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    Summary
    EudraCT Number:2014-004804-31
    Sponsor's Protocol Code Number:SHP-ELA-401
    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:2015-08-06
    Trial 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 number2014-004804-31
    A.3Full title of the trial
    A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients with MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long-term study on changes in height and weight of children with MPS II receiving Elaprase and who started the treatment before the age of 6 years.
    A.4.1Sponsor's protocol code numberSHP-ELA-401
    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 SponsorShire Human Genetic Therapies, Inc. (Shire), a wholly owned subsidiary of Takeda Pharmaceutical Company Limited
    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 supportShire Human Genetic Therapies, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Human Genetic Therapies, Inc
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number001 617 551 8768
    B.5.5Fax number001 781 482 1820
    B.5.6E-mailkriszina.kisfalvi@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 holderShire Human Genetic Therapies AB, Svärdvägen 11D, 182 33 Danderyd, Sweden
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/01/078
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIDURSULFASE
    D.3.9.1CAS number 50936-59-9
    D.3.9.4EV Substance CodeSUB22927
    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
    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
    The primary objectives of this study are to assess longitudinal changes in the following
    parameters in patients with MPS II who began Elaprase treatment at <6 years of age and who are receiving treatment with Elaprase:
    • height
    • weight
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to assess longitudinal changes in the following
    parameters in patients with MPS II who began Elaprase treatment at <6 years of age and who are receiving treatment with Elaprase:
    • urinary GAG levels
    • liver and spleen volume
    • joint mobility, as measured by Joint Range of Motion (JROM) scores, including global,
    upper-limb, and lower-limb joint scores
    • distance walked, as measured by the 6 Minute Walk Test (6MWT)
    • quality of life, as measured by the Hunter-syndrome Functional Outcome in Clinical
    Understanding Scale (HS-FOCUS) questionnaire (shortened version)
    • impact of illness on ability to function in daily life, as measured by the Childhood Health
    Assessment Questionnaire (CHAQ)
    • adaptive behavior as measured by the Vineland Adaptive Behavior Scales (VABS-II)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet ALL of the inclusion criteria for his group in order to enroll in the trial.
    1. The patient is male.
    2. The patient is Elaprase-naïve at study entry.
    3. The patient must have a documented diagnosis of MPS II. Of the 3 criteria below, the
    combinations (3a AND 3b) or (3a AND 3c) will be accepted as diagnostic of MPS II:
    a. The patient has a deficiency in I2S enzyme activity of ≤10% of the lower limit of
    the normal range as measured in plasma, fibroblasts, or leukocytes (based on the
    reference laboratory’s normal range).
    AND
    b. The patient has a documented mutation in the I2S gene.
    OR
    c. The patient has a normal enzyme activity level of one other sulfatase as measured
    in plasma, fibroblasts, or leukocytes (based on the normal range of measuring
    laboratory).
    4. The patient will be <6 years of age at the start of Elaprase treatment.
    5. The patient, patient’s parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved
    informed consent form after all relevant aspects of the study have been explained and
    discussed. Consent of the patient's parent(s) or legally authorized guardian(s) and the
    patient’s assent, as relevant, must be obtained
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria are not eligible for enrollment into this study.
    1. The patient has received treatment with any investigational drug or device within the 30 days prior to study entry.
    2. The patient has received or is receiving treatment with idursulfase-IT.
    3. The patient has received growth hormones, a cord blood infusion, or a bone marrow
    transplant at any time.
    4. The patient has received blood product transfusions within 90 days prior to Screening.
    5. The patient is unable to comply with the protocol as determined by the Investigator.
    6. The patient has a history of severe or life-threatening hypersensitivity to the active substance or to any of the excipients if hypersensitivity is not controllable.
    E.5 End points
    E.5.1Primary end point(s)
    Following are the primary variables to be assessed in this study:
    • Height and weight
    • Height and weight Z-scores
    • Safety assessments
    E.5.1.1Timepoint(s) of evaluation of this end point
    Data will be collected from the baseline visit and throughout the study and evaluated following the End-of-study visit.
    E.5.2Secondary end point(s)
    Changes in the following additional variables will be assessed in this study:
    • Urinary GAG (uGAG) levels normalized to urine creatinine
    • Normalized uGAG divided by upper limit of normal for age (uGAG/ULN)
    • Joint mobility, as measured by JROM scores, including global, upper-limb, and lower-limb joint scores
    • Distance walked, as measured by the 6MWT
    • Quality of life, as measured by the HS-FOCUS (shortened version), including individual
    domain scores from the 5 functional domains: walking/standing, grip/reach, schooling/work, activities, and breathing
    • Impact of illness on ability to function in daily life, as measured by the CHAQ Parent Report, including the Disability Index (based on 8 subscales: dressing, hygiene, arising, eating, walking, reach, grip and activities), Discomfort Index and Health Status index
    • Adaptive behavior, as measured by the VABS- II: standardized scores for each of
    4 domains: Communication; Daily Living Skills; Socialization; Motor Skills; as well as the
    Adaptive Behavior Composite (ABC) score
    • Antibody status
    E.5.2.1Timepoint(s) of evaluation of this end point
    Data will be collected from the baseline visit and throughout the study and evaluated following the End-of-study visit.
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    This study is designed to evaluate growth in patients with MPS II who initiate treatment with Elaprase at <6 years of age.
    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 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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
    Costa Rica
    Dominican Republic
    Malaysia
    Oman
    Philippines
    Saudi Arabia
    Serbia
    Thailand
    United States
    Viet Nam
    Germany
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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: 20
    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: 20
    F.1.1.6Adolescents (12-17 years) No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children for whom the parent(s) or legal guardian will need to give consent and the child itself assent as required.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 3
    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)
    Once the participation of a subject in the trial has ended, the long term care of the participant will remain the responsibility of the primary treating physician.
    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 Decision2015-12-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-04-12
    P. End of Trial
    P.End of Trial StatusCompleted
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