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    Summary
    EudraCT Number:2016-000630-22
    Sponsor's Protocol Code Number:TV50717-CNS-30047
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2018-09-18
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-000630-22
    A.3Full title of the trial
    An Open-Label, Long-Term Safety Study Including a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period of TEV-50717 (Deutetrabenazine) for the Treatment of Tourette Syndrome in Children and Adolescents
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study in children and adolescents with Tourette Syndrome to investigate the safety of TEV-50717 (experimental drug).
    A.4.1Sponsor's protocol code numberTV50717-CNS-30047
    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 SponsorTeva Branded Pharmaceutical Products R&D, 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 supportNuvelution TS Pharma, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNuvelution TS Pharma, Inc.
    B.5.2Functional name of contact pointElizabeth Garofalo, MD
    B.5.3 Address:
    B.5.3.1Street Address101 Main Street,
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1734-478-9697
    B.5.6E-mailinfo@nvtpharma.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 No
    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.3.2Product code TEV-50717
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeutetrabenazine
    D.3.9.1CAS number 1392826-25 3
    D.3.9.2Current sponsor codeTEV-50717
    D.3.9.3Other descriptive nameDEUTETRABENAZINE
    D.3.9.4EV Substance CodeSUB179485
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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.IMP: 2
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code TEV-50717
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeuterabenazine
    D.3.9.1CAS number 1392826-25 3
    D.3.9.2Current sponsor codeTEV-50717
    D.3.9.3Other descriptive nameDEUTETRABENAZINE
    D.3.9.4EV Substance CodeSUB179485
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number9
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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.IMP: 3
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code TEV-50717
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeuterabenazine
    D.3.9.1CAS number 1392826-25 3
    D.3.9.2Current sponsor codeTEV-50717
    D.3.9.3Other descriptive nameDEUTETRABENAZINE
    D.3.9.4EV Substance CodeSUB179485
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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.IMP: 4
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code TEV-50717
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeuterabenazine
    D.3.9.1CAS number 1392826-25 3
    D.3.9.2Current sponsor codeTEV-50717
    D.3.9.3Other descriptive nameDEUTETRABENAZINE
    D.3.9.4EV Substance CodeSUB179485
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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.IMP: 5
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code TEV-50717
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDeuterabenazine
    D.3.9.1CAS number 1392826-25 3
    D.3.9.2Current sponsor codeTEV-50717
    D.3.9.3Other descriptive nameDEUTETRABENAZINE
    D.3.9.4EV Substance CodeSUB179485
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number18
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 3
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 4
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 5
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Tics associated with Tourette Syndrome(TS)
    E.1.1.1Medical condition in easily understood language
    Tics associated with Tourette Syndrome(TS)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    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 objective of this study is to evaluate the safety and tolerability of long-term therapy with TEV-50717.
    E.2.2Secondary objectives of the trial
    The secondary objective of this study are:
    •to evaluate the efficacy of long-term therapy with TEV-50717 in reducing the severity of TS tics
    •to confirm long-term maintenance of effect by means of a double-blind, placebo-controlled, randomized drug withdrawal period after 28 weeks of open-label treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    a. Patient is younger than 18 years of age on day 1.
    b. Patient weighs at least 44 pounds (20 kg) on day 1.
    c. Patient is able to swallow IMP whole.
    d. Patient and caregiver/adult are willing to adhere to IMP regimen and comply with all
    study procedures.
    e. Patient is in good general health, as indicated by medical and psychiatric history as
    well as physical and neurological examination.
    f. In the investigator’s opinion, the patient and caregiver/adult have the ability to
    understand the nature of the study and its procedures, and the patient is expected to
    complete the study as designed.
    g. Patient and caregiver/adult provide written informed consent/assent, depending on the
    child’s age, as appropriate, according to local regulations.
    h. Females who are postmenarchal or ≥12 years of age may be included only if they
    have a negative beta-human chorionic gonadotropin (β-HCG) test on day 1 or are
    sterile. Definitions of sterile are given in Appendix L.
    i. Females who are postmenarchal or ≥12 years of age whose male partners are
    potentially fertile (ie, no vasectomy) must use highly effective birth control methods
    for the duration of the study (ie, starting at screening) and for 30 days or 5 half-lives,
    whichever is longer after last dose of IMP. Further details are included in
    Appendix L.
    E.4Principal exclusion criteria
    a. Patient is 18 years of age or older.
    b. Patient has a neurologic disorder other than TS that could obscure
    the evaluation of tics.
    c. The patient's predominant movement disorder is stereotypy
    (coordinated movements that repeat continually and identically)
    associated with autism spectrum disorder.
    d. Patient has a confirmed diagnosis of bipolar disorder, schizophrenia,
    or another psychotic disorder.
    e. Patient has clinically significant depression at screening or day 1.
    f. Patient has a history of suicidal intent or related behaviors within 2
    years of screening:
     previous intent to act on suicidal ideation with a specific plan,
    irrespective of level of ambivalence, at the time of suicidal thought
     previous suicidal preparatory acts or behavior
    g. Patient has a history of a previous actual, interrupted, or aborted
    suicide attempt.
    h. Patient has a first-degree relative who has completed suicide.
    i. Patient has clinically significant obsessive-compulsive disorder (OCD)
    on day 1 that, in the opinion of the investigator, is the primary cause of
    impairment.
    j. Patient has received comprehensive behavioral intervention for tics
    for TS or cognitive behavioral therapy for OCD within 4 weeks of
    screening.
    k. Patient has received any of the following concomitant medications
    for tics within the specified exclusionary windows of screening prior to
    dosing for washout:
     within 3 months: depot neuroleptics, botulinum toxin, or
    tetrabenazine
     within 4 weeks: cannabidiol oil or valbenazine
     within 21 days: reserpine
     within 14 days: neuroleptics (oral), typical and atypical
    antipsychotics (see Appendix A, Table 7), metoclopramide, levodopa,
    and dopamine agonists
    Note: Use of stimulant medications, including amphetamine,
    methylphenidate, and lisdexamfetamine, is allowed if primary use is for
    the treatment of ADHD, dosing has been stable for at least 2 weeks
    before screening.
    Note: Use of atomoxetine is allowed if the primary use is for the
    treatment of ADHD, dosing has been stable for at least 4 weeks before
    screening.
    Note: Use of benzodiazepines is allowed if the primary use is not for tics,
    and dosing has been stable for at least 4 weeks before screening.
    Note: Use of topiramate (up to 200 mg/day) is allowed if the dosing has
    been stable for at least 4 weeks before screening.
    Note: Use of guanfacine or clonidine is allowed regardless of indication
    (ie, if prescribed for tics or Tourette syndrome) if the dosing has been
    stable for at least 4 weeks before screening and no changes to dose or
    frequency are anticipated during the course of the study.
    l. Patient has an unstable or serious medical illness at screening or day 1.
    m. Patient has a QT interval corrected for heart rate using Fridericia's
    formula (QTcF) interval value >450 msec (males) or >460 msec
    (females), or >480 msec (with right bundle branch block) on 12-lead
    ECG at screening. Patient requires treatment with drugs known to
    prolong the QT interval (see Appendix A Table 8 for a complete list ofprohibited QT-prolonging drugs).
    n. Patients with a history of torsade de pointes, congenital long QT
    syndrome, bradyarrhythmias, or uncompensated heart failure.
    o. Patient has evidence of hepatic impairment, as indicated by:
     aspartate aminotransferase (AST) or alanine aminotransferase (ALT)
    >2.5 × the upper limit of the normal range (ULN) at screening
     alkaline phosphatase (ALP) or total bilirubin (Tbil) >2 × ULN at
    screening
    Note: Patients with Gilbert's Syndrome are eligible to participate if
    approved by the medical monitor.
    Note: Patients with abnormalities in 2 or more of the following clinical
    laboratory parameters must be approved for enrollment by the medical
    monitor: AST, ALT, ALP, and Tbil.
    p. Patient has evidence of clinically significant renal impairment,
    indicated by a serum creatinine >1.5 × ULN at screening.
    q. Patient has received a monoamine oxidase inhibitor within 14 days of
    the day 1 visit.
    r. Patient has a known allergy to any of the components of the IMP.
    s. Patient has participated in an investigational drug or device study
    (with the exception of Study SD 809 C 17, Study TV50717-CNS 30046, or
    Study TV50717-CNS 30060) and received IMP/intervention within 30
    days.
    t. The patient is a pregnant or lactating female or plans to become
    pregnant during the study.
    u. Patient has a history of, or acknowledges, alcohol-related disorder in
    the previous 12 months, as defined in the Diagnostic and Statistical
    Manual of Mental Disorders, Fifth Edition (DSM-V™).
    v. Patient has a positive urine drug screen test result or is unable to
    refrain from substance abuse throughout the study.
    w. Patient has a DSM diagnosis based on the MINI Kid modules
    performed at screening that, in the opinion of the investigator, makes
    the patient unsuitable for the study.
    For complete wording refer to Protocol Amendment 4.
    E.5 End points
    E.5.1Primary end point(s)
    Safety endpoints Part A:
    •incidence of adverse events
    •observed values and changes from day 1 in vital signs
    •observed values and changes from day 1 in the Children’s Depression Inventory, Second Edition (CDI 2; Parent and Self-report versions)
    •observed values in the Children’s Columbia-Suicide Severity Rating Scale (C-SSRS)
    •observed values in electrocardiogram (ECG) parameters and shifts from day 1 for clinically significant abnormal findings
    •observed values and changes from day 1 in clinical laboratory parameters (hematology, serum chemistry, and urinalysis)

    The following safety endpoints will be assessed in Part B (Blinded, Randomized Drug Withdrawal Period and Titration Post-Drug Withdrawal):
    •incidence of adverse events
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A: Baseline to Week 54
    Part B: week 28 to Week 30
    E.5.2Secondary end point(s)
    Efficacy Endpoints:
    Part A (Day 1, Titration, and Maintenance):
    •change in the Total Tic Score (TTS) of the Yale Global Tic Severity Scale (YGTSS) from day 1 to each visit in which the scale is administered
    •change in the Tourette Syndrome-Clinical Global Impression (TS-CGI) score from day 1 to each visit in which the scale is administered
    •change in the Tourette Syndrome-Patient Global Impression of Impact (TS-PGII) score from day 1 to each visit in which the scale is administered
    •change in the Child and Adolescent Gilles de la Tourette Syndrome – Quality of Life (C&A-GTS QOL) activities of daily living (ADL) subscale score from day 1 to each visit in which the scale is administered

    Part B (Blinded, Randomized Drug Withdrawal Period and Titration Post-Drug Withdrawal):
    •change in the TTS of the YGTSS from week 28 to week 30
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy endpoints
    Part A: day 1 to each visit in which the scale is administered
    Part B: week 28 to week 30
    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) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Study Includes a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period between week 28-30
    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 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Argentina
    Australia
    Canada
    Colombia
    Denmark
    France
    Hungary
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    Poland
    Romania
    Russian Federation
    Serbia
    Spain
    Sweden
    Ukraine
    United States
    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
    The end of study is defined as the date of the week 56 visit of the last participant
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months30
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months30
    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: 227
    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: 113
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 114
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 227
    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)
    Patients will have a follow-up telephone contact to evaluate safety 1 week after the end of the washout period (2 weeks after their last dose of IMP).
    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 Decision2018-09-18
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
    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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