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    Summary
    EudraCT Number:2023-001027-16
    Sponsor's Protocol Code Number:LPS16578
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2023-11-22
    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 number2023-001027-16
    A.3Full title of the trial
    A Phase 4, single-arm, open-label safety and efficacy study of Aldurazyme® (laronidase) as enzyme replacement therapy in participants with Mucopolysaccharidosis I (MPS I) in China
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    China post-marketing surveillance (PMS) study of Aldurazyme®
    A.4.1Sponsor's protocol code numberLPS16578
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1260-3947
    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 SponsorGenzyme Europe B. V
    B.1.3.4CountryNetherlands
    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 supportGenzyme Europe B. V
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenzyme Europe B. V
    B.5.2Functional name of contact point-
    B.5.3 Address:
    B.5.3.1Street AddressPaasheuvelweg 25
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105 BP
    B.5.3.4CountryNetherlands
    B.5.4Telephone number----
    B.5.5Fax number----
    B.5.6E-mailcontactus@sanofi.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 Yes
    D.2.1.1.1Trade name Aldurazyme®
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAldurazyme
    D.3.4Pharmaceutical form Concentrate for 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 INNLaronidase
    D.3.9.1CAS number 210589-09-6
    D.3.9.4EV Substance CodeSUB20048
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 I
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis I
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10056886
    E.1.2Term Mucopolysaccharidosis I
    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 safety and tolerability of Aldurazyme in Chinese MPS I participants
    • To evaluate the efficacy of Aldurazyme on urinary glycosaminoglycans (uGAGs) after 26 weeks of treatment in Chinese MPS I participants
    E.2.2Secondary objectives of the trial
    • To evaluate the effect on uGAG level in Chinese MPS I participants
    • To evaluate liver volume (hepatomegaly) with Aldurazyme treatment in Chinese MPS I participants
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA activity <10% of normal.

    Participants have to be able to stand independently and walk a minimum of 5 meters in 6 minutes.

    A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
    Is not a woman of childbearing potential (WOCBP).
    OR
    Is a WOCBP and agrees to use an acceptable contraceptive method during the intervention period and at a minimum until 7 days after the last dose of study intervention.

    A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within the screening period before the first dose of study intervention.

    If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.

    Contraceptive/barrier method is not applicable for male participants.
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation.
    Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment.
    Received an investigational drug, or device, other than Aldurazyme, within 30 days prior to study enrollment.
    Received an investigational gene therapy.
    E.5 End points
    E.5.1Primary end point(s)
    1/ Participants with adverse events (AEs)
    2/ The change during the TE period in electrocardiogram (ECG), clinical laboratory parameters (hematology and biochemistry), and vital signs
    3/ The percent change of uGAGs
    E.5.1.1Timepoint(s) of evaluation of this end point
    1/ and 2/: Baseline to Week 27
    3/: Baseline to Week 26
    E.5.2Secondary end point(s)
    1/ The percent change of uGAGs
    2/ The absolute change of uGAGs
    3/ The percent change of liver volume (Abdominal B type ultrasound examination)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/ and 2/: Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20
    3/: Baseline to Week 26
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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 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
    China
    E.8.7Trial has a data monitoring committee No
    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
    LPLV
    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 months11
    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: 12
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 8
    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 No
    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 16
    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)
    none
    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: China
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