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    Summary
    EudraCT Number:2011-004800-40
    Sponsor's Protocol Code Number:MGM116041
    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:2012-01-11
    Trial results View 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 ConcernedSpain - AEMPS
    A.2EudraCT number2011-004800-40
    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 la eficacia a largo plazo de clorhidrato de migalastat en monoterapia 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 to see the safety and usefulness of the drug in patients with Fabry Disease.
    Un estudio que utiliza Migalastat para ver la seguridad y utilidad del fármaco en pacientes con la enfermedad de Fabry.
    A.4.1Sponsor's protocol code numberMGM116041
    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 SponsorGlaxoSmithKline Research & Development Limited
    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 supportGlaxoSmithKline Research & Development Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGlaxoSmithKline Research & Development
    B.5.2Functional name of contact pointClinical Trials Helpdesk
    B.5.3 Address:
    B.5.3.1Street Address1-3, Iron Bridge Road
    B.5.3.2Town/ cityUxbridge, Middlesex
    B.5.3.3Post codeTW8 9GS
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44 20 8990 4466
    B.5.5Fax number+44 20 8990 1234
    B.5.6E-mailGSKClinicalSupportHD@gsk.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 numberORPHA89184
    D.3 Description of the IMP
    D.3.1Product nameMigalastat Hydrochloride
    D.3.2Product code GR181413
    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 Hydrochloride
    D.3.9.1CAS number 75172-81-5
    D.3.9.2Current sponsor codeAT1001
    D.3.9.3Other descriptive nameGR181413A
    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 disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme alfa-galactosidase A.
    La enfermedad de Fabry es una enfermedad rara del almacenamiento lisosomal ligada al cromosoma X causada por mutaciones en el gen GLA que codifica la enzima lisosomal, alfa-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 deorden inherente que resulta en la acumulación de un tipo particular de grasas 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 14.1
    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 completed treatment in a previous study of migalastat HCl.
    Determinar 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.
    Explorar la eficacia y la farmacodinamia a largo plazo del migalastat HCl en sujetos con enfermedad de Fabry que hayan completado el tratamiento en un estudio previo con 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 as per the study protocol 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 has a legally authorized
    representative who has given written informed consent.

    6. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
    1.Sujeto con enfermedad de Fabry que haya completado el tratamiento en un estudio previo con migalastat HCl administrado en monoterapia.
    2.Mujeres o varones de 16 años de edad en adelante.Nota: los sujetos de menos de 18 años de edad se incluirán exclusivamente en los centros que cumplan todas las aprobaciones administrativas y éticas exigidas para ello.
    3.Las mujeres podrán participar siempre que: a)No estén en edad fértil; o b) estén en edad fértil y NO estén embarazadas ni en periodo de lactancia, dispongan de una prueba de embarazo negativa en orina en la visita basal (visita 1) y acepten uno de los métodos anticonceptivos recogidos en el Apéndice 1 desde el momento de recibir la primera dosis del fármaco del estudio hasta 30 días después de la finalización del estudio.Una mujer se considera que no se encuentra en edad fértil si se ha sometido a histerectomía, ovariectomía bilateral o ligadura de trompas documentada o es postmenopáusica y carece de menstruaciones desde hace > dos años. Las mujeres postmenopáusicas sin menstruación desde < de dos años deben confirmar su estado a partir de los niveles de hormona foliculina (FSH) y estradiol. Una mujer se considera en edad fértil si tiene ovarios funcionales, trompas y útero intactos, sin ningún daño que pudiera causar esterilidad. Se incluyen aquí mujeres con oligomenorrea (incluso intensa) y mujeres que se encuentren en periodo perimenopáusico o que hayan comenzado la menstruación.
    4.Los varones deben aceptar el uso de uno de los métodos contraceptivos recogidos en el Apéndice 1. Este criterio debe mantenerse desde el momento de la administración de la primera dosis de la medicación del estudio hasta 30 días después de la finalización del estudio.
    5.El sujeto está dispuesto y es capaz de otorgar el consentimiento informado y la autorización por escrito para el uso y publicación de su información de salud personal (ISP) o dispone de un representante legal autorizado que ha otorgado el consentimiento informado.
    6. Pacientes franceses: en Francia un sujeto será considerado apto para participar en este estudio sólo si está afiliado o es beneficiario de la seguridad social.
    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.
    1. La última FGe disponible del estudio previo fue <30 ml/min/1,73 m2, a menos que se disponga de una FG medida de los 3 meses previos a la visita basal (visita 1) >30 ml/min/1,73 m2.
    2.El sujeto se ha sometido o va a someterse a un transplante renal o se encuentra actualmente en diálisis.
    3. El sujeto está recibiendo o ha recibido otro fármaco en investigación (salvo migalastat HCl) en los 30 días previos al comienzo del estudio.
    4.El sujeto es incapaz de cumplir los requisitos del estudio, o el investigador lo juzga no apto para la inclusión.
    E.5 End points
    E.5.1Primary end point(s)
    The safety assessments are:
    - 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)
    Las evaluaciones de la seguridad comprenden lo siguiente:
    - Acontecimientos adversos (AA), acontecimientos adversos relacionados con posibles ideas suicidas (AARPIS) y acontecimientos adversos graves (AAG).
    - Retirada debida a AA.
    - Constantes vitales (presión arterial y frecuencia cardiaca) y peso.
    - Hematología, bioquímica y parámetros del análisis de orina.
    - Ecocardiografía (ECO).
    - Electrocardiogramas (ECG).
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs will be collected from the start of study treatment (i.e., Visit 1) and until the follow-up contact.
    AAs se recogerán desde el comienzo del tratamiento del estudio (pe visita 1) y hasta el contacto de seguimiento.
    E.5.2Secondary end point(s)
    The efficacy/pharmacodynamic assessments are:
    - Estimated glomerular filtration rate (eGFR) based on the Modification of Diet in Renal Disease (MDRD) equation.
    - Measurement of 24-hour urine protein
    - Evaluation of left ventricular mass index (LVMi ), as measured by echocardiography
    - Ejection fraction, as measured by echocardiography
    - Evaluation of urine globotriaosylceramide (GL-3) from 24-hour urine collection
    - Evaluation of leukocyte alfa-galactosidase A (alfa-Gal A) activity
    - Evaluation of patient reported assessment of pain as assessed by the Brief Pain Inventory (BPI) short form
    - Evaluation of patient reported Quality of Life as assessed by the Short Form -36 survey (SF-36).
    Las evaluaciones de la eficacia y la farmacodinamia son:
    - FG estimada (FGe) basada en la ecuación modificación de la dieta en las nefropatías (MDRD).
    - Determinación de proteínas en la orina de 24-horas.
    - Medición del índice de masa del ventrículo izquierdo (índice MVI) mediante ecocardiografía.
    -Determinación de la fracción de eyección mediante ecocardiografía.
    -Determinación de globotriaosilceramida (GL-3)en orina recogida en 24h.
    -Determinación de la actividad leucocitaria de alfa-galactosidasa ( alfa-Gal A).
    -Evaluación de la intensidad del dolor comunicada por el paciente mediante el cuestionario abreviado del inventario del dolor (BPI).
    -Evaluación de la calidad de vida realizada por el paciente mediante el cuestionario abreviado de 36 preguntas (SF-36).
    Para cada criterio de valoración de la eficacia se evaluará el cambio desde el momento basal, definido (como en la tabla 1 del protocolo) como el valor obtenido en la última visita de tratamiento del estudio previo de migalastat HCl.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from baseline will be evaluated for each efficacy endpoint, where 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.
    Para cada criterio de valoración de la eficacia se evaluará el cambio desde el momento basal, definido (como en la tabla 1 del protocolo) como el valor obtenido en la última visita de tratamiento del estudio previo de migalastat HCl.
    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.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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 EEA9
    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
    Australia
    Brazil
    Denmark
    Egypt
    France
    Italy
    Spain
    Turkey
    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
    The duration of the treatment will vary among subjects. Within each participating country, subjects may continue to receive treatment with migalastat HCl within this protocol until one of the study conclusion conditions listed in section 3.1 of the study protocol apply.
    This protocol will be open until the end of 2016, after which time the continued utility of the study will be evaluated by the sponsor.
    La duración del tratamiento variará entre los sujetos. En cada uno de los países participantes, los sujetos pueden continuar recibiendo tratamiento con migalastat HCl con arreglo al protocolo hasta que se produzca una de las situaciones listadas en la sección 3.1 del protocolo. Este protocolo se mantendrá activo hasta finales de 2016, momento en que el promotor evaluará si está justificado continuar 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 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: 96
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    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)
    The investigator is responsible for ensuring that consideration has been given to the poststudy
    care of the patient's medical condition whether or not GSK is providing specific post study treatment.
    El investigador es responsable de garantizar que se ha valorado la asistencia posterior al estudio para la situación médica del paciente, tanto si GSK proporciona tratamiento específico después del estudio como si no.
    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 Decision2012-02-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-02-17
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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