficacy Analysis
    Subject analysis set type
    		 Intention-to-treat
    Subject analysis set description
    All randomized subjects

    Primary: Mobility Testing (Bilateral)

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    End point title
    		 Mobility Testing (Bilateral)
    End point description
    The standardized mobility test measures the ability to navigate a randomly selected course layout at different levels of environmental illumination and relates to the subject’s extent of visual field and light sensitivity, as well as visual acuity.
    End point type
    Primary
    End point timeframe
    One year (change from baseline)
    End point values
    		 Intervention Control
    Number of subjects analysed
    21
    10
    Units: Bilateral mobility test change score
        arithmetic mean (standard deviation)
    1.8 ( 1.1 )
    0.2 ( 1 )
    Statistical analysis title
    		 Bilateral Mobility Test Change Score
    Statistical analysis description
    Mean change in bilateral mobility testing change score from baseline to 1 year, compared between intervention and control groups
    Comparison groups
    Control v Intervention
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.001 [1]
    Method
    		 Permutation test
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.72
         upper limit
    		 2.41
    Variability estimate
    Standard deviation
    Dispersion value
    1.07
    Notes
    [1] - Method: permutation test based on Wilcoxon rank-sum.

    Secondary: Full-field light sensitivity threshold (FST) testing: white light

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    End point title
    		 Full-field light sensitivity threshold (FST) testing: white light
    End point description
    Measurable units: Change in Log 10 (cd.s/m2) Log 10 (candela seconds per meter squared).
    End point type
    Secondary
    End point timeframe
    One year (change from baseline)
    End point values
    		 Intervention Control
    Number of subjects analysed
    21
    10
    Units: Change in Log 10 (cd.s/m2)
        arithmetic mean (standard deviation)
    -2.08 ( 0.29 )
    0.04 ( 0.44 )
    Statistical analysis title
    		 Full-field Light Sensitivity Threshold Testing
    Statistical analysis description
    Full-field Light Sensitivity Threshold Testing: White Light. Mean change in full-field light sensitivity threshold testing (averaged over both eyes) from baseline to 1 year, compared between intervention and control groups.
    Comparison groups
    Intervention v Control
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 Mixed models analysis
    Parameter type
    		 Mean difference (net)
    Point estimate
    -2.11
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.19
         upper limit
    		 -1.04
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.52

    Secondary: Monocular Mobility Testing

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    End point title
    		 Monocular Mobility Testing
    End point description
    Measures the ability to navigate the mobility test using only the assigned first eye
    End point type
    Secondary
    End point timeframe
    One year (change from baseline)
    End point values
    		 Intervention Control
    Number of subjects analysed
    21
    10
    Units: Mobility test change score
        arithmetic mean (standard deviation)
    1.9 ( 1.2 )
    0.2 ( 0.6 )
    Statistical analysis title
    		 Monocular Mobility Testing
    Statistical analysis description
    Mean change in monocular mobility testing change score from baseline to 1 year, compared between intervention and control groups
    Comparison groups
    Intervention v Control
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority [2]
    P-value
    		 = 0.001 [3]
    Method
    		 Permutation test
    Parameter type
    		 Mean difference (final values)
    Point estimate
    1.7
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.89
         upper limit
    		 2.52
    Variability estimate
    Standard deviation
    Dispersion value
    0.89
    Notes
    [2] - Parameter type: permutation test based on Wilcoxon rank-sum.
    [3] - Method: permutation test based on Wilcoxon rank-sum

    Secondary: Visual Acuity

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    End point title
    		 Visual Acuity
    End point description
    Measurement of the sharpness of vision, determined by the ability to read letters on a standardized chart from a specified distance. Measured as a change in Logarithm of the minimum angle of resolution (LogMAR).
    End point type
    Secondary
    End point timeframe
    One year (change from baseline)
    End point values
    		 Intervention Control
    Number of subjects analysed
    21
    10
    Units: Change in LogMAR
        arithmetic mean (standard deviation)
    -0.16 ( 0.07 )
    0.01 ( 0.1 )
    Statistical analysis title
    		 Visual Acuity
    Statistical analysis description
    Mean change in visual acuity from baseline to 1 year, compared between intervention and control groups.
    Comparison groups
    Intervention v Control
    Number of subjects included in analysis
    31
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.17
    Method
    		 Longitudinal repeated measures model
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.16
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.41
         upper limit
    		 0.08
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.12

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From 15 November 2012 to 6 April 2015
    Adverse event reporting additional description
    The safety population (n=29) includes all subjects who received injection in either eye for the intervention group and all control group subjects who did not withdraw, or were not withdrawn, prior to any of the following people knowing the treatment assignment: the subject, parent, Principal Investigator, or Medical Monitor.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    14
    Reporting groups
    Reporting group title
    Intervention
    Reporting group description
    		 Intervention arm

    Reporting group title
    Control
    Reporting group description
    		 Control arm

    Serious adverse events
    		 Intervention Control
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Nervous system disorders
    Convulsion
    Additional description: Associated with pre-existing complex seizure disorder.
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Adverse drug reaction
    Additional description: Associated with pre-existing complex seizure disorder and complications of oral surgery, respectively.
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 3%
    Non-serious adverse events
    		 Intervention Control
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    20 / 20 (100.00%)
    9 / 9 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Oral fibroma
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Chills
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Facial pain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Fatigue
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    7 / 20 (35.00%)
    1 / 9 (11.11%)
         occurrences all number
    9
    2
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    3
    0
    Menometrorrhagia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Menstruation irregular
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    6 / 20 (30.00%)
    1 / 9 (11.11%)
         occurrences all number
    9
    1
    Dyspnoea
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Epistaxis
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Nasal congestion
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Oropharyngeal pain
         subjects affected / exposed
    6 / 20 (30.00%)
    4 / 9 (44.44%)
         occurrences all number
    6
    4
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Attention deficit/hyperactivity disorder
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Emetophobia
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Insomnia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Blood alkaline phosphatase increased
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Blood cholesterol increased
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Blood pressure increased
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Electrocardiogram T wave inversion
    Additional description: Related to the administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Intraocular pressure increased
    Additional description: Related to the administration procedure in 3 subjects
         subjects affected / exposed
    4 / 20 (20.00%)
    0 / 9 (0.00%)
         occurrences all number
    5
    0
    Weight decreased
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    Injury, poisoning and procedural complications
    Animal bite
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Ankle fracture
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Excoriation
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Eye injury
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Foot fracture
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Joint sprain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Laceration
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Muscle strain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Headache
    Additional description: Related to administration procedure in one subject
         subjects affected / exposed
    7 / 20 (35.00%)
    2 / 9 (22.22%)
         occurrences all number
    15
    5
    Migraine
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Presyncope
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Syncope
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Blood and lymphatic system disorders
    Leukocytosis
    Additional description: Related to use of systemic steroids
         subjects affected / exposed
    9 / 20 (45.00%)
    0 / 9 (0.00%)
         occurrences all number
    23
    0
    Eye disorders
    Cataract
    Additional description: Related to administration procedure
         subjects affected / exposed
    3 / 20 (15.00%)
    0 / 9 (0.00%)
         occurrences all number
    4
    0
    Conjunctival Cyst
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye inflammation
    Additional description: Related to administration procedure
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    6
    0
    Eye irritation
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye pain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye pruritus
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Eye swelling
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Foreign body sensation in eyes
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Iritis
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Macular degeneration
    Additional description: Macular thinning following non-surgical closure of macular hole (below). Related to administration procedure.
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Macular hole
    Additional description: Resolved to macular thinning (above). Related to administration procedure.
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Maculopathy
    Additional description: Bilateral epiretinal membrane. Related to administration procedure.
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Photopsia
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Pseudopapilloedema
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Retinal haemorrhage
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Retinal tear
    Additional description: Related to administration procedure
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Gastrointestinal disorders
    Abdominal distension
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Abdominal pain upper
         subjects affected / exposed
    2 / 20 (10.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Bowel movement irregularity
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Constipation
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Diarrhoea
         subjects affected / exposed
    2 / 20 (10.00%)
    1 / 9 (11.11%)
         occurrences all number
    2
    1
    Gastritis
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Gastroesophageal reflux disease
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Lip pain
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Nausea
    Additional description: Related to administration procedure in one subject
         subjects affected / exposed
    6 / 20 (30.00%)
    1 / 9 (11.11%)
         occurrences all number
    9
    1
    Vomiting
    Additional description: Related to administration procedure in one subject
         subjects affected / exposed
    8 / 20 (40.00%)
    2 / 9 (22.22%)
         occurrences all number
    9
    6
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Eczema
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Rash
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Swelling face
    Additional description: Related to administration procedure
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    3 / 20 (15.00%)
    1 / 9 (11.11%)
         occurrences all number
    3
    1
    Urine abnormality
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Musculoskeletal pain
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Neck pain
         subjects affected / exposed
    0 / 20 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    Infections and infestations
    Conjunctivitis viral
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Ear infection
         subjects affected / exposed
    1 / 20 (5.00%)
    1 / 9 (11.11%)
         occurrences all number
    1
    1
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Nasopharyngitis
         subjects affected / exposed
    7 / 20 (35.00%)
    2 / 9 (22.22%)
         occurrences all number
    9
    2
    Pharyngitis streptococcal
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    2
    0
    Sinusitis
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 20 (10.00%)
    3 / 9 (33.33%)
         occurrences all number
    2
    3
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    Hypoglycaemia
         subjects affected / exposed
    1 / 20 (5.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    13 Jan 2014
    Sponsorship of the trial was changed from the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia to Spark Therapeutics, Inc. This change was described in the 20 August 2013 clinical protocol.

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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