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    Summary
    EudraCT Number:2012-005054-30
    Sponsor's Protocol Code Number:MRZ60201_3070_1
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2015-09-25
    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
    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 ConcernedFrance - ANSM
    A.2EudraCT number2012-005054-30
    A.3Full title of the trial
    Prospective, multicenter, randomized, double-blind, parallel-group, dose-response study of three doses Xeomin® (incobotulinumtoxinA, NT 201) for the treatment of lower limb spasticity in children and adolescents (age 2 - 17 years) with cerebral palsy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Dose-response study of efficacy and safety of Botulinum toxin type A to treat spasticity of the leg(s) in cerebral palsy
    A.4.1Sponsor's protocol code numberMRZ60201_3070_1
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/042/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMerz Pharmaceuticals GmbH
    B.1.3.4CountryGermany
    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 supportMerz Pharmaceuticals GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMerz Pharmaceuticals GmbH
    B.5.2Functional name of contact point
    B.5.6E-mailclinicaltrials@merz.de
    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 Xeomin
    D.2.1.1.2Name of the Marketing Authorisation holder34009 571 886 0 3
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameNT 201
    D.3.2Product code NT 201
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNn.a.
    D.3.9.2Current sponsor codeNT 101
    D.3.9.3Other descriptive nameCLOSTRIDIUM BOTULINUM NEUROTOXIN TYPE A (150KD), FREE OF COMPLEXING PROTEINS
    D.3.9.4EV Substance CodeSUB26174
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Lower limb spasticity due to cerebral palsy
    E.1.1.1Medical condition in easily understood language
    Lower limb spasticity due to cerebral palsy
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10058977
    E.1.2Term Spastic paresis
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The purpose of this study is to determine whether injections of Botulinum toxin type A into muscles of the leg(s) are effective in treating children/adolescents (age 2-17 years) with increased muscle tension/uncontrollable muscle stiffness (spasticity) due to cerebral palsy.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Female or male subject of 2 to 17 years of age (inclusive).
    2. Uni- or bilateral cerebral palsy with clinical need for uni- or bilateral LL injections with BoNT for the treatment of spasticity.
    3. Ashworth Scale [AS] score ≥2 in plantar flexors (at least unilaterally).
    4. Clinical need for a total dose of 16 U/kg BW NT 201 (maximum of 400 U) for the treatment of LL spasticity according to the clinical judgment of the investigator.
    E.4Principal exclusion criteria
    1. Fixed contracture defined as severe restriction of the range of joint movement on passive stretch or predominant forms of muscle hypertonia other than spasticity (e.g., dystonia) in the target limb(s).
    2. Surgery on pes equinus on side(s) intended to be treated with BoNT injections in this study within 12 months prior to Screening Visit (V1), in the screening period or planned for the time of participation in this study.
    3. Hip flexion requiring BoNT injection.
    4. Limitation of hip abduction to less than 40° or pre-diagnosed migrational percentage greater than 30.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline (Day 1) in the AS score of plantar flexors (for subjects with bilateral pes equinus body side to be decided by investigator at screening and to be kept throughout the entire study) at Day 29 (Week 4) of the 1st injection cycle.

    Co-primary efficacy variable (to fulfil the PMC, for US regulatory authorities only):
    • Investigator’s Global Impression of Change of Plantar Flexor Spasticity Scale [GICS-PF] (for subjects with bilateral treatment on same body side as chosen for the primary efficacy variable) at Day 29 (Week 4) of the 1st injection cycle.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 4 of 1st injection cycle
    E.5.2Secondary end point(s)
    - For subjects with bilateral treatment: Change from baseline in the AS score of plantar flexors of the other treated body side at Day 29 (Week 4) of the 1st and 2nd injection cycle.
    - Change from baseline in the AS score of plantar flexors (for subjects with bilateral pes equinus body side to be decided by investigator at screening and to be kept throughout the entire study) at Day 29 (Week 4) of the 2nd injection cycle.
    - Changes from baseline in AS score of plantar flexors (for subjects with bilateral pes equinus body side to be decided by investigator at screening and to be kept throughout the entire study) at Day 57 (Week 8) and Day 85 (Week 12) of the 1st and of the 2nd injection cycle.
    - For subjects with unilateral treatment: Changes from baseline in AS score of knee flexors or thigh adductors at Day 29 (Week 4) of the 1st and of the 2nd injection cycle.
    - Changes from baseline in modified Tardieu Scale [MTS] of plantar flexors (for subjects with bilateral pes equinus body side to be decided by investigator at screening and to be kept throughout the entire study) at Day 29 (Week 4), Day 57 (Week 8), and Day 85 (Week 12) of the 1st and of the 2nd injection cycle.
    - Investigator’s, Child’s/Adolescent’s, and Parent’s/Caregiver’s Global Impression of Change Scale [GICS] at Day 29 (Week 4) of the 1st and 2nd injection cycle.
    - Investigator’s Global Impression of Change of Plantar Flexor Spasticity [GICS-PF] (for subjects with bilateral treatment on same body side as chosen for the primary efficacy variable) at Day 29 (Week 4) of the 1st and 2nd injection cycle.
    - Changes from baseline in Gross Motor Function Measure [GMFM]-66 score at the End of 1st Cycle Visit V6 and at the End of Study Visit V11.
    - Change in scores of pain intensity (from subjects) and pain frequency (from parent/caregiver) assessed with ‘Questionnaire on Pain caused by Spasticity [QPS]’ to all post baseline visits of the 1st and of the 2nd injection cycle.
    - Time to reinjection for each of the three dose groups for the 1st and 2nd injection cycle.
    E.5.2.1Timepoint(s) of evaluation of this end point
    between week 4 and week 72 (for details see definition of endpoints)
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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) 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    dose-response study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    same IMP, 3 different doses arms
    E.8.2.4Number of treatment arms in the trial3
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    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
    Austria
    Czech Republic
    Estonia
    France
    Germany
    Israel
    Korea, Republic of
    Poland
    Romania
    Russian Federation
    Slovakia
    South Africa
    Spain
    Turkey
    Ukraine
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    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: 300
    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: 180
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 120
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    underage subjects and underage subjects with intellectual disability
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state22
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 160
    F.4.2.2In the whole clinical trial 300
    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)
    No specific post-study arrangements are made and no specific post-study care will be performed after this study, however subjects with duration of both injection cycles between 12 and 16 weeks may subsequently enroll into the open-label long-term study MRZ60201_3071_1 in which all subjects receive 4 injection treatments with active treatment. Recruitment in MRZ60201_3071_1 is competitive with newly recruited subjects, so enrollment can not be guaranteed.
    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 Decision2014-10-16
    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
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