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    Summary
    EudraCT Number:2018-001133-40
    Sponsor's Protocol Code Number:AT1001-025
    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:2020-05-14
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2018-001133-40
    A.3Full title of the trial
    An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Migalastat in Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment
    A.4.1Sponsor's protocol code numberAT1001-025
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/174/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmicus Therapeutics, Inc.
    B.1.3.4CountryUnited States
    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, Inc.
    B.4.2CountryUnited States
    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 number1609 662 2000
    B.5.5Fax number1609 662 5010
    B.5.6E-mailclinicaltrials@amicusrx.com
    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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/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.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee 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 Subjects with Amenable GLA Variants and Severe Renal Impairment
    E.1.1.1Medical condition in easily understood language
    Fabry disease with severe kidney 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
    To characterize the pharmacokinetics (PK) of migalastat and validate the population PK (popPK) model and simulations for dose selection in Fabry subjects with severe renal impairment
    E.2.2Secondary objectives of the trial
    • To assess the safety and tolerability of migalastat in Fabry subjects with severe renal impairment
    • To evaluate the pharmacodynamics (PD) of migalastat in subjects with Fabry disease and severe renal impairment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects aged 16 years or older, diagnosed with Fabry disease.
    2. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information or research-related health information or subject has a legally authorized representative who has provided written informed consent and authorization, and provides assent, if applicable.
    3. Subject has a GLA variant (the gene encoding human α-Gal A) recorded in their medical records that is amenable to migalastat.
    - Subjects who do not have a GLA genotype result from a prior Amicus study must have a blood sample drawn for confirmatory GLA genotyping at Visit 1.
    - For subjects without a known amenable GLA variant, GLA genotyping must be performed prior to Visit 2.
    - For subjects with a GLA variant that has not yet been tested in the Migalastat Amenability Assay, amenability testing must be completed before Visit 2.
    4. Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 in his/her medical history within the last 3 months and has an eGFR value of < 30 mL/min/1.73 m2 at Visit 1 per the central laboratory.
    5. If of reproductive potential, both male and female patients agree to use a medically accepted method of contraception during the study and for up to 30 days after the last dose of migalastat.
    E.4Principal exclusion criteria
    1. Subject has an eGFR of ≥ 30 mL/min/1.73 m2 at Visit 1 per the central laboratory.
    2. Subject has undergone kidney transplantation or is currently on dialysis, or is planned to start dialysis within 3 months of Visit 1.
    3. Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days before Visit 1.
    4. Subject has undergone any gene therapy at any time prior to the study and anticipates undergoing gene therapy during the study.
    5. Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1.
    6. Subject has clinically significant unstable cardiac disease in the opinion of the investigator (eg, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association Class III or IV congestive heart failure).
    7. Subject has any intercurrent illness or condition 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.
    8. Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
    9. Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta).
    10. Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca within the 30 days before Visit 1.
    11. Female subject is pregnant or breast-feeding.
    12. Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetic parameters and model validation will be estimated based on concentrations of migalastat in plasma and urine.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Plasma sampling:
    -Visit 2 (Week 0): Just prior to first dose of migalastat and 3 hours postdose
    -Visit 3 (Week 4-8): Subjects starting at a Q4D regimen will have PK assessments conducted predose and at 1, 2, 3, 4, 6, 8, 12, 24, 48, and 96 hours postdose. Subjects on a Q7D regimen will have PK assessments conducted predose and at 1, 2, 3, 4, 6, 8, 12, 24, 48, 96, and 168 hours postdose.
    -Visit 4 (Week 12): Just prior to last administration of migalastat and 3 hours postdose
    Urine sampling:
    -Visit 3 (Week 4-8): Within 1 hour before dosing followed by a postdose total urine collection for 24 hours in 4 incremental collections: from 0 to 4 hours, 4 to 8 hours, 8 to 12 hours, and 12 to 24 hours. Collection intervals will be the same for subjects on a Q4D regimen and a Q7D regimen.
    E.5.2Secondary end point(s)
    • Safety parameters include adverse events, clinical laboratory measures, physical examinations, vital signs, and electrocardiograms.
    • Change from baseline in eGFRMDRD, eGFRCKD-EPI, plasma lyso-Gb3, and LVMi.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety parameters include:
    -adverse events: Day -30 to -1, prior to Week (W) 0, W 0, W 4-8, W 12 or upon early termination and W 14
    -clinical laboratory measures: Day -30 to -1, prior to W 0, W 0, W 12 or upon early termination and W 14
    -physical examinations: Day -30 to -1, prior to W 0, W 0, W 12 or upon early termination and W 14
    -vital signs: Day -30 to -1, prior to W 0, W 0, W 4-8, W 12 or upon early termination and W 14
    -electrocardiograms: Day -30 to -1, prior to W 0, W 0, W 12 or upon early termination and W 14
    Change from baseline in:
    -eGFRMDRD, eGFRCKD-EPI: W 0, W 12 or upon early termination and W 14
    -plasma lyso-Gb3: W 0, W 4-8 and W 12 or upon early termination
    -LVMi: W 0 and W 12 or upon early termination
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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.7.1.3.1Other 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 trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Belgium
    France
    Italy
    Japan
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 4
    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.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 12
    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)
    After completion of the study treatment period, subjects may be offered the opportunity to participate in a separate open-label extension study.
    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 Decision2019-11-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-06
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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