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    Summary
    EudraCT Number:2010-020199-45
    Sponsor's Protocol Code Number:MOR-005
    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:2011-12-27
    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 number2010-020199-45
    A.3Full title of the trial
    A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis
    IVA (Morquio A Syndrome)
    Studio di estensione multicentrico, multinazionale per valutare l'efficacia e la sicurezza a lungo termine di BMN 110 nei pazienti con mucopolisaccaridosi IVA (sindrome di Morquio A)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 3 extension study to evaluate the long term efficacy and safety
    of BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio A
    syndrome)
    Studio di estensione di fase III per valutare l’efficacia e la sicurezza a lungo termine di BMN 110 nei pazienti con mucopolisaccaridosi IVA (sindrome di Morquio A)
    A.4.1Sponsor's protocol code numberMOR-005
    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 SponsorBIOMARIN PHARMACEUTICAL 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number0800 234 36421960043420
    B.5.5Fax number+1 415 884 6944
    B.5.6E-mailclinicaltrials@bmrn.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/3/09/657
    D.3 Description of the IMP
    D.3.1Product nameBM110
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 9025-60-9
    D.3.9.2Current sponsor coderhGALNS
    D.3.9.3Other descriptive namerecombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mucopolysaccharidosis type IVA
    Mucopolisaccaridosi tipo IVA
    E.1.1.1Medical condition in easily understood language
    Morquio A Syndrome
    Sindrome di Morquio A
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10028095
    E.1.2Term Mucopolysaccharidosis IV
    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 evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA
    • valutare l’efficacia e la sicurezza a lungo termine della somministrazione di BMN 110 alla dose di 2,0 mg/kg/ogni settimana (qw) e di 2,0 mg/kg/a settimane alternate (qow) in pazienti con MPS IVA
    E.2.2Secondary objectives of the trial
    To evaluate the long-term effect of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow on changes in biochemical markers of inflammation and bone and cartilage metabolism, in patients with MPS IVA.
    • valutare gli effetti a lungo termine della somministrazione di BMN 110 alla dose di 2,0 mg/kg/ogni settimana (qw) e di 2,0 mg/kg/a settimane alternate (qow) sui cambiamenti nei marcatori biochimici di infiammazione e del metabolismo osseo e cartilagineo nei pazienti con MPS IVA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To be eligible for study participation, a patient must meet all of the following inclusion criteria: • Must have completed MOR-004. • Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures. • If sexually active, must be willing to use an acceptable method of contraception while participating in the study. • If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study.
    Per essere idoneo a partecipare al presente studio, il paziente deve soddisfare tutti i seguenti criteri d’inclusione: •deve aver completato lo studio MOR-004; •deve essere disposto e in grado di fornire un consenso informato scritto firmato. Oppure, nel caso di pazienti di età inferiore ai 18 anni (o un’altra età come definito dalle leggi o normative regionali) deve fornire un assenso scritto (qualora sia richiesto) e presentare un consenso informato scritto, firmato da un legale rappresentante autorizzato che abbia ricevuto la descrizione relativa alla natura dello studio e prima dell’esecuzione delle procedure relative alla ricerca; •qualora sessualmente attivo, deve essere disposto a utilizzare un metodo contraccettivo adeguato durante la partecipazione allo studio; •se di sesso femminile e in età fertile, deve presentare un test di gravidanza negativo al basale ed essere disposta a essere sottoposta a ulteriori test di gravidanza durante lo studio
    E.4Principal exclusion criteria
    • Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study. • Has used any investigational product (other than BMN 110 in MOR-004), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments. • Was enrolled in a previous BMN 110 study, other than MOR-004. • Has a concurrent disease or condition, including but not limited to,symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation, or pose a safety risk, as determined by the Investigator. • Has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
    •in caso di gravidanza o allattamento al basale oppure qualora stia programmando una gravidanza (propria o della partner) in qualsiasi momento durante lo studio; •qualora abbia utilizzato un prodotto sperimentale (diverso dal BMN 110 nel MOR-004) o un dispositivo medico sperimentale nei 30 giorni precedenti al basale; o gli viene richiesto di utilizzare una sostanza sperimentale prima del termine di tutte le valutazioni programmate nello studio; •è stato arruolato in uno studio precedente con BMN, diverso da MOR-004; •soffre di una malattia o condizione concomitante, incluse a titolo esemplificativo ma non esaustivo, instabilità sintomatica del tratto cervicale della colonna, compressione del midollo spinale clinicamente rilevante o patologia cardiaca grave che potrebbe interferire con la sua partecipazione allo studio o costituire un rischio per la sicurezza, come stabilito dallo Sperimentatore; •soffre di una condizione per la quale, secondo lo Sperimentatore, vi è un alto rischio di scarsa aderenza alla terapia o di non completamento dello studio da parte del paziente
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Variables: • endurance tests: ─ 6 minute walk (6MW) test ─ 3 minute stair climb (3MSC) test • urine KS concentration (normalized to creatinine) • respiratory function tests: ─ forced expiratory time (FET) ─ forced expiratory volume in 1 second (FEV1) ─ forced inspiratory vital capacity (FIVC) ─ forced vital capacity (FVC) ─ maximum voluntary ventilation (MVV) • anthropometric measurements (standing height, length, sitting height, and weight) • skeletal X-rays of lumbar spine and lower extremity • MPS Health Assessment Questionnaire • audiometry examinations Safety Variables: • adverse events (AEs) • standard clinical laboratory tests (serum chemistry, hematology, and urinalysis) • vital signs • echocardiograms • electrocardiograms (ECGs) • routine physical examinations (including standard neurologic examination) • concomitant medications • immunogenicity tests • cervical spine (flexion–extension) X-rays
    Variabile di efficacia •prove di resistenza:─ test a piedi 6 minuti (6 MW) ─ Test delle scale 3 minuti (3MSC) • concentrazione delle urine (normalizzazione creatinina) • prove di funzionalità respiratoria: ─ tempo di espirazione forzato (FET) ─ volume espiratorio forzato in 1 secondo (FEV1) ─ capacità vitale forzata inspiratoria (FIVC) ─ capacità vitale forzata (FVC) ─ la massima ventilazione volontaria (MVV) • misure antropometriche (altezza in piedi, lunghezza, altezza da seduti, e peso) • raggi X della colonna lombare e arti inferiori • Questionario della salute MPS • esami audiometriaci Variabili di sicurezza: • eventi avversi (EA) • test di laboratorio standard (ematochimici analisi delle urine e) •segni vitali •Ecocardiogramma •elettrocardiogrammi (ECG) • esami di routine fisiche (incluso esame neurologico) • farmaci concomitanti • test di immunogenicità • raggi X colonna cervicale (flessione-estensione)
    E.5.1.1Timepoint(s) of evaluation of this end point
    • 6MW test, 3MSC test: baseline, wk 12, every 24 wks in Part 1, every 48 wks in Part 2 • respiratory function tests: baseline, every 24 wks in Part 1, every 48 wks in Part 2 • anthropometric measurements, MPS Health Assessment Questionnaire, audiometry examinations: baseline, every 24 wks in Part 1 and Part 2 • adverse events, vital signs, concomitant medications: baseline, weekly in Part 1, weekly or every other week in Part 2 • echocardiograms, electrocardiograms: baseline, every 48 wks in Part 2 • routine physical examinations, immunogenicity tests, standard clinical laboratory tests, urine KS concentration: baseline, every 12 wks in Part 1, every 24 wks in Part 2 • x-rays of cervical spine, lumbar spine and lower extremity: baseline,every 72 wks in Part 2
    6MW,3MSC:baseline, sett 12,ogni 24 sett nella parte 1,ogni 48 sett parte 2 • prove di funzionalità respiratoria: basale, ogni 24 sett nella parte 1, ogni 48 sett parte 2 • misure antropometriche, Questionario MPS, esami audiometrici: basale, ogni 24 sett Parte 1 e Parte 2 • eventi avversi, segni vitali, farmaci concomitanti: basale, ogni sett parte 1, settimanalmente o ogni due sett parte 2 ecocardiogramma,ECG: baseline,ogni 48 sett parte 2 • esami di routine fisiche, prove di immunogenicità, test clinici di laboratorio standard, la concentrazione delle urine KS: baseline, ogni 12 sett parte 1, ogni 24 sett parte 2 •radiografie della colonna della colonna cervicale, colonna lombare e arti inferi baseline, ogni 72 settimane nella parte 2
    E.5.2Secondary end point(s)
    Exploratory Variables: • blood inflammatory biomarkers • blood biochemical markers of bone and cartilage metabolism
    Variabili esplorative: • biomarker di infiammazione nel sangue • marcatori biochimici del sangue del metabolismo osseo e cartilagine
    E.5.2.1Timepoint(s) of evaluation of this end point
    Exploratory Variables: • blood inflammatory biomarkers, and blood biochemical markers of bone and cartilage metabolism: baseline, every 24 wks in Part 1 and Part 2
    Variabili esplorativa: • biomarcatori infiammatori del sangue, e marcatori biochimici del metabolismo osseo e cartilagine: basale, ogni 24 settimane nella Parte 1 e Parte 2
    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 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 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) 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 trial2
    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 EEA19
    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
    Argentina
    Australia
    Brazil
    Canada
    Colombia
    Japan
    Korea, Democratic People's Republic of
    Qatar
    Saudi Arabia
    Switzerland
    Taiwan
    Turkey
    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
    LVLS
    LVLS
    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: 110
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 70
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors
    Minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 90
    F.4.2.2In the whole clinical trial 162
    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)
    Treatment will be provided through this extension study for up to 240 weeks or until one of the following occurs: the patient withdraws consent and discontinues from the study, the patient is discontinued from the study at the discretion of the Investigator, or the study is terminated. Patients will be transitioned to commercial therapy once BMN 110 is granted marketing authorisation
    Il trattamento sarà fornito con questo studio di estensione fino a 240 settimane o fino a quando non si verificheranno uno dei seguenti casi: il paziente ritira il consenso del paziente e interrompe lo studio, il paziente viene ritirato dallo studio per decisione dello sperimentatore, o lo studio è terminato . I pazienti passeranno alla terapia in commercio una volta che avrà ottenuto la autorizzazione all’'immissione in commercio
    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 Decision2011-12-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-10-20
    P. End of Trial
    P.End of Trial StatusCompleted
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