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    Clinical Trial Results:
    An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease

    Summary
    EudraCT number
    2014-002701-38
    Trial protocol
    AT   BE   GB   ES   DK  
    Global end of trial date
    23 Oct 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    06 Nov 2020
    First version publication date
    06 Nov 2020
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    AT1001-042
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02194985
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Amicus Therapeutics, Inc.
    Sponsor organisation address
    1 Cedar Brook Drive, Cranbury, NJ, United States, 08512
    Public contact
    Medical Affairs, Amicus Therapeutics, medinfo@amicusrx.com
    Scientific contact
    Medical Affairs, Amicus Therapeutics, medinfo@amicusrx.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
    26 Aug 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Oct 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Oct 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To assess long-term safety of migalastat hydrochloride (HCl) in the treatment of participants with Fabry disease who have completed treatment in a previous study of migalastat HCl.
    Protection of trial subjects
    This study was conducted in accordance with International Conference on Harmonisation (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which the study was conducted.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    14 Mar 2015
    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
    Spain: 1
    Country: Number of subjects enrolled
    Turkey: 3
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    United States: 25
    Country: Number of subjects enrolled
    Argentina: 1
    Country: Number of subjects enrolled
    Australia: 13
    Country: Number of subjects enrolled
    Austria: 2
    Country: Number of subjects enrolled
    Belgium: 1
    Country: Number of subjects enrolled
    Brazil: 3
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Denmark: 12
    Country: Number of subjects enrolled
    Egypt: 1
    Country: Number of subjects enrolled
    France: 4
    Country: Number of subjects enrolled
    Italy: 3
    Country: Number of subjects enrolled
    Japan: 5
    Worldwide total number of subjects
    84
    EEA total number of subjects
    30
    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)
    72
    From 65 to 84 years
    12
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    Participants entered this extension study immediately upon completion of their final treatment visit in a previous migalastat HCl study.

    Period 1
    Period 1 title
    Overall (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Arm title
    Migalastat HCl 150 mg
    Arm description
    Migalastat HCl 150 milligram (mg) was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).
    Arm type
    Experimental

    Investigational medicinal product name
    Migalastat HCl 150 mg
    Investigational medicinal product code
    Other name
    AT1001
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.

    Number of subjects in period 1
    Migalastat HCl 150 mg
    Started
    84
    Received at Least 1 Dose of Study Drug
    84
    Completed
    73
    Not completed
    11
         Physician decision
    4
         Consent withdrawn by subject
    2
         Adverse event, non-fatal
    1
         Met Protocol Defined Stopping Criteria
    3
         Lost to follow-up
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall
    Reporting group description
    All participants who took at least 1 dose of the study drug after they had enrolled into this study.

    Reporting group values
    Overall Total
    Number of subjects
    84 84
    Age categorical
    Units: Subjects
        In utero
    0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0
        Newborns (0-27 days)
    0 0
        Infants and toddlers (28 days-23 months)
    0 0
        Children (2-11 years)
    0 0
        Adolescents (12-17 years)
    0 0
        Adults (18-64 years)
    72 72
        From 65-84 years
    12 12
        85 years and over
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    51.9 ± 12.27 -
    Gender categorical
    Units: Subjects
        Female
    50 50
        Male
    34 34

    End points

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    End points reporting groups
    Reporting group title
    Migalastat HCl 150 mg
    Reporting group description
    Migalastat HCl 150 milligram (mg) was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).

    Subject analysis set title
    Safety Population
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study.

    Subject analysis set title
    Intent-to-Treat Population
    Subject analysis set type
    Intention-to-treat
    Subject analysis set description
    All participants who received at least 1 dose of study drug after they enrolled into this open-label extension study.

    Primary: Number Of Participants Experiencing Adverse Events (AEs)

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    End point title
    Number Of Participants Experiencing Adverse Events (AEs) [1]
    End point description
    An AE was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Adverse Events section.
    End point type
    Primary
    End point timeframe
    Day 1 (after dosing) to End of Study
    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: Quantitative statistical analysis was not performed for this end point; no treatment groups were compared in this single-group study.
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84
    Units: participants
    number (not applicable)
        Participants with at least 1 AE
    80
        Participants with at least 1 serious AE
    26
        Participants discontinued due to AEs
    1
        Participants with AEs leading to death
    0
        Participants with AEs related to study drug
    24
        Participants with AEs unrelated to study drug
    56
        Participants with at least 1 mild AE
    19
        Participants with at least 1 moderate AE
    46
        Participants with at least 1 severe AE
    15
    No statistical analyses for this end point

    Secondary: Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)

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    End point title
    Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
    End point description
    The annualized rate of change in the eGFR was assessed per participant by the slope of the simple linear regression between the observed values and the assessment times. It was calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (eGFR [CKD-EPI]) and the Modification of Diet in Renal Disease (MDRD) equation (eGFR [MDRD]). The equations are as follows: eGFR [MDRD] = 175 × (1/Serum Creatinine in mg/deciliter^1.154) × (1/Age in years^0.203) × 0.742 [if female] × 1.212 [if black] × 0.808 [if Japanese]; eGFR [CKD-EPI] = 141 × min(Serum creatinine [Scr]/k, 1)α × max(Scr/k, 1) - 1.209 × 0.993Age × 1.018 [if female] × 1.159 [if black], where Scr is serum creatinine, k is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1. Participants with at least a Baseline and a post-Baseline value are presented.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [2]
    Units: mL/min/1.73 m^2
    arithmetic mean (standard deviation)
        eGFR[MDRD]
    -1.6107 ± 5.62761
        eGFR[CKD-EPI]
    -1.3528 ± 4.84795
    Notes
    [2] - Intent-to-Treat (ITT) Population
    No statistical analyses for this end point

    Secondary: Change From Baseline In eGFR At End Of Study

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    End point title
    Change From Baseline In eGFR At End Of Study
    End point description
    The change from baseline in eGFR was calculated using eGFR[CKD-EPI]) and eGFR[MDRD]) equations. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [3]
    Units: mL/min/1.73 m^2
    arithmetic mean (standard deviation)
        Baseline eGFR[MDRD] (n=82)
    79.0 ± 22.56
        End of Study eGFR[MDRD] (n=73)
    75.8 ± 22.90
        Change from Baseline eGFR[MDRD] (n=72)
    -1.4 ± 10.62
        Baseline eGFR[CKD-EPI] (n=82)
    84.70 ± 23.092
        End of Study eGFR[CKD-EPI] (n=73)
    82.02 ± 23.890
        Change from Baseline eGFR[CKD-EPI] (n=72)
    -0.93 ± 9.828
    Notes
    [3] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study

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    End point title
    Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
    End point description
    Concentrations of lyso-Gb3 were measured in plasma using a qualified assay. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [4]
    Units: nmol/L
    arithmetic mean (standard deviation)
        Baseline (n=83)
    13.294 ± 17.5566
        End of Study (n=74)
    6.724 ± 7.5343
        Change from Baseline (n=74)
    -4.785 ± 9.0283
    Notes
    [4] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study

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    End point title
    Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study
    End point description
    The activity of the α-galactosidase A (α-Gal A) enzyme was measured in leukocyte lysate by a validated fluorometric assay method, using 4-methylumbelliferone as a reference. The activity values obtained were normalized to protein (measured using a colorimetric assay). Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Results for male participants are reported. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    34 [5]
    Units: nmol/hr/mg
    arithmetic mean (standard deviation)
        Baseline (n=27)
    6.882 ± 6.6857
        End of Study (n=30)
    5.290 ± 5.4054
        Change from Baseline (n=24)
    -1.375 ± 3.3432
    Notes
    [5] - Male participants in ITT population who had analyzable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In 24-hour Urine Protein To End Of Study

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    End point title
    Change From Baseline In 24-hour Urine Protein To End Of Study
    End point description
    A 24-hour urine sample was collected to measure 24-hour urine protein. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [6]
    Units: mg/day
    arithmetic mean (standard deviation)
        Baseline (n=83)
    478.9 ± 948.59
        End of Study (n=66)
    394.0 ± 547.77
        Change from Baseline (n=66)
    5.4 ± 233.49
    Notes
    [6] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In Left Ventricular Mass (LVM) To End Of Study

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    End point title
    Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
    End point description
    LVM was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [7]
    Units: gram
    arithmetic mean (standard deviation)
        Baseline (n=70)
    178.879 ± 78.6761
        End of Study (n=25)
    157.896 ± 47.9947
        Change from Baseline (n=24)
    -0.803 ± 18.8522
    Notes
    [7] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study

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    End point title
    Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
    End point description
    LVMi was measured by echocardiography. Baseline was defined as the data collected at Month 0, if not available, it was the last visit of the previous (feeder) study if done within 6 months. End of Study was the last recorded observation for each participant (approximately 30 days after last treatment). Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [8]
    Units: g/m^2
    arithmetic mean (standard deviation)
        Baseline (n=68)
    96.513 ± 36.5691
        End of Study (n=25)
    83.912 ± 22.7633
        Change from Baseline (n=68)
    -0.809 ± 11.8056
    Notes
    [8] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Secondary: Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire

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    End point title
    Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
    End point description
    The SF-36 is a participant self-rated questionnaire that is a general measure of perceived health status comprising 36 questions, which yields an 8-scale health profile (Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, and Mental Health). Scores on each item are summed and averaged (range: 0=worst to 100=best). Scores were normed to the US population. Higher score indicates less disability. A positive change from baseline indicates improvement. Baseline was defined as the data collected in the last visit of the previous (feeder) study. Only participants with both a Baseline value and an End of Study value were included in the change from baseline analysis.
    End point type
    Secondary
    End point timeframe
    Baseline, End of Study
    End point values
    Migalastat HCl 150 mg
    Number of subjects analysed
    84 [9]
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline Physical Functioning (n=84)
    48.313 ± 9.2345
        End of Study Physical Functioning (n=75)
    46.415 ± 10.2466
        Change from Baseline Physical Functioning (n=75)
    -1.276 ± 7.1627
        Baseline Role Physical (n=84)
    46.197 ± 10.0761
        End of Study Role Physical (n=75)
    45.929 ± 10.5723
        Change from Baseline Role Physical (n=75)
    0.390 ± 7.3500
        Baseline Bodily Pain (n=84)
    46.889 ± 10.5983
        End of Study Bodily Pain (n=75)
    46.737 ± 10.3368
        Change from Baseline Bodily Pain (n=75)
    0.425 ± 8.1342
        Baseline General Health (n=84)
    44.016 ± 10.2497
        End of Study General Health (n=75)
    42.352 ± 10.1185
        Change from Baseline General Health (n=75)
    -0.811 ± 5.8672
        Baseline Vitality (n=84)
    46.375 ± 11.8451
        End of Study Vitality (n=75)
    45.429 ± 12.4340
        Change from Baseline Vitality (n=75)
    -0.674 ± 8.5076
        Baseline Social Functioning (n=84)
    47.433 ± 10.4985
        End of Study Social Functioning (n=75)
    47.648 ± 9.5165
        Change from Baseline Social Functioning (n=75)
    0.401 ± 8.4356
        Baseline Role Emotional (n=84)
    47.547 ± 10.3897
        End of Study Role Emotional (n=75)
    46.141 ± 10.9054
        Change from Baseline Role Emotional (n=75)
    -0.929 ± 10.6679
        Baseline Mental Health (n=84)
    48.501 ± 10.6878
        End of Study Mental Health (n=75)
    49.578 ± 10.5732
        Change from Baseline Mental Health (n=75)
    0.872 ± 6.2528
        Baseline Physical Component (n=84)
    46.184 ± 10.2268
        End of Study Physical Component (n=75)
    44.825 ± 10.4292
        Change from Baseline Physical Component (n=75)
    -0.508 ± 6.2494
        Baseline Mental Component (n=84)
    47.917 ± 11.5480
        End of Study Mental Component (n=75)
    48.175 ± 10.9497
        Change from Baseline Mental Component (n=75)
    0.187 ± 8.2714
    Notes
    [9] - ITT population who had analysable data at the specified time points.
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline to up to 4.4 years (includes safety follow-up)
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    Migalastat HCl 150 mg
    Reporting group description
    Migalastat HCl 150 mg was administered orally once every other day for a median duration of 3.1 years (ranged from approximately 1 month to 4.3 years).

    Serious adverse events
    Migalastat HCl 150 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    26 / 84 (30.95%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Breast cancer
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Invasive lobular breast carcinoma
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Air embolism
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypertension
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypotension
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Device malfunction
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Drug hypersensitivity
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Uterine prolapse
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Pneumothorax
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Suicidal ideation
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Heart rate increased
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Rib fracture
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences causally related to treatment / all
    0 / 7
         deaths causally related to treatment / all
    0 / 0
    Atrioventricular block complete
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Left ventricular hypertrophy
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Ventricular tachycardia
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Convulsion
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Embolic stroke
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Headache
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Neuralgia
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Syncope
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Transient ischaemic attack
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Visual impairment
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Abdominal wall haematoma
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Barrett's oesophagus
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Large intestine perforation
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Biliary dyskinesia
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Subcutaneous emphysema
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal failure
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Arthritis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Osteoarthritis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Periarthritis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tendon calcification
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Appendicitis perforated
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Biliary sepsis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diverticulitis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infective exacerbation of bronchiectasis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    2 / 84 (2.38%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Diabetes mellitus
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Lipomatosis
         subjects affected / exposed
    1 / 84 (1.19%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Migalastat HCl 150 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    74 / 84 (88.10%)
    Vascular disorders
    Hypotension
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    16 / 84 (19.05%)
         occurrences all number
    22
    Oedema peripheral
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    18
    Pyrexia
         subjects affected / exposed
    9 / 84 (10.71%)
         occurrences all number
    12
    Pain
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    11
    Asthma
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Cough
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Dyspnoea
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    6
    Dyspnoea exertional
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Psychiatric disorders
    Depression
         subjects affected / exposed
    8 / 84 (9.52%)
         occurrences all number
    9
    Insomnia
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    6
    Investigations
    Albumin urine present
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    6
    Blood uric acid increased
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    6
    Protein urine present
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Blood creatinine increased
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Glomerular filtration rate decreased
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Injury, poisoning and procedural complications
    Overdose
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    18
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Nervous system disorders
    Paraesthesia
         subjects affected / exposed
    13 / 84 (15.48%)
         occurrences all number
    18
    Headache
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    18
    Dizziness
         subjects affected / exposed
    10 / 84 (11.90%)
         occurrences all number
    11
    Hypoaesthesia
         subjects affected / exposed
    10 / 84 (11.90%)
         occurrences all number
    17
    Migraine
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Neuralgia
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Ear and labyrinth disorders
    Tinnitus
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    8
    Vertigo
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    7
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    10 / 84 (11.90%)
         occurrences all number
    21
    Diarrhoea
         subjects affected / exposed
    9 / 84 (10.71%)
         occurrences all number
    15
    Abdominal pain
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    18
    Abdominal pain upper
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    8
    Constipation
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    7
    Dyspepsia
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Gastrooesophageal reflux disease
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    6
    Vomiting
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    12
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    16 / 84 (19.05%)
         occurrences all number
    23
    Pain in extremity
         subjects affected / exposed
    14 / 84 (16.67%)
         occurrences all number
    21
    Musculoskeletal pain
         subjects affected / exposed
    10 / 84 (11.90%)
         occurrences all number
    13
    Back pain
         subjects affected / exposed
    9 / 84 (10.71%)
         occurrences all number
    18
    Muscle spasms
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    7
    Muscular weakness
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    11
    Tendonitis
         subjects affected / exposed
    7 / 84 (8.33%)
         occurrences all number
    7
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    17 / 84 (20.24%)
         occurrences all number
    29
    Upper respiratory tract infection
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    20
    Urinary tract infection
         subjects affected / exposed
    12 / 84 (14.29%)
         occurrences all number
    17
    Influenza
         subjects affected / exposed
    10 / 84 (11.90%)
         occurrences all number
    14
    Sinusitis
         subjects affected / exposed
    9 / 84 (10.71%)
         occurrences all number
    10
    Bronchitis
         subjects affected / exposed
    6 / 84 (7.14%)
         occurrences all number
    9
    Metabolism and nutrition disorders
    Hypercholesterolaemia
         subjects affected / exposed
    5 / 84 (5.95%)
         occurrences all number
    5

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Oct 2015
    • Revised the lowest age limit for enrollment from 16 to 18 years of age. The participants under the age of 18 were allowed to enroll at sites with required relevant regulatory and ethics approvals. • Added a new stopping criterion: An eGFR value of < 30 mL/min/1.73 m^2. • Removed a requirement to collect historical (up to 5-year) Fabry disease data from the participants. • Revised contraception requirements for both male and female participants, clarifying that contraception methods must be medically accepted, and abstinence was not allowed.

    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
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    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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