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    EudraCT Number:2014-002701-38
    Sponsor's Protocol Code Number:AT1001-042
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-01-16
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2014-002701-38
    A.3Full title of the trial
    An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease
    Estudio de extensión abierto para evaluar la seguridad y eficacia de la monoterapia con Clorhidrato de Migalastat en sujetos con enfermedad de Fabry
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study Using Migalastat Hydrochloride to See the Long Term Safety and Effectiveness of the Drug in Patients with Fabry Disease
    Estudio con Clorhidrato de Migalastat para comprobar la eficacia a largo plazo de la medicación en Pacientes con la Enfermedad de Fabry.
    A.4.1Sponsor's protocol code numberAT1001-042
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02194985
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    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 code08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number16096622000
    B.5.5Fax number16096625010
    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 hydrochloride
    D.3.2Product code AT1001
    D.3.4Pharmaceutical form Capsule
    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 is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A.
    La enfermedad de Fabry es una enfermedad rara de almacenamiento lisosómico ligada al cromosoma X provocada por la mutación del gen (GLA), que codifica la enzima lisosómica ?-galactosidasa A
    E.1.1.1Medical condition in easily understood language
    Fabry disease is an inherited disorder that results from the buildup of a particular type of fat in the body's cells.
    La enfermedad de Fabry es un trastorno hereditario que resulta de la acumulación de un tipo particular de grasa en las células del cuerpo.
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    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 assess long-term safety of migalastat HCl in the treatment of subjects with Fabry disease who have completed treatment in a previous study of migalastat HCl.
    Evaluar la seguridad a largo plazo del migalastat HCl en el tratamiento de sujetos con enfermedad de Fabry que hayan completado el tratamiento en un estudio previo con migalastat HCl.
    E.2.2Secondary objectives of the trial
    To explore long-term efficacy/pharmacodynamics of migalastat HCl in subjects with Fabry disease who have completed treatment in a previous study of migalastat HCl
    Investigar la eficacia/farmacodinámica del migalastat HCl en sujetos con enfermedad de Fabry que hayan completado el tratamiento en un estudio previo de migalastat HCl.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject with Fabry disease who completed treatment in a previous study of migalastat HCl given as monotherapy.
    2. Male or female subjects 16 years of age or older.
    Note: Subjects under the age of 18 will be enrolled only at sites with all required regulatory and ethics approvals to do so.
    3. A female subject is eligible to participate if she is:
    a. Of non-childbearing potential, or
    b. Of childbearing potential and NOT pregnant or nursing, has a negative urine pregnancy test at the Baseline Visit (Visit 1), and agrees to one of the methods of avoiding pregnancy listed in Appendix 1 from the time of first dose of study medication until 30 days after study completion.
    A female is considered ?Non-childbearing potential? if she is status-post hysterectomy, status-post surgical removal of both ovaries, has current, documented tubal ligation, or is postmenopausal and >2 years without menses. Female subjects who are post-menopausal <2 years must be confirmed menopausal by Follicle Stimulating Hormone (FSH) and estradiol levels.
    A female is considered ?childbearing potential? if she has functional ovaries, ducts, and uterus with no impairment that would cause sterility. This includes women with oligomenorrhea (even severe), and women who are perimenopausal or who have just begun to menstruate.
    4. Male subjects must agree to use one of the contraception methods listed in Appendix 1. This criterion must be followed from the time of the first dose of study medication until 30 days after study completion.
    5. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI) or research related health information or has a legally authorized representative who has given written informed consent.
    1. Sujeto con enfermedad de Fabry que haya completado el tratamiento en un estudio previo de migalastat HCI administrado como monoterapia.
    2. Sujetos varones o mujeres mayores de 16 años.
    Nota: Los sujetos menores de 18 años solo participarán en los centros que cuenten con todos las autorizaciones éticas y normativas para ello.
    3. Una mujer es apta para participar si:
    a. no tiene capacidad para concebir; o
    b. tiene capacidad para concebir pero no está embarazada ni en periodo de lactancia, tiene resultado negativo en test de embarazo en la visita inicial (Visita 1) y acepta uno de los métodos anticonceptivos incluidos en el apéndice 1 a partir de la primera dosis de medicación del estudio hasta 30 días después de su terminación.
    Se considera que una mujer no tiene ?capacidad para concebir? si se ha realizado histerectomía, ovariectomía bilateral, ligadura de trompas actual documentada o si es posmenopaúsica y lleva más de 2 años sin menstruación. Las mujeres que sean posmenopaúsicas con menos de 2 años deben confirmar la menopausia mediante los niveles de hormona foliculoestimulante (FSH) y de estradiol.
    Una mujer se considera con ?capacidad para concebir? si tiene ovarios, trompas y útero funcionales sin deficiencias que pudieran causar infertilidad. Esto incluye a mujeres con oligomenorrea (incluso severa), y mujeres perimenopaúsicas o que acaban de empezar a menstruar.
    4. Los sujetos masculinos deben aceptar el uso de los métodos anticonceptivos enumerados en el apéndice 1. Este criterio debe seguirse desde el momento de la primera dosis de medicación del estudio hasta 30 días después de la finalización del estudio.
    5. El sujeto tiene la capacidad y disposición de facilitar consentimiento informado y autorización por escrito para el uso y difusión de la información médica personal (IMP) o la información médica relacionada con la investigación, o tiene un representante legal autorizado que preste dicho consentimiento informado.
    E.4Principal exclusion criteria
    1. The last available estimated glomerular filtration rate (eGFR) in the previous study was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of Baseline Visit (Visit 1), which is >30 mL/min/1.73m2.
    2. The subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis.
    3. The subject is treated or has been treated with another investigational drug (except migalastat HCl) within 30 days of study start.
    4. Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator.
    5. Had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1.
    6. Has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure).
    7. Has a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (e.g., miglustat, miglitol).
    8. Requires treatment with Glyset® (miglitol) or Zavesca® (miglustat).
    9. 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.
    10. Patients with severe or unsuitable concomitant medical condition (cardiovascular, neurological, hepatic, renal, metabolic, hematological, immunological, pulmonary, or gastrointestinal disorder). The medical monitor or designee must be contacted to discuss the stability of a subject?s medical condition(s) and the potential impact of the condition(s) on trial participation.
    11. Patients with clinically significant abnormal laboratory value(s) and clinically significant electrocardiogram (ECG) findings at baseline. The medical monitor or designee must be contacted to discuss the stability of a subject?s medical condition(s) and the potential impact of the condition(s) on trial participation.
    1. La última tasa de filtración glomerular (eGFR) disponible en el estudio previo fue < 30 ml/min/1,73 m2; a menos que exista una GFR medida disponible en los 3 meses posteriores a la visita inicial (Visita 1), que sea > 30 ml/min/1,73 m2.
    2. El sujeto ha recibido, o está programado para recibir, un trasplante de riñón o está actualmente en diálisis.
    3. El sujeto está o ha sido tratado con otro fármaco en investigación (excepto migalastat HCl) en los 30 días anteriores al inicio del estudio.
    4. El sujeto no puede cumplir los requisitos del estudio, o se considera de otro modo no apto para su participación en el estudio, en opinión del investigador.
    5. Existe registro de accidente isquémico transitorio, accidente cerebrovascular, angina inestable o infarto de miocardio en los 3 meses previos a la visita 1.
    6. Tiene enfermedad cardiaca inestable clínicamente significativa en opinión del investigador (p. ej.: enfermedad cardiaca que precise gestión activa, como arritmia sintomática, angina inestable o insuficiencia cardiaca congestiva de clase III o IV según la New York Heart Association (NYHA).
    7. Tiene un historial de alergia o sensibilidad a AT1001 (incluidos excipientes) u otros iminoazúcares (p. ej.: miglustat, miglitol).
    8. Necesita tratamiento con Glyset® (miglitol) o Zavesca® (miglustat).
    9. Tiene cualquier enfermedad o trastorno concomitante que pueda impedir al sujeto de completar los requisitos del protocolo o sugiera al investigador que el posible sujeto puede tener un riesgo inaceptable por participar en este estudio.
    10. Los pacientes con problemas médicos concomitantes severos o no aptos (trastorno cardiovascular, neurológico, hepático, renal, metabólico, hematológico, inmunológico, pulmonar o gastrointestinal). Se debe contactar con el monitor médico o la persona designada para hablar sobre la estabilidad de las condiciones médicas del sujeto y sobre el impacto potencial de las condiciones en la participación en el ensayo.
    11. Los pacientes a quienes se les detecten valores analíticos anormales y electrocardiograma (ECG) clínicamente significativos en el inicio. Se debe contactar con el monitor médico o la persona designada para hablar sobre la estabilidad de las condiciones médicas del sujeto y sobre el impacto potencial de las condiciones en la participación en el ensayo.
    E.5 End points
    E.5.1Primary end point(s)
    ? Adverse events (AEs), possible suicidality related AEs (PSRAEs) and serious adverse events (SAEs)
    ? Withdrawal due to AEs
    ? Vital signs (blood pressure and heart rate) and body weight
    ? Hematology, chemistry, and urinalysis parameters
    ? Echocardiography (ECHO)
    ? Electrocardiograms (ECGs)
    ? Stopping criteria
    ? Acontecimientos adversos (AA), posible tendencia al suicido por AA (PSRAE) y acontecimientos adversos graves (AAG)
    ? Retirada debida a un AA
    ? Constantes vitales (tensión arterial y frecuencia cardiaca) y peso corporal
    ? Parámetros de hematología, bioquímica y análisis de orina
    ? Electrocardiogramas (ECG)
    ? Criterios de interrupción
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs will be collected from the start of the study treatment (i.e., visit 1) and until the follow-up contact.
    Los AA se recogerán desde el inicio del tratamiento del estudio (ej. visit 1) y hasta el contacto de seguimiento.
    E.5.2Secondary end point(s)
    ? Estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease (MDRD) equation
    ? Evaluation of left ventricular mass index (LVMi), as measured by echocardiography
    ? Ejection fraction, as measured by echocardiography
    ? Fractional shortening, as assessed by echocardiography
    ? Measurement of LV internal dimension (LVIDd and LVIDs) and wall thickness, as assessed by echocardiography
    ? Evaluation of white blood cell (WBC) ?-Gal A activity
    ? Evaluation of subject reported Quality of Life as assessed by the Short Form 36 Survey (SF 36)
    ? Tasa de filtración glomerular estimada (TFGe), basada en la ecuación de modificación de la dieta en la enfermedad renal (MDER)
    ? Evaluación de índice de masa del ventrículo izquierdo (IMVI), medido por ecocardiografía
    ? Fracción de eyección, medido por ecocardiografía
    ? Fracción de acortamiento, medido por ecocardiografía
    ? Medición de la dimensión interna del ventrículo izquierdo (DIVId y DIVIs) y grosor de la pared, evaluado por ecocardiografía
    ? Evaluación de la actividad de la ?-Gal A de los leucocitos (LEU)
    ? Calidad de vida notificada en la evaluación del sujeto siguiendo la encuesta de salud 36 abreviada (SF-36)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from baseline will be evaluated for each efficacy endpoint, when baseline is defined (as in Table 1 of the study protocol) as the value from the last treatment visit of the previous migalastat HCl study.
    El cambio desde el inicio se evaluará por cada criterio de valoración de la eficacia, en el cual el inicio se define (igual que en la Tabla 1 del Protocolo) como el valor desde la última visita del tratamiento del estudio de migalastat HCl anterior.
    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 No
    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 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
    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 planned duration of the treatment will vary among subjects and will continue until the date of regulatory approval or marketing authorization and/or commercialization in the participating subject?s country, or study termination by the Sponsor, Amicus Therapeutics (Amicus).
    La duración planificada del tratamiento variará según el sujeto y continuará hasta la fecha de la aprobación normativa o la autorización de comercialización y/o marketing en el país del sujeto participante, o cuando el promotor, Amicus Therapeutics (Amicus), finalice el estudio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 96
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 100
    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 Decision2015-03-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-28
    P. End of Trial
    P.End of Trial StatusCompleted
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