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    EudraCT Number:2012-003775-20
    Sponsor's Protocol Code Number:HGT-MLD-071
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-02-14
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedCzechia - SUKL
    A.2EudraCT number2012-003775-20
    A.3Full title of the trial
    An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy
    Otevřené pokračování studie HGT-MLD-070 hodnotící dlouhodobou bezpečnost a účinnost intratekálního podání přípravku HGT-1110 u pacientů s metachromatickou leukodystrofií
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection
    A.4.1Sponsor's protocol code numberHGT-MLD-071
    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 Genetics Therapies 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 supportShire HGT, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Pharmaceutical Company Limited
    B.5.2Functional name of contact pointDavid Whiteman
    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 number0017814829369
    B.5.5Fax number0017812661365
    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/3/10/813
    D.3 Description of the IMP
    D.3.1Product nameSHP611
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot available
    D.3.9.2Current sponsor codeSHP611
    D.3.9.3Other descriptive nameRecombinant Human Arylsulfatase A (rhASA)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    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
    Treatment of Metachromatic Leukodystrophy
    E.1.1.1Medical condition in easily understood language
    Treatment of inherited arylsulfatase A deficiency
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10067609
    E.1.2Term Metachromatic leukodystrophy
    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 collect long-term safety data in patients with MLD who are receiving HGT-1110 and have participated in study HGT-MLD-070 through Week 40.
    E.2.2Secondary objectives of the trial
    - To evaluate the effects of IT administration of HGT-1110 on gross motor function
    - To evaluate the effects of IT administration of HGT-1110 on adaptive behavior
    - To evaluate the effect of IT administration of HGT-1110 on health status and the ability to carry out activities in daily life
    - To assess repeated-dose pharmacokinetics of HGT-1110 in serum
    - To assess concentrations of HGT-1110 in CSF
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient has participated in Study HGT-MLD-070 through Week 40.
    2. Patient must have no safety or medical issues that contraindicate participation
    3. The patient, patient’s parent or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.
    E.4Principal exclusion criteria
    1.The patient is unable to comply with the protocol (eg, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the Investigator.
    2.The patient has any known or suspected hypersensitivity to agents used for anesthesia or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions
    3.The patient is pregnant or breastfeeding.
    4.The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 within 6 months prior to study enrollment or at any time during the study.
    5. The patient has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
    6. The patient’s body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
    7. The patient has a known or suspected local or general infection
    8. The patient is at risk of abnormal bleeding due to a medical condition or therapy
    9. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    10. The patient has a functioning CSF shunt device
    11. The patient has shown an intolerance to an implanted device
    E.5 End points
    E.5.1Primary end point(s)
    Safety will be measured by the following endpoints:
    - Reporting of treatment-emergent adverse events
    - Change from baseline in clinical laboratory testing (serum chemistry including liver function tests, hematology, and urinalysis)
    - Change from baseline in vital signs, physical examinations, and CSF chemistries (including cell counts, glucose, albumin, and protein)
    - Determination of the presence of anti-HGT-1110 antibodies in CSF and/or serum

    E.5.1.1Timepoint(s) of evaluation of this end point
    - Adverse events, vital signs, physical examinations, and CSF chemistries are checked every other week
    - Change from baseline in clinical laboratory testing and determination of the presence of anti-HGT-1110 antibodies at quaterly visits (weeks 52, 66, 78, 92 and 104) and biannually hereafter (weeks 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416, 442, 468, 494, 520, 546, 572, 598 and 624)
    E.5.2Secondary end point(s)
    The secondary endpoints of this study are:
    - Change from baseline at end of study in motor function using the Gross Motor Function Measure-88 (GMFM-88) total score
    - Change from baseline at end of study in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior Scales, Second Edition (VABS II)
    - Change from baseline at end of study in the domain-specific Caregiver Observed MLD Functioning and Outcomes Reporting Tool (COMFORT) scores
    - Repeated-dose pharmacokinetic parameter estimates for HGT-1110 in serum
    - Concentrations of HGT-1110 in CSF at selected time points after repeated investigational drug product administration
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy outcome assessments will be performed quarterly from Week 40 through Week 104 at Weeks 52, 66, 78, 92, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416, 442, 468, 494, 520, 546, 572, 598 and 624. These efficacy outcome assessments are the GMFM-88, GIMF-C, GIMF-S, FEES, the VABS-II, the COMFORT questionnaire, sample collection for biomarker assessments (serum, CSF, and urine).
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    extension to phase I/II trial
    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 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 trial3
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    United Kingdom
    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
    Last patient visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years10
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years10
    E.8.9.2In all countries concerned by the trial months7
    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: 24
    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) Yes
    F. of subjects for this age range: 9
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 15
    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 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 Yes
    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. of subjects incapable of giving consent
    Subjects who are under age and may have mental disabilities
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 24
    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)
    If safety and efficacy is established and the sponsor continues with development of HGT- 1110 the intention is to continue the extension study until study drug is commercially available or another mechanism for managed access is established. This will enable access to the drug by patients beyond the current trial duration.
    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 Decision2014-06-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-02-12
    P. End of Trial
    P.End of Trial StatusOngoing
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