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    Summary
    EudraCT Number:2011-005703-33
    Sponsor's Protocol Code Number:MOR-006
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-03-01
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-005703-33
    A.3Full title of the trial
    A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation
    A.4.1Sponsor's protocol code numberMOR-006
    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.5.6E-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 namerecombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
    D.3.2Product code BMN 110
    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.3.8INN - Proposed INNNot Available
    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.3.10 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Mucopolysaccharidosis Type IVA
    E.1.1.1Medical condition in easily understood language
    Morquio A Syndrome
    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 16.0
    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
    The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 (as defined by the domains of upper extremity function and dexterity, mobility, pain, and self care and functional abilities) in a patient population that has limited ambulation.
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of 2.0 mg/kg/week BMN 110 on respiratory function in patients with MPS IVA who have limited ambulation.
    • To evaluate the effect of 2.0 mg/kg/week BMN 110 on sleep apnea in patients with MPS IVA who have limited ambulation.
    • To evaluate the effect of 2.0 mg/kg/week BMN 110 on urine KS levels in patients with MPS IVA who have limited ambulation.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Is willing and able to provide written, signed informed consent (or the patient’s legally authorized representative) after the nature of the study has been explained and prior to performance of any research-related procedure. Patients who do not meet country and local age requirements for informed consent must be willing and able to provide written assent after the nature of the study has been explained and prior to performance of any research-related procedure.
    • Has 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.
    • Is ≥ 5 years of age.
    • If sexually active, is willing to use an acceptable method of contraception while participating in the study.
    • Females of childbearing potential must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
    • Is willing and able to perform all study procedures as physically possible.
    E.4Principal exclusion criteria
    • Is able to walk ≥ 30 meters as assessed by the 6MWT.
    • Has previous hematopoietic stem cell transplant (HSCT).
    • Has received previous treatment with BMN 110.
    • Has a known hypersensitivity to any of the components of BMN 110.
    • Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the first 24 weeks of the study.
    • Has used any other investigational product or investigational medical device within 30 days prior to the Screening Visit or requires any investigational agent prior to completion of all scheduled study assessments.
    • Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study.
    • Has a concurrent disease or condition, including but not limited to symptomatic cervical spine instability or severe cardiac disease or complete paralysis due to a spinal cord injury (defined as an inability to move arms and legs), that would interfere with study participation or safety 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.
    E.5 End points
    E.5.1Primary end point(s)
    There are no statistical criteria for termination of the study.

    1) Primary efficacy assessments and procedures are the FDT and GPT (upper extremity function and dexterity), 25FWT (mobility), BPI short form (pain tool for adult patients) or APPT (pain tool for pediatric patients), and the PODCI (QOL questionnaire for pediatric patients) or SF-36® (QOL questionnaire for adult patients).
    E.5.1.1Timepoint(s) of evaluation of this end point
    1) Every 12 weeks (Baseline, Week 12, Week 24, Week 36, and Week 48 [or the Early Termination Visit (ETV)]).
    E.5.2Secondary end point(s)
    1) Safety assessments (AEs, vital signs, concomitant medications)
    2) Clinical laboratory tests
    3) Physical examinations (including neurological examination; and presence or absence of corneal clouding)
    4) Blood sampling for immunogenicity testing
    5) ECHOs and ECGs
    6) Radiographs of the cervical spine will be performed at all sites excluding those in Germany
    7) Magnetic resonance imaging (MRI) scans of the cervical spine
    8) Respiratory function tests (RFTs) for assessment of FET, FEV1, FIVC, FVC, MVV and optional TLC
    9) Sleep apnea assessment measured by Apnea-Hypopnea Index (AHI)
    10) Urine samples for measurement of urinary KS and urinary creatinine
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Throughout this 48-week study
    2) Screening, Baseline, and Weeks 12, 24, 36, and 48 (or ETV)
    3) Screening and Weeks 12, 24, 36, and 48 (or ETV)
    4) Baseline, Weeks 2, 4, 6, 12, and Weeks 24, 36, and 48 (or ETV)
    5) Screening and Weeks 24 and 48 (or ETV) visits
    6) Baseline and at Week 48 (or ETV)
    7) Screening and Week 48 (or ETV)
    8) Baseline and Weeks 24 and 48 (or ETV)
    9) Baseline and Weeks 24 and 48 (or ETV)
    10) Baseline, Weeks 2, 4, 6, and 12, and then Weeks 24, 36, and 48 (or ETV)
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Germany
    Italy
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 20
    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 may participate in the study for up to 49 weeks during the initial treatment phase and up to an additional 96 weeks during the extension phase or until one of the following occurs: the patient (or the patient’s legally authorized representative) withdraws consent or participation in the study, the patient is discontinued from the study at the discretion of the Investigator or BioMarin, or the study is terminated.
    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 Decision2012-02-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-10-22
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