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    EudraCT Number:2017-000146-21
    Sponsor's Protocol Code Number:AT1001-020
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-03-22
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2017-000146-21
    A.3Full title of the trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    ASPIRE Paediatric Fabry Study
    A.4.1Sponsor's protocol code numberAT1001-020
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03500094
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/328/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmicus Therapeutics, UK Ltd
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmicus Therapeutics, UK Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmicus Therapeutics, Inc
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address1 Cedar Brook Drive
    B.5.3.2Town/ cityCranbury
    B.5.3.3Post codeNJ 08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number001609662-2000
    B.5.5Fax number001609662-5010
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D. name Galafold
    D. of the Marketing Authorisation holderAmicus Therapeutics UK Ltd
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberE/3/06/368
    D.3 Description of the IMP
    D.3.1Product nameMigalastat
    D.3.2Product code AT1001
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNmigalastat
    D.3.9.1CAS number 75172-81-5
    D.3.9.2Current sponsor codeAT1001
    D.3.9.3Other descriptive nameMIGALASTAT HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB30354
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fabry disease and with amenable GLA variants
    E.1.1.1Medical condition in easily understood language
    Fabry's disease
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Stage 1:
    • to characterize the PK of migalastat in adolescents with Fabry disease and to validate extrapolation of migalastat plasma exposure in adults to adolescents weighing ≥ 45 kg for the 150 mg migalastat capsule administered every other day (QOD)
    • to evaluate the safety of migalastat treatment in pediatric subjects diagnosed with Fabry disease and who have variants in the gene encoding α galactosidase A (α Gal A) (GLA) amenable to treatment with migalastat
    Stage 2:
    • to evaluate the safety of migalastat treatment in pediatric subjects diagnosed with Fabry disease and who have GLA variants amenable to treatment with migalastat
    E.2.2Secondary objectives of the trial
    • to characterize the pharmacodynamics (PD) of migalastat in pediatric subjects diagnosed with Fabry disease and who have GLA variants amenable to treatment with migalastat
    • to evaluate the efficacy of migalastat in pediatric subjects diagnosed with Fabry disease and who have GLA variants amenable to treatment with migalastat
    • to evaluate the relationship between exposure to migalastat and response
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.have a parent or legally-authorized representative who is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
    2.male or female, diagnosed with Fabry disease aged between 12 and < 18 years at baseline, and who might benefit from specific treatment for their condition, in the opinion of the investigator
    3.confirmed amenable GLA variant determined using the migalastat amenability assay
    4.weight of ≥ 45 kg (99 pounds) at screening
    5.treatment-naïve or discontinued ERT treatment at least 14 days prior to screening
    6.have at least one complication (ie, historical or current laboratory abnormality and/or sign/symptom) of Fabry disease
    7.able to swallow capsules
    8.if of reproductive potential, agree to use medically accepted methods of contraception throughout the duration of the study and for up to 30 days after last dose of study medication
    E.4Principal exclusion criteria
    1.moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at screening)
    2.advanced kidney disease requiring dialysis or kidney transplantation
    3.history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
    4.subject has received any gene therapy at any time or anticipates starting gene therapy during the study period
    5.requires treatment with Glyset® (miglitol) or Zavesca® (miglustat), within 6 months before screening or throughout the study
    6.requires treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta) within 14 days before screening or throughout the study
    7.received any investigational/experimental drug, biologic or device within 30 days before screening
    8.any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
    9.is pregnant or breast-feeding, or is planning to become pregnant during the study period
    10.in the opinion of the investigator, the subject and/or parent or legally authorized representative is unlikely or unable to comply with the study requirements
    E.5 End points
    E.5.1Primary end point(s)
    1.incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug

    2.changes in clinical laboratory test results

    3.changes in vital signs

    4.changes in physical examination findings

    5.change in body weight and height percentile

    6.changes in ECG results

    7.changes in echocardiogram parameters

    8.change in Tanner stage

    9.use of concomitant medications

    10.population pharmacokinetics (popPK) model that describes the relationship between weight and age and migalastat pharmacokinetics in pediatric subjects

    11.popPK: Cmax :maximum observed plasma concentration

    12.popPK: Cmin : minimum observed plasma concentration

    13.popPK: tmax : time to reach Cmax

    14.popPK: AUC0-T: area under the plasma concentration-time curve from time 0 over the dosing interval (ie, 48 hours)

    15.popPK: t½ : terminal elimination half-life

    16.popPK: CLss/F : apparent oral clearance at steady-state concentration

    17.popPK: Vss/F concentration: apparent oral volume of distribution at steady-state
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Month 12

    2. baseline over time; up to 12 months

    3.baseline over time; up to 12 months

    4.baseline over time; up to 12 months

    5.baseline over time; up to 12 months

    6.baseline over time; up to 12 months

    7.baseline to Month 12/ET

    8.baseline to Month 12/ET

    9.baseline to Month 12/ET

    10. baseline to Month 12/ET

    11.Day 15-30, Months 6 and 12/ET

    12.Day 15-30, Months 6 and 12/ET

    13.Day 15-30, Months 6 and 12/ET

    14.Time Frame: Day 15-30, Months 6 and 12/ET

    15.Day 15-30, Months 6 and 12/ET

    16.popPK: Day 15-30, Months 6 and 12/ET

    17.popPK: Day 15-30, Months 6 and 12/ET
    E.5.2Secondary end point(s)
    1.change in plasma levels of lyso-Gb3

    2.change in eGFR

    3.change in urine protein and albumin levels

    4.change in LVMi and other echocardiogram parameters

    5.change in gastrointestinal signs and symptoms and pain, as measured by e-diary responses (Short FABPRO-GI and Pain Questionnaire)

    6.mean Patient Global Impression of Change (PGI-C) values

    7.change in FPHPQ scores

    8.change in PedsQL scores
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.baseline to Months 3, 6, and 12/ET

    2.baseline to Months 3, 6, and 12/ET

    3.baseline to Months 1, 3, 6, and 12/ET

    4.baseline to Month 12/ET

    5.baseline to Month 12/ET

    6.Months 3, 6 , 9 and 12/ET

    7.baseline to Month 12/ET

    8.baseline to Month 12/ET

    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of study is defined as date of database lock.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 20
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 20
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 20
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-04-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-29
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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