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    Summary
    EudraCT Number:2011-005682-20
    Sponsor's Protocol Code Number:MOR-008
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-01-31
    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 ConcernedGermany - BfArM
    A.2EudraCT number2011-005682-20
    A.3Full title of the trial
    A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA Patients
    A.4.1Sponsor's protocol code numberMOR-008
    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 number080036421960043420
    B.5.5Fax number+1415884 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 nameBMN 110
    D.3.2Product code N/A
    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
    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
    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 the study is:
    • To evaluate the safety of 2.0 and 4.0 mg/kg/week BMN 110 administered for 27 weeks

    The primary objective for extending the Phase 2 study is:
    • To evaluate the long-term safety of 2.0 and 4.0 mg/kg/week BMN 110 in patients with MPS IVA
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks to enhance endurance
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 25 weeks on overall exercise capacity (VO2 max)
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on respiratory function tests (RFTs)
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 25 weeks on muscle strength
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on cardiac function
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on pain
    • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on plasma and urinary KS levels
    • To determine the pharmacokinetic (PK) parameters of 2.0 mg/kg/week and 4.0 mg/kg/week BMN 110

    The secondary objectives of the extension phase of the study are described on page 8 of the revised protocol.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • 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 at least 7 years of age
    • Is able to walk ≥ 200 meters as assessed by the 6MWT
    • If sexually active, is willing to use an acceptable method of contraception while participating in the study. For purposes of this study, hormonal contraception is considered to be one of the choices of an acceptable method of contraception, but only if, in consultation with his or her physician, the patient has been using or has indicated a preference for the use of this method prior to seeking enrollment in the study.
    • If female of childbearing potential, must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study

    (Refer to the Study Protocol for a complete list of inclusion criteria)
    E.4Principal exclusion criteria
    • Inability to perform an exercise test due to limited mobility
    • Body weight greater than 95 kg at screening
    • Severe, untreated sleep apnea as measured during screening with a home sleep testing device
    • Requirement for supplemental oxygen
    • Use of ventilator assistance in the 3 months prior to study entry
    • 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 previous hematopoietic stem cell transplant
    • Has received previous treatment with BMN 110
    • Has a known hypersensitivity to BMN 110 or its excipients
    • Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the duration of the study
    • Is pregnant or breastfeeding during the Screening Period or planning to become pregnant (self or partner) at any time during the study.
    • Has clinically significant bronchial asthma.

    (Refer to the Study Protocol for a complete list of exclusion criteria)
    E.5 End points
    E.5.1Primary end point(s)
    Initial Treatment Phase:
    Evaluate the safety of 2.0 and 4.0 mg/kg/week BMN 110 administered for 27 weeks

    Extension Phase 2:
    To evaluate the long-term safety of 2.0 mg/kg/week BMN 110 in patients with MPS IVA
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessed at Screening and each visit
    E.5.2Secondary end point(s)
    • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks to enhance endurance
    • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks on respiratory function tests (RFTs)
    • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks on urinary KS levels
    • Determine the pharmacokinetic (PK) parameters of 2.0 mg/kg/week and 4.0 mg/kg/week BMN 110
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Enhance endurance: Assessed at Screening, Weeks 12, 24, 52, every 52 weeks.
    • Respiratory function tests (RFTs): Assessed at Screening, Weeks 12, 24, 52, every 52 weeks.
    • Urinary KS levels: Assessed at Screening, Weeks 1, 2, 4, 6, 12, 24, 52, every 26 weeks.
    • Pharmacokinetic (PK) parameters of BMN 110 Assessed at Weeks 0 and 23
    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 Yes
    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) 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 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.8.3 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 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
    Canada
    Germany
    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
    End of trial is defined as the last visit of the last subject undergoing the trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 22
    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: 11
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 11
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 25
    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 complete the initial 27 week study period will be allowed to continue their treatment in the extension phase which will allow treatment in this study for up to 196 weeks total or until one of the following occurs: the patient (or their legally authorized representative) withdraws consent and/or discontinues from the study, the patient is discontinued from the study at the discretion of the Investigator or BioMarin, or the study is terminated by BioMarin.
    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 Decision2012-06-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-06-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-11-20
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