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    Clinical Trial Results:
    A Cross-sectional Study of Renal Function in Treatment-naïve, Young Male Patients with Fabry Disease

    Summary
    EudraCT number
    2012-001966-14
    Trial protocol
    ES  
    Global end of trial date
    17 Aug 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    02 Mar 2017
    First version publication date
    02 Mar 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AGAL19110
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01839526
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Genzyme Corporation
    Sponsor organisation address
    500 Kendall Street, Cambridge, MA, United States, 02142
    Public contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    02 Nov 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    17 Aug 2016
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To document renal function and other Fabry disease manifestations across age in treatment-naïve, young male subjects with Fabry disease.
    Protection of trial subjects
    The study was conducted by investigators experienced in the treatment of pediatric subjects. The parent(s) or guardian(s) as well as the children were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time. In addition to the consent form for the parent(s)/guardian(s), an assent form in child-appropriate language was provided and explained to the child. Repeated invasive procedures were minimized. The number of blood samples as well as the amount of blood drawn were adjusted according to age and weight. A topical anesthesia may have been used to minimize distress and discomfort.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 May 2013
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Argentina: 3
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Austria: 1
    Country: Number of subjects enrolled
    Brazil: 2
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 3
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Finland: 1
    Country: Number of subjects enrolled
    France: 3
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Norway: 4
    Country: Number of subjects enrolled
    Poland: 5
    Country: Number of subjects enrolled
    United States: 8
    Worldwide total number of subjects
    39
    EEA total number of subjects
    23
    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)
    16
    Adolescents (12-17 years)
    12
    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
    The study was conducted at 19 centers in 13 countries between 13 May 2013 and 17 August 2016. A total of 45 subjects were screened, of whom 6 were screen failures. Screen failures were due to failure to meet inclusion criteria.

    Pre-assignment
    Screening details
    Out of 45 screened subjects, 39 subjects were enrolled in the study.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Arm title
    Fabry Disease - All Subjects
    Arm description
    All male subjects, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.
    Arm type
    No intervention

    Investigational medicinal product name
    Non interventional medicinal product (NIMP): Iohexol
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Small dose of iohexol was administered for determining glomerular filtration rate (GFR).

    Number of subjects in period 1
    Fabry Disease - All Subjects
    Started
    39
    Completed
    39

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Period
    Reporting group description
    All male subjects, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.

    Reporting group values
    Overall Period Total
    Number of subjects
    39 39
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    13.6 ± 6.3 -
    Gender categorical
    Units: Subjects
        Female
    0 0
        Male
    39 39

    End points

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    End points reporting groups
    Reporting group title
    Fabry Disease - All Subjects
    Reporting group description
    All male subjects, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.

    Primary: Fabry Disease Parameter: GFR Estimated from Serum Creatinine (eGFR)

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    End point title
    Fabry Disease Parameter: GFR Estimated from Serum Creatinine (eGFR) [1]
    End point description
    GFR was assessed by estimation from serum creatinine level (eGFR) using an age-appropriate method based on the subject's age at the screening visit (for the purpose of determining a subject’s eligibility for measurement of iohexol glomerular filtration rate [iGFR]) and at the clinical investigational visit (for use in statistical analyses). For subjects <18 years old, the Bedside Schwartz equation was used to calculate eGFR, while for subjects >18 years old, the CKD-EPI equation was used. Analysis was performed on safety population which included all subjects who signed the informed consent and deemed eligible to enroll based on screening assessments. Number of subjects analyzed = subjects with available data for this point. Here, 'n' signifies number of subjects with available data for specified category.
    End point type
    Primary
    End point timeframe
    Screening, Day 1
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the endpoint is descriptive in nature, no statistical analysis is provided.
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: mL/minute/1.73m^2
    arithmetic mean (standard deviation)
        Screening (n=39)
    113.9 ± 20.1
        Day 1 (n=37)
    115.8 ± 22.7
    No statistical analyses for this end point

    Primary: Fabry Disease Parameter: Glomerular Filtration Rate (GFR) Measured Using Plasma Iohexol Clearance (iGFR)

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    End point title
    Fabry Disease Parameter: Glomerular Filtration Rate (GFR) Measured Using Plasma Iohexol Clearance (iGFR) [2]
    End point description
    GFR was calculated by plasma iohexol clearance after the required medication washout. Analysis was performed on safety population. Number of subjects analyzed = subjects with available data for this endpoint.
    End point type
    Primary
    End point timeframe
    Day 1 onwards up to safety follow-up phone contact (up to Day 28)
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the endpoint is descriptive in nature, no statistical analysis is provided.
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    38
    Units: mL/min/1.73^2
        arithmetic mean (standard deviation)
    105.3 ± 21.5
    No statistical analyses for this end point

    Primary: Fabry Disease Parameter: Protein Excretion Assessed from Three First-Morning Urine Voids

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    End point title
    Fabry Disease Parameter: Protein Excretion Assessed from Three First-Morning Urine Voids [3]
    End point description
    Protein excretion was evaluated from the 3 first morning urine voids, each obtained at least 1 week apart and not more than 2 weeks apart. Mean value for each parameter was calculated and reported. Parameters determined for each urine sample were albumin; total protein; creatinine (for calculation of albumin:creatinine ratio [ACR] and protein-creatinine ratio [PCR]); retinol binding protein (RBP); and Beta-2 microglobulin. Analysis was performed on safety population. Number of subjects analyzed = subjects with available data for this endpoint.
    End point type
    Primary
    End point timeframe
    Day 1 after the medication washout up to follow-up phone contact (Day28)
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: As the endpoint is descriptive in nature, no statistical analysis is provided.
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: specified in categories
    arithmetic mean (standard deviation)
        Albumin (mg/dL)
    4.6 ± 18.3
        Albumin/Creatinine (mg/g)
    56.3 ± 270.9
        Beta-2 microglobulin (mg/L)
    0.1 ± 0.2
        Creatinine (mmol/L)
    10.4 ± 4.7
        Protein (mg/dL)
    9.6 ± 20.9
        Protien/Creatinine (mg/g)
    98.2 ± 294.6
        Retinol binding protein (ug/L)
    110.1 ± 72.9
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Electrocardiogram (ECG) Parameters

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    End point title
    Fabry Disease Parameter: Electrocardiogram (ECG) Parameters
    End point description
    ECG parameters included heart rate, PR interval, QRS interval, QT interval, RR interval, QTcB interval and QTcF interval. PR interval is the time on ECG tracing from the start of the P wave to the start of the R wave. PR interval represents the time from the onset of atrial depolarization until the onset of ventricular depolarization. QRS interval is the time from the start of the Q wave to the end of S wave. QRS interval represents depolarization of the ventricular myocardium. QT interval is a measure of the time between the start of the Q wave and the end of the T wave and represents electrical depolarization and repolarization of the ventricles. The QTcB includes a Bazett’s correction factor for changes in heart rate and QTcF includes a Fredericia’s correction factor for changes in heart rate. The RR interval is the time between QRS complexes. Analysis was performed on safety population. Number of subjects analyzed = subjects with available data for this endpoint.
    End point type
    Secondary
    End point timeframe
    Day 1 onwards up to safety follow-up phone contact (up to Day 28)
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    35
    Units: specified in the categories
    arithmetic mean (standard deviation)
        Heart rate (beats/minute)
    71.06 ± 13.57
        PR interval (msec)
    137.66 ± 21.65
        QRS interval (msec)
    83.77 ± 9.16
        QT interval (msec)
    367.89 ± 31.77
        RR interval (msec)
    873.94 ± 166.25
        QTcB interval (msec)
    396.46 ± 26.36
        QTcF interval (msec)
    386.31 ± 23.02
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Number of Subjects with Clinically Significant Echocardiogram Findings

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    End point title
    Fabry Disease Parameter: Number of Subjects with Clinically Significant Echocardiogram Findings
    End point description
    Analysis was performed on safety population.
    End point type
    Secondary
    End point timeframe
    Day 1 onwards up to safety follow-up phone contact (up to Day 28)
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: Subjects
        Normal
    34
        Abnormal, but not clinically significant
    3
        Abnormal, Clinically significant
    1
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Number of Subjects with Gastrointestinal (GI) Symptoms at Screening

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    End point title
    Fabry Disease Parameter: Number of Subjects with Gastrointestinal (GI) Symptoms at Screening
    End point description
    GI symptoms were collected at the screening visit by asking the subject-specific questions about the following GI symptoms: abdominal pain; diarrhea; nausea; vomiting (subjects who answered "yes" were reported); maximum number of bowel movements per day in the past week; stool consistency on average in the past week; severity of abdominal pain on average in the past week; severity of bloating on average in the past week. Analysis was performed on safety population.
    End point type
    Secondary
    End point timeframe
    At screening
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: Subjects
        Abdominal pain
    20
        Diarrhea
    17
        Nausea
    8
        Vomiting
    7
        Number of bowel movements per day (1-2)
    26
        Number of bowel movements per day (3-4)
    2
        Number of bowel movements per day (5-7)
    3
        Number of bowel movements per day (8-10)
    0
        Number of bowel movements per day (>10)
    0
        Number of bowel movements per day (unknown)
    8
        Stool consistency on average (very hard)
    0
        Stool consistency on average (hard)
    1
        Stool consistency on average (formed)
    28
        Stool consistency on average (loose)
    7
        Stool consistency on average (watery)
    0
        Stool consistency on average (unknown)
    3
        Severity of abdominal pain on average (mild)
    4
        Severity of abdominal pain on average (moderate)
    5
        Severity of abdominal pain on average (severe)
    0
        Severity of abdominal pain on average (extreme)
    1
        Severity of abdominal pain on average (unknown)
    1
        Severity of abdominal pain on average (none)
    28
        Severity of bloating on average (mild)
    3
        Severity of bloating on average (moderate)
    4
        Severity of bloating on average (severe)
    2
        Severity of bloating on average (extreme)
    0
        Severity of bloating on average (unknown)
    2
        Severity of bloating on average (none)
    28
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Quality of Life assessed by Pediatric Pain Questionnaire (PedsQL) Scores

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    End point title
    Fabry Disease Parameter: Quality of Life assessed by Pediatric Pain Questionnaire (PedsQL) Scores
    End point description
    PedsQL completed by subjects between 5 and 17 years of age. It comprises of 2 questions:‘How do you feel now’ and ‘Worst pain this week’, each measured on a 0-10 cm VAS scale (0=no pain; 10=severe pain). Analysis was performed on safety population. Number of subjects analyzed = subjects with available data for this endpoint.
    End point type
    Secondary
    End point timeframe
    At screening
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    28
    Units: milimeters
    median (full range (min-max))
        How you feel now
    0 (0 to 42)
        Worst pain this week
    1.5 (0 to 83)
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Quality of Life assessed by Brief Pain Inventory Short Form (BPI [SF]) Scores

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    End point title
    Fabry Disease Parameter: Quality of Life assessed by Brief Pain Inventory Short Form (BPI [SF]) Scores
    End point description
    BPI [SF] was completed by subjects >=18 years of age. BPI (SF) comprises of 2 dimensions of pain: severity/intensity (4 items: worst, least, average and now pain) and interference with daily function (7 items: pain interference with general activity, walking, work, mood, enjoyment of life, relations with others and sleep). Each item was rated by subject on a scale ranges from 0-10, where lower scores indicated less pain. In this endpoint, average pain reported by subjects in last 24 hours along with the mean pain interference was reported. Analysis was performed on safety population. Number of subjects analyzed=subjects with available data for this endpoint.
    End point type
    Secondary
    End point timeframe
    At screening
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    11
    Units: units on a scale
    arithmetic mean (standard deviation)
        BPI (SF): Average pain in last 24 hours
    2.5 ± 2
        BPI (SF): Mean pain interfered
    0.909 ± 1.344
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Plasma Globotriaosylceramide (GL-3) Concentrations

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    End point title
    Fabry Disease Parameter: Plasma Globotriaosylceramide (GL-3) Concentrations
    End point description
    Accumulation of Globotriaosylceramide in blood was evaluated by measuring levels of total GL-3 and lyso-GL-3 using tandem mass spectrometry. Blood samples were collected for all subjects and obtained either before or after the medication washout. Analysis was performed on safety population.
    End point type
    Secondary
    End point timeframe
    Day 1 onwards up to safety follow-up phone contact (up to Day 28)
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: units specified in the categories
    arithmetic mean (standard deviation)
        GL-3 (microgram/mL)
    13.5 ± 6.8
        Lyso-GL-3 (nanogram/mL)
    144.2 ± 97.7
    No statistical analyses for this end point

    Secondary: Fabry Disease Parameter: Urine Globotriaosylceramide (GL-3) Concentrations

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    End point title
    Fabry Disease Parameter: Urine Globotriaosylceramide (GL-3) Concentrations
    End point description
    Accumulation of Globotriaosylceramide concentration in urine was evaluated by measuring levels of total GL-3 and lyso-GL-3 using tandem mass spectrometry. Urine samples were obtained either before or after the medication washout. Analysis was performed on safety population.
    End point type
    Secondary
    End point timeframe
    Day 1 up to Day 28 (Before or after the medication washout)
    End point values
    Fabry Disease - All Subjects
    Number of subjects analysed
    39
    Units: units specified in the categories
    arithmetic mean (standard deviation)
        GL-3 (miligram/milimoles Cr)
    0.34 ± 0.27
        Lyso-GL-3 (nanogram/milimoles Cr)
    83.9 ± 107.1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    All Adverse Events (AE) were collected from signature of the informed consent form up to the final visit (Day 28) regardless of seriousness or relationship to investigational product.
    Adverse event reporting additional description
    AEs in the study subject population would be expected to be related to Fabry or concomitant diseases, or related to procedures (i.e., iohexol). All subjects were observed for approximately 4 weeks after iohexol administration. All AEs from the time of written informed consent through completion of the safety follow-up phone contact were reported.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    ed 19.0
    Reporting groups
    Reporting group title
    All Subjects: Age range (5 to <12 years)
    Reporting group description
    All male subjects in the age range of 5 to <12 years, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.

    Reporting group title
    All Subjects: Age range (>=18 years)
    Reporting group description
    All male subjects in the age of >=18 years, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.

    Reporting group title
    All Subjects: Age range (12 to <18 years)
    Reporting group description
    All male subjects in the age range of 12 to <18 years, with Fabry disease and who had not received any interventional treatment for the disease within 30 days of screening. They were observed in this cross-sectional study.

    Serious adverse events
    All Subjects: Age range (5 to <12 years) All Subjects: Age range (>=18 years) All Subjects: Age range (12 to <18 years)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    All Subjects: Age range (5 to <12 years) All Subjects: Age range (>=18 years) All Subjects: Age range (12 to <18 years)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 16 (18.75%)
    2 / 11 (18.18%)
    4 / 12 (33.33%)
    Cardiac disorders
    Dilatation Ventricular
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Left Atrial Dilatation
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Epistaxis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Paraesthesia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Pyrexia
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Vomiting
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    2 / 12 (16.67%)
         occurrences all number
    0
    0
    2
    Skin and subcutaneous tissue disorders
    Erythema
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Pain In Extremity
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Metabolism and nutrition disorders
    Vitamin D Deficiency
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Ear Infection Fungal
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Sinusitis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 11 (0.00%)
    0 / 12 (0.00%)
         occurrences all number
    1
    0
    0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    0 / 16 (0.00%)
    1 / 11 (9.09%)
    0 / 12 (0.00%)
         occurrences all number
    0
    1
    0
    Urinary Tract Infection
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 11 (0.00%)
    1 / 12 (8.33%)
         occurrences all number
    0
    0
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Apr 2012
    -Clarified that both a medical/surgical history and a Fabry specific history was required in the subjects. -Added a physical examination at the clinical investigation visit and clarified that the physical examination at screening include an assessment of angiokeratomas and Tanner stage. -Added respiratory rate and temperature to the vital signs assessment. -Clarified that laboratory samples were collected at the screening visit. -Added a table describing the Tanner Stages for reference. -Reflected changes in company ownership, study team changes and department name changes.

    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.
    Enrollment period not extended and screening stopped for slow recruitment and in accordance with provisions of the protocol. Not linked to any safety concern.
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