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    Summary
    EudraCT Number:2011-004800-40
    Sponsor's Protocol Code Number:MGM116041
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-03-12
    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 ConcernedItaly - Italian Medicines Agency
    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.
    Uno studio di estensione in aperto per valutare la sicurezza e l'efficacia a lungo termine della monoterapia con migalastat HCl nei soggetti con malattia di 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.
    Studio per valutare l'efficacia e la sicurezza di Migalastat in pazienti con malattia di 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 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 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 numberEU/3/06/368
    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.3.9.4EV Substance CodeNA
    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 α- galactosidase A.
    La malattia di Fabry è una rara malattia legata al cromosoma X che determina un accumulo lisosomiale a causa della mutazione nel gene (GLA) che codifica per l'enzima lisosomiale α- galattosidasi 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 malattia di Fabry è una malattia ereditaria che determina la formazione di un particolare tipo di grasso nelle cellule del corpo.
    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 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 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.
    Valutare la sicurezza a lungo termine di migalastat HCl nel trattamento dei soggetti con malattia di Fabry che hanno completato il trattamento in uno studio precedente su 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.
    Analizzare l’efficacia/la farmacodinamica a lungo termine di migalastat HCl nei soggetti con malattia di Fabry che hanno completato il trattamento in uno studio precedente su 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 ''childbearingpotential'' 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. Soggetti con malattia di Fabry che hanno completato il trattamento in uno studio precedente su migalastat HCl somministrato come monoterapia. 2. Soggetti di sesso maschile o femminile di età ≥ 16 anni Nota: i soggetti di età inferiore a 18 anni saranno arruolati solo nei centri che dispongono di tutte le necessarie approvazioni etiche e di regolamentazione. 3. Un soggetto di sesso femminile può partecipare se: A. Non è potenzialmente fertile oppure B. È potenzialmente fertile ma NON in gravidanza o in allattamento, presenta alla visita di baseline (Visita 1) un test di gravidanza sulle urine negativo e accetta di usare uno dei metodi contraccettivi di cui all'Appendice 1 dall'assunzione della prima dose di farmaco sperimentale fino a 30 giorni dopo il completamento dello studio. Una donna è considerata “potenzialmente non fertile” se ha subito un’isterectomia, la rimozione chirurgica di entrambe le ovaie, la chiusura delle tube (documentata) oppure è in stato di post-menopausa e da &gt;2 anni non ha il ciclo mestruale. I soggetti di sesso femminile in post-menopausa da &lt;2 anni devono presentare livelli di ormone follicolostimolante (FSH) e di estradiolo nel range previsto per la post-menopausa. Una donna è considerata “potenzialmente fertile” se ha ovaie, dotti e utero funzionanti, senza menomazioni che causino sterilità. Rientrano in questo gruppo donne con oligomenorrea (anche grave) e donne in perimenopausa o che hanno appena avuto il ciclo mestruale. 4. I soggetti di sesso maschile devono acconsentire a usare uno dei metodi contraccettivi di cui all’Appendice 1. Questo criterio deve essere rispettato dall’assunzione della prima dose di farmaco sperimentale fino a 30 giorni dopo il completamento dello studio. 5. Il soggetto accetta ed è in grado di fornire un consenso informato scritto e l’autorizzazione all’uso e alla divulgazione delle informazioni personali sulla salute (PHI) oppure ha un rappresentante legale autorizzato a fornire il consenso informato scritto. Soggetti francesi: in Francia un soggetto sarà idoneo all'inclusione nello studio solo se affiliato a o beneficiario di una categoria di previdenza sociale.
    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. L’ultimo eGFR disponibile nello studio precedente era &lt;30 ml/min/1,73m2; a meno che non siano disponibili misure di GFR effettuate nei 3 mesi precedenti la visita di baseline (Visita 1), pari a &gt;30 ml/min/1,73 m2. 2. Il soggetto è stato sottoposto o deve essere sottoposto a trapianto di rene oppure è attualmente in dialisi. 3. Il soggetto viene trattato o è stato trattato con un altro farmaco sperimentale (tranne migalastat HCl) nei 30 giorni precedenti l’inizio dello studio. 4. Il soggetto non è in grado di rispettare i requisiti dello studio oppure viene giudicato dallo Sperimentatore, per altri motivi, inadatto all'ingresso nello studio.
    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)
    Le valutazioni relative alla sicureza sono: - Eventi avversi (AEs), eventi avversi legati alla possibile suicidalità (PSRAEs), eventi avversi seri (SAEs). - Ritiro dallo studio per AEs - Segni vitali (pressione e frequenza) e peso corporeo. - Parametri ematologici, biochimici e urinari. -Esame echocardiografico. -Elettrocardiogramma.
    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.
    Gli eventi avversi saranno raccolti dall'inizio del trattamento (visita 1) sino all'ultimo contatto di follw-up.
    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 α-galactosidase A (α-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).
    Valutazioni di efficacia e di farmacodinamica: - stima della filtrazione glomerulare dasata sulla equazione di modificazione della dieta nella patologia renale (MDRD). - Misurazione delle proteine nelle urine delle 24 ore. - Misurazione dell'indice di massa ventricolare sinistra misurata mediante echocardiografia. -Frazione di eiezione,misurata mediante echocardiografia. - Misurazione della globotriaosylceramide (GL-3) nelle urine delle 24 ore. -Valutazione dell'attività di α-galactosidase A (α-Gal A) nei leucociti. -Valutazione del dolore mediante il questionario Brief Pain Inventory (BPI). - Valutazione della Qualità della Vita, misurata mediante il questionario ''Short Form -36 survey (SF-36)''.
    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.
    Modifiche rispetto al baseline verranno esaminate per ciascun end point, dove per baseline si intende (tavola 1 del protocollo)come il valore dall'ultima visita delprecedente studio con migalastat.
    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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 No
    E.8.4.1Number of sites anticipated in Member State concerned1
    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
    Egypt
    Turkey
    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 durata del trattamento varierà da soggetto a soggetto sino a che si verificheranno le condizioni indicatenella sezione 3.1 del protocollo. Lo studio rimarrà aperto sino alla fine del 2016, dopo di ché lo Sponsor valuterà se continuare o meno.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months55
    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 state4
    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.
    Lo Sperimentatore ha la responsabilità di assicurare ai pazienti le cure mediche per la loro condizione clinica, indipendentemente dal fatto che GSK fornisca o meno trattamenti specifici al termine dello studio.
    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 Decision2012-05-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-02-17
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