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    Summary
    EudraCT Number:2014-005638-71
    Sponsor's Protocol Code Number:UX003-CL301
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2014-12-22
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2014-005638-71
    A.3Full title of the trial
    A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An efficacy and safety study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection
    A.4.1Sponsor's protocol code numberUX003-CL301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02230566
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/151/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUltragenyx 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 supportUltragenyx Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Operation
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number14158278989
    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/3/12/973
    D.3 Description of the IMP
    D.3.1Product nameRecombinant human beta-glucuronidase
    D.3.2Product code UX003
    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 1638194-78-1
    D.3.9.2Current sponsor codeUX003
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
    D.3.9.4EV Substance CodeSUB128266
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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
    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 7 (MPS 7, Sly syndrome)
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Efficacy of UX003 in MPS 7 subjects based on the totality of the clinical data on a per subject basis. No primary endpoint will be declared. (US only)
    • Efficacy of UX003 in MPS7 subjects as determined by the percent reduction of uGAG excretion after 24 weeks of treatment relative to the pre-treatment baseline. (EU and rest of World)

    E.2.2Secondary objectives of the trial
    • Safety and tolerability following up to 48 weeks of UX003 exposure
    • Efficacy of UX003 in MPS 7 subjcts as measured by a multi-domain clinical responder index following 24 weeks of UX003 exposure
    • Efficacy following 24 weeks of treatment as determined by the proportion of subjects achieving a positive ICR outcome
    • Clinical effects including pulmonary function, walking distance, shoulder flexion, fine motor function, and gross motor function following 24 weeks of UX003 exposure
    • Efficacy of UX003 in MPS7 subjects as determined by the percent reduction of uGAG excretion after 24 weeks of treatment relative to the pre-treatment baseline. (US only)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing
    2) Elevated uGAG excretion at a minimum of 3-fold over the mean normal for age (at Screening)
    3) Apparent clinical signs of lysosomal storage disease as judged by the Investigator, including at least one of the following: enlarged liver and spleen, joint limitations, airway obstruction or pulmonary problems, limitation of mobility while still ambulatory
    4) Aged 5 to 35 years, inclusive
    5) Willing and able to provide written informed consent, or in the case of subjects under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
    6) Sexually active subjects must be willing to use acceptable highly effective methods of contraception while participating in the study and for 30 days following the last dose
    7) Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have not experienced menarche, or have had tubal ligation at least one year prior to Screening, or who have had total hysterectomy
    8) Naïve to treatment with UX003
    E.4Principal exclusion criteria
    1) Undergone a successful bone marrow or stem cell transplant or has any degree of detectable chimaerism with donor cells
    2) Major surgery within 3 months prior to study entry or planned major surgery during the study that may not allow safe participation in the study
    3) Presence or history of any hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
    4) Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study
    5) Use of any investigational product (drug or device or combination) within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
    6) Presence of a condition of such severity and acuity that, in the opinion of the Investigator, warrants immediate surgical intervention or other treatment or may not allow safe participation in the study
    7) Concurrent disease or condition, or laboratory abnormality that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduce additional safety concerns.
    E.5 End points
    E.5.1Primary end point(s)
    No primary endpoint for US.
    For EU and Rest of World, efficacy of UX003 in MPS7 subjects as determined by the percent reduction of uGAG excretion after 24 weeks of treatment relative to the pre-treatment baseline.

    E.5.1.1Timepoint(s) of evaluation of this end point
    For EU and Rest of World, efficacy (total urinary GAG excretion): Urine samples will be collected every other week up to Week 48, and 2 samples will be collected during the baseline week.
    E.5.2Secondary end point(s)
    • Safety and tolerability following up to 48 weeks of UX003 exposure
    • Efficacy of UX003 in MPS 7 subjcts as measured by a multi-domain clinical responder index (MDRI), consisting of 6MWT, FVC, shoulder flexion, visual acuity, BOT-2 fine motor and BOT-2 gross motor, following 24 weeks of UX003 exposure
    • Efficacy following 24 weeks of treatment as determined by the proportion of subjects achieving a positive Individualized clinical response (ICR) outcome
    • Clinical effects including pulmonary function, walking distance, shoulder flexion, fine motor function, and gross motor function following 24 weeks of UX003 exposure
    • Efficacy of UX003 in MPS7 subjects as determined by the percent reduction of uGAG excretion after 24 weeks of treatment relative to the pre-treatment baseline. (US only)
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Safety: Assessed up to Week 48. Serious adverse events (SAEs) will be recorded beginning at the time the subject signs the informed consent form (ICF) through 30 days following the last study visit. Non-serious
    AEs will be recorded from the time of signing the ICF through the last study visit.
    • MDRI: assessed every 8 weeks.
    • ICR outcome: assessed every 8 weeks.
    • Clinical effects including pulmonary function, walking distance, shoulder flexion, fine motor function, and gross motor function, assessed every 8 weeks.
    • For US only - total urinary GAG excretion: Urine samples will be collected every other week up to Week 48, and 2 samples will be collected during the baseline week.
    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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    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: 8
    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: 4
    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: 4
    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
    Pediatric patients.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 12
    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)
    Subjects who elect to continue UX003 treatment after completing this study UX003-CL301 may be transitioned to a separate long-term, open-label extension study, as appropriate.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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