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    Summary
    EudraCT Number:2014-000464-17
    Sponsor's Protocol Code Number:191622-137
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-23
    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 number2014-000464-17
    A.3Full title of the trial
    BOTOX® in the Treatment of Urinary Incontinence Due to Overactive Bladder in Patients 12 to 17 Years of Age
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    BOTOX® in the Treatment of Urinary Incontinence Due to Overactive Bladder in Patients 12 to 17 Years of Age
    A.4.1Sponsor's protocol code number191622-137
    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 SponsorAllergan Limited
    B.1.3.4CountryUnited Kingdom
    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 supportAllergan Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAllergan Limited
    B.5.2Functional name of contact pointAllergan Ltd. EU Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address1st Floor, Marlow International, The Parkway
    B.5.3.2Town/ cityMarlow, Buckinghamshire
    B.5.3.3Post codeSL7 1YL
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number441628 494444
    B.5.5Fax number441628 494449
    B.5.6E-mailml-eu_reg_affairs@allergan.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 BOTOX®
    D.2.1.1.2Name of the Marketing Authorisation holderAllergan Pharmaceuticals Ireland
    D.2.1.2Country which granted the Marketing AuthorisationIreland
    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 nameBOTOX®
    D.3.2Product code 9060X
    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 INNBOTULINUM TOXIN TYPE A
    D.3.9.1CAS number 93384-43-1
    D.3.9.4EV Substance CodeSUB13117MIG
    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 Yes
    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
    Urinary Incontinence Due to Overactive Bladder
    E.1.1.1Medical condition in easily understood language
    Urinary Incontinence Due to Overactive Bladder
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10046543
    E.1.2Term Urinary incontinence
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and efficacy of BOTOX for the treatment of urinary incontinence due to OAB in patients 12 to 17 years of age who have not been adequately managed with anticholinergic therapy. To evaluate the safety and efficacy of repeated BOTOX treatments in this patient population.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. written informed consent has been obtained from the legally authorized representative and written minor assent has been obtained, in accordance with local laws and institutional review board (IRB)/independent ethics committee (IEC) requirements

    2. written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable (eg, Written Authorization for Use and Release of Health and Research Study Information (US sites) and written Data Protection consent (European sites)

    3. male or female, aged ≥ 12 years to ≤ 17 years of age at the time of informed consent

    4. patient has symptoms of OAB (frequency and urgency) with urinary incontinence for a period of at least 6 months immediately prior to screening, determined by patient history

    5. patient experiences a total of ≥ 2 episodes of daytime urinary urgency incontinence in the 2-day patient bladder diary completed during the screening period (daytime is defined as time between waking up to start the day and going to bed to sleep for the
    night)

    6. patient experiences urinary frequency, defined as an average of ≥ 8 micturitions (toilet voids) per day ie, a total of ≥ 16 micturitions in the 2-day patient bladder diary completed during the screening period

    7. patient has not been adequately managed with 1 or more anticholinergic agents for the treatment of OAB in the opinion of the investigator. This includes patients who are still incontinent despite anticholinergic therapy, experiencing intolerable side effects, or are unwilling to continue to take the medication for any reason.

    8. patient is willing and able to use CIC to empty the bladder at any time after study treatment if it is determined to be necessary by the investigator

    9. patient agrees to a minimum fluid intake of 1500 mL/m2 BSA, not to exceed 3000 mL/m2 BSA per day, during the patient bladder diary completion days at screening and prior to clinic visits during the study

    10. negative urine pregnancy test for females who are postmenarche

    11. patient is able to complete study requirements including completion of bladder diaries and HRQOL questionnaires (these can also be completed by the parent/legally authorized representative), and is likely to attend study visits in the opinion of the investigator
    E.4Principal exclusion criteria
    1. patient has an uncontrolled systemic disease, or has previous or current diagnosis of malignancy

    2. patient has symptoms of OAB due to any known neurological reason (eg, spina bifida, spinal cord injury, or cerebral palsy)

    3. patient has a history of 2 or more UTIs treated with antibiotics within 6 months of randomization/day 1 or is taking prophylactic antibiotics to prevent chronic UTI

    4. patient has a history or evidence of any pelvic or urological abnormalities, except OAB, including:
    o bladder neck surgery resulting in an open bladder neck, or reconstructive surgery of the lower urinary tract (eg, urostomy, urinary diversion, or bladder augmentation)
    o anatomical evidence of bladder outlet obstruction (including functional outlet obstruction), urethral or urethral valve obstruction/stricture at screening
    o surgery of the urinary tract (including minimally invasive surgery) within 6 months of screening (except those listed above which are exclusionary for any time period)
    o circumcision within 1 month of screening
    o clinically relevant kidney abnormality, or clinically relevant vesicoureteric reflux, or disease of the bladder (other than OAB) that
    may affect bladder function

    5. patient has predominance of stress incontinence, or “giggle” incontinence, or any condition other than OAB that in the investigator’s opinion may account for the patient being incontinent

    6. patient has unmanaged, unresolved bowel problems (eg, constipation, encopresis)

    7. patient currently uses or plans to use medications or therapies to treat symptoms of OAB, including nocturnal enuresis or nocturia.
    Patients previously receiving these medications must have discontinued their use prior to the start of the first screening procedure as follows:
    o for desmopressin, at least one day prior
    o for anticholinergic therapy, at least 7 days prior
    o for intravesical anticholinergic therapy, at least 4 weeks prior
    o for mirabegron, at least 14 days prior

    8. patient currently uses or plans to use an implantable or nonimplantable electrostimulation/neuromodulation device for treatment of OAB. (If a nonimplantable device is used, it must be discontinued at least 7 days prior to the first screening procedure; if a device is implanted, it must be inactive for at least 4 weeks prior to the first screening procedure; neither should be used during the study.)

    9. patient plans to start using psychiatric medications or medications for attention deficit hyperactivity disorder during the study. If the patient is already using such medications they should be on a stable dose prior to randomization/day 1 and agree to remain on the same dose during the study if possible when medically indicated.

    10. patient uses CIC or an indwelling catheter to manage their OAB

    11. patient has had previous or current botulinum toxin therapy of any serotype for any urological condition, or treatment with botulinum toxin of any serotype within 3 months of randomization/day 1 for any other condition or use

    12. patient has been treated with intravesical capsaicin or resiniferatoxin within 12 months of screening

    13. patient has a PVR urine volume of > 40 mL at screening. The PVR measurement can be repeated once on the same day; the patient is to be excluded if the repeated measure is above 40 mL.

    14. patient has a daytime (waking hours) total volume of urine voided > 3000 mL, collected over one daytime period during the 2-day bladder diary collection period prior to randomization/day 1

    15. postmenarche female patients of childbearing potential who are pregnant, nursing, or planning to become pregnant during the study (postmenarche female patients must also either be sexually abstinent or use another acceptable form of contraception)

    16. patient has known allergy or sensitivity to components of any botulinum toxin preparation (including the study medication preparation), anesthetics or antibiotics to be used during the study

    17. patient has hemophilia, or other clotting factor deficiencies, or disorders that cause bleeding diathesis

    18. patient cannot withhold any antiplatelet, anticoagulant therapy, or other medications with anticoagulant effects for 3 days prior to randomization/day 1. Note: some medications may need to be withheld for >3 days, per clinical judgment of the investigator

    19. patient has any medical condition that may put them at increased risk with exposure to BOTOX including diagnosed myasthenia gravis, Eaton-Lambert syndrome, or amyotrophic lateral sclerosis

    20. current enrollment in an investigational drug or device study, or
    participation in such a study within 30 days of entry into this study (or longer if local requirements specify)

    21. patient has a condition or is in a situation which in the investigator's opinion may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient's participation in the study
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy measure is the number of daytime urinary incontinence episodes as recorded in the 2-day bladder diary during the week preceding each study visit. The primary timepoint is week 12 after treatment 1. For patients who receive multiple treatments in this study, the timepoint of main focus is week 12 after each treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 after each treatment
    E.5.2Secondary end point(s)
    The following secondary efficacy measures will be collected after each treatment using the bladder diary:
    • number of daytime micturition episodes
    • number of daytime urgency episodes
    • presence or absence of night time urinary incontinence
    • volume voided per micturition

    In addition, the following duration of effect measures are considered secondary efficacy
    measures:
    • time to patient request and time to patient qualification for retreatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    Daily after each treatment
    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 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 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 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 EEA24
    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
    Australia
    Belgium
    Canada
    Czech Republic
    France
    Germany
    Hong Kong
    Italy
    Netherlands
    Norway
    Poland
    South Africa
    Taiwan
    United Kingdom
    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
    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 months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    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: 108
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 108
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 71
    F.4.2.2In the whole clinical trial 108
    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)
    Standard of care
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Medicines for Children Network (MCRN)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-10
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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