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    Summary
    EudraCT Number:2011-003197-84
    Sponsor's Protocol Code Number:MOR-007
    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 number2011-003197-84
    A.3Full title of the trial
    A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    Studio clinico multinazionale di fase 2, in aperto, per valutare la sicurezza e l'efficacia di BMN 110 in pazienti pediatrici di eta' inferiore ai 5 anni affetti da mucopolisaccaridosi IVA (sindrome di Morquio A)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of Age
    Uno studio per valutare la sicurezza e l'efficacia di BMN 110 in pazienti di eta' inferiore ai 5 anni affetti da MPS IVA
    A.4.1Sponsor's protocol code numberMOR-007
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/090/2011
    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 Address164 Shaftesbury Avenue
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeWC2H 8HL
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number0046708194881
    B.5.5Fax number00442074200829
    B.5.6E-mailbiomarin-europe@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 nameBMN 110
    D.3.2Product code NA
    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 drug substance
    D.3.9.4EV Substance CodeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mucopolysaccharidosis Type IVA
    Mucopolisaccaridsi 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 safety and tolerability of infusions of BMN 110 at a dose of 2.0 mg/kg/week over a 52-week period in MPS IVA patients less than 5 years of age.
    Valutare la sicurezza e la tollerabilità di infusioni di BMN 110 alla dose di 2,0 mg/kg/settimana per un periodo di 52 settimane in pazienti affetti da MPS IVA di età inferiore a 5 anni.
    E.2.2Secondary objectives of the trial
    1-To evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age. 2-To evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age.
    1- Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di ridurre i livelli urinari di cheratan solfato (KS) nei pazienti affetti da MPS IVA di età inferiore a 5 anni. 2-Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di influire sulla rapidità della crescita nei pazienti affetti da MPS IVA di età inferiore a 5 anni.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1- Less than 5 years of age. 2-Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA. 3-Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.
    1-Età inferiore a 5 anni. 2- Diagnosi clinica documentata di MPS IVA, basata su segni e sintomi di MPS IVA, e riduzione documentata dell’attività dell’enzima GALNS nei fibroblasti o nei leucociti, oppure test genetico di conferma della diagnosi di MPS IVA. 3- Consenso informato scritto fornito da un genitore o da un rappresentante legalmente autorizzato a seguito della spiegazione della natura di questo studio e prima di qualsivoglia procedura correlata alla ricerca.
    E.4Principal exclusion criteria
    • Previous hematopoietic stem cell transplant (HSCT). • Previous treatment with BMN 110. • Known hypersensitivity to any of the components of BMN 110. • Major surgery within 3 months prior to study entry or planned major surgery during the 52-week treatment period. • Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments. • 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 safety as determined by the Investigator. • 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.
    •Individui sottoposti a precedente trapianto di cellule staminali emopoietiche (TCSE) • Precedente trattamento con BMN 110. • Individui affetti da ipersensibilità nota a un qualsiasi componente di BMN 110. • Individui sottoposti a importanti interventi di chirurgia nei 3 mesi precedenti all'ingresso nello studio o per i quali risulti programmato un intervento di chirurgia importante nel corso del periodo di trattamento della durata di 52 settimane. • Individui che abbiano utilizzato un qualsiasi prodotto o dispositivo medico sperimentale nei 30 giorni precedenti allo Screening o che richiedano l'impiego di un qualsiasi prodotto sperimentale prima del completamento di tutte le valutazioni programmate inerenti lo studio. • Individui affetti da malattie o condizioni concomitanti che interferirebbero con la partecipazione del paziente allo studio o con la sua sicurezza secondo quanto stabilito dallo Sperimentatore, comprese ma non limitate a una instabilità sintomatica della rachide cervicale, una compressione spinale clinicamente rilevante o una grave cardiopatia. • Individui che si trovino in una condizione che, a giudizio dello Sperimentatore, ponga il paziente ad alto rischio di una scarsa compliance al trattamento o di interruzione prematura dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    Evaluate safety and tolerability of infusions of BMN 110 at a dose of 2.0 mg/kg/week over a 52-week period in MPS IVA patients less than 5 years of age.
    Valutare la sicurezza e la tollerabilità di infusioni di BMN 110 alla dose di 2,0 mg/kg/settimana per un periodo di 52 settimane in pazienti affetti da MPS IVA di età inferiore a 5 anni.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessed at Screening and each visit
    Valutato allo screening e ad ogni visita
    E.5.2Secondary end point(s)
    1- Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age 2- Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age
    1- Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di ridurre i livelli urinari di KS nei pazienti affetti da MPS IVA di età inferiore a 5 anni. 2-Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di influire sulla rapidità della crescita nei pazienti affetti da MPS IVA di età inferiore a 5 anni.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Evaluate the ability of 2.0 mg/kg/week BMN 110 to reduce urinary keratan sulfate (KS) levels in MPS IVA patients less than 5 years of age: Assessed at Baseline, Weeks 2, 4, 8, 13, 26, 39, and 52 • Evaluate the ability of 2.0 mg/kg/week BMN 110 to affect growth velocity in MPS IVA patients less than 5 years of age: Assessed at Baseline and every 13 weeks
    • Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di ridurre i livelli urinari di cheratansolfato solfato (KS), nei pazienti MPS IVA di età inferiore a 5 anni: Valutati al basale, Settimane 2, 4, 8, 13, 26, 39, e 52 • Valutare la capacità di 2,0 mg/kg/settimana di BMN 110 di influenzare la velocità di crescita nei pazienti con MPS IVA di età inferiore a 5 anni: Valutati al Basale e ogni 13 settimane
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 No
    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 EEA3
    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 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
    End of trial is defined as the last visit of the last subject undergoing the trial.
    La fine dello studio clinico è definita come l'ultima visita dell'ultimo soggetto sottoposto allo studio clinico.
    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 months18
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months18
    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: 15
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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
    Children (2-11 years). Infants and toddlers (28 days - 23 months).
    Bambini (2-11 anni) Neonati e bambini (28 giorni - 23 mesi).
    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 10
    F.4.2.2In the whole clinical trial 15
    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)
    Patients who completed this study will be given the option to enroll in an extension study.
    Ai pazienti che hanno completato questo studio sarà data la possibilità di iscriversi a uno studio di estensione.
    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 Decision2012-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-26
    P. End of Trial
    P.End of Trial StatusCompleted
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