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    Summary
    EudraCT Number:2022-004193-39
    Sponsor's Protocol Code Number:TAK-665-4001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-12-04
    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 number2022-004193-39
    A.3Full title of the trial
    A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
    A.4.1Sponsor's protocol code numberTAK-665-4001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05058391
    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 SponsorTakeda Biopharmaceuticals India Pvt. Ltd.
    B.1.3.4CountryIndia
    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 supportTakeda Biopharmaceuticals India Pvt. Ltd.
    B.4.2CountryIndia
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda
    B.5.2Functional name of contact pointStudy Director
    B.5.3 Address:
    B.5.3.1Street AddressBuilding No. 8, 6th Floor, Tower C DLF Cyber City
    B.5.3.2Town/ cityGurgaon
    B.5.3.3Post code122002
    B.5.3.4CountryIndia
    B.5.6E-mailclinicaltransparency@takeda.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 Elaprase
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda
    D.2.1.2Country which granted the Marketing AuthorisationIndia
    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.3.4Pharmaceutical form Concentrate for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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
    Hunter syndrome (Mucopolysaccharidosis II, [MPS II])
    E.1.1.1Medical condition in easily understood language
    Hunter syndrome, is a rare, inherited disease caused by a deficiency in an enzyme called iduronate-2-sulfatase. This causes glycosaminoglycans (GAGs) to build up in the body and cause damage.
    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
    To evaluate the safety of Elaprase in pediatric and adult subjects with Hunter syndrome during 53 weeks of study duration.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of Elaprase in pediatric and adult subjects with Hunter syndrome during 53 weeks of study duration.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Male or female Elaprase naïve subjects (and who are not part of any other program at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Hunter syndrome based on the following documented biochemical and genetic criteria:
    - Documented deficiency in iduronate 2-sulfatase (IDS [12S]) enzyme activity of less than or equal to 10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory).
    - A normal enzyme activity level of one other sulfatase as measured in plasma, fibroblasts, or leukocytes (based on normal range of measuring laboratory).
    - The patient has a documented mutation in the IDS gene.
    E.4Principal exclusion criteria
    - Subject has received hematopoietic stem cell transplantation or a bone marrow transplant at any time.
    - Subject is suffering from any comorbid conditions (including hepatic impairment, acute or chronic) or having any other clinical observation or history during the screening examination, which would interfere with the objectives of the study as per investigators judgement.
    - Subject has a chronic kidney disease with estimated Glomerular Filtration rate less than 15 mL/min/1.73 m2 and/or is on dialysis.
    - Subject has participated in any other clinical study or received any investigational compound or non-investigational idursulfase beta within the past 30 days before informed consent
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of AEs, serious AEs (SAEs), treatment emergent AEs, treatment emergent SAEs, adverse drug reactions (ADRs), discontinuation due to AEs, infusion-related reactions, and death.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From start of the study drug administration up to EOS (Week 53)
    E.5.2Secondary end point(s)
    - Change from baseline in percentage Forced Vital Capacity (%FVC) as a measure of respiratory function at Weeks 27 and 53.
    - Change from baseline in 6 Minute Walk Test (6MWT) as a measure of physical functional capacity at Weeks 27 and 53.
    - Change from baseline in Cardiac Left Ventricular Mass Index (LVMI) and Ejection Fraction at Weeks 27 and 53.
    - Change from baseline in liver volume at Weeks 27 and 53.
    - Change from baseline in spleen volume at Weeks 27 and 53.
    - Change from baseline in uGAG levels at Weeks 14, 27, 40, and 53.
    - Change from baseline in Global Joint Range of Motion (JROM) score at Weeks 27 and 53
    - Changes from baseline in anthropometric parameters including height in subjects <18 years at baseline and weight in all subjects at Weeks 27 and 53.
    - Changes from baseline in the health-related quality of life based on Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) (shortened version) and Childhood Health Assessment Questionnaire (CHAQ) in subjects between age of ≥1 year to ≤18 years at Weeks 27 and 53.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, Weeks 27 and 53
    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.8.2.4Number of treatment arms in the trial1
    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
    India
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    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: 5
    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: 1
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Minors
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 5
    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: India
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