Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43846   clinical trials with a EudraCT protocol, of which   7282   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2012-004877-26
    Sponsor's Protocol Code Number:191622-120
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-06-06
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2012-004877-26
    A.3Full title of the trial
    BOTOX® in the Treatment of Urinary Incontinence Due to Neurogenic Detrusor Overactivity in Patients 8 to 17 Years of Age
    BOTOX® nel trattamento dell'incontinenza urinaria dovuta a iperattività detrusoriale neurogena in pazienti di età compresa tra 8 e 17 anni
    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 Neurogenic Detrusor Overactivity in Patients 8 to 17 Years of Age
    BOTOX® nel trattamento dell'incontinenza urinaria dovuta a iperattività detrusoriale neurogena in pazienti di età compresa tra 8 e 17 anni
    A.4.1Sponsor's protocol code number191622-120
    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 number+441628494444
    B.5.5Fax number+441628494449
    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.3.9.2Current sponsor codeAGN 191622
    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 Neurogenic Detrusor Overactivity
    E.1.1.1Medical condition in easily understood language
    Urinary Incontinence Due to Neurogenic Detrusor Overactivity
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    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
    The objective of this study is to evaluate the safety and efficacy of BOTOX for the treatment of urinary incontinence due to Neurogenic Detrusor Overactivity (NDO) in patients 8 to 17 years of age who have not been adequately managed with anticholinergic therapy.
    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 ≥ 8 years to ≤ 17 years of age at the time of informed consent.

    4. patient has NDO based on either:
    - presence of an IDC during the urodynamic assessment performed in the screening period or on day 1 (prior to randomization), or
    - documented presence of an IDC from an historical urodynamic assessment within 12 months of screening

    5. patient has NDO due to either:
    - spinal dysraphism, which includes spina bifida and all forms of tethered cord (eg, myelomeningocele, meningocele), or
    - acquired NDO from a spinal cord injury, with the injury having occurred at least 6 months prior to screening, or
    - acquired NDO due to transverse myelitis with diagnosis at least 18 monthsprior to screening

    6. spinal lesion/injury must be at thoracic level T1 or below

    7. patient must be regularly using CIC to empty the bladder (CIC can be performed by either the patient or the parent/caregiver):
    - the CIC schedule must be at least 3 times per day
    -CIC must have been initiated at least 3 months prior to screening

    8. patient has a total of ≥ 4 daytime urinary incontinence episodes over the 2-day bladder diary completed during the screening period, despite regular CIC(daytime is defined as time between waking up to start the day and going to bed to sleep for the night)

    9. patient has not been adequately managed with 1 or more anticholinergic agents for the treatment of NDO 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:
    - if continuing to take anticholinergics, patients should be willing to
    maintain a stable dose throughout the study (this does not include
    intravesical anticholinergics, which are not permitted). The stable dose, as determined by the investigator, should have been established prior to the start of screening.
    - if not continuing to take anticholinergics, patients should stop their use for at least 7 days prior to the start of the screening bladder diary.

    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/caregiver), and is likely to attend study visits in the opinion of the investigator
    E.4Principal exclusion criteria
    1. patient has an uncontrolled systemic disease, previous or current diagnosis of malignancy.

    2. patient has had surgery of the spinal cord within 6 months of screening.

    3. patient has a history or evidence of any pelvic or urological abnormalities, except NDO, including:
    o bladder neck surgery resulting in an open bladder neck, or reconstructive surgery of the lower urinary tract (eg, urostomy, urinary diversion)
    o anatomical evidence of bladder 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
    4. patient has cerebral palsy
    5. patient has uncontrolled epilepsy, defined as:
    o more than 1 generalized seizure per month within 3 months prior to screening, or history of prolonged seizures or repetitive seizure activity requiring administration of a rescue benzodiazepine (oral, rectal, etc) more than once a month, seizures lasting more than 10 minutes, status epilepticus, or epilepsy with autonomic involvement within 9 months
    prior to screening.

    6. patient has history of dysphagia, aspiration pneumonia, or significant lung disease (eg, bronchiectasis)

    7. patient has predominance of stress incontinence, in the opinion of the investigator

    8. patient currently uses or plans to use a baclofen pump

    9. patient currently uses or plans to use an implantable or nonimplantable electrostimulation/neuromodulation device for treatment of NDO. (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).

    10. patient uses an indwelling catheter for treatment of NDO

    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 been treated with an intravesical anticholinergic within 4 weeks of screening

    14. patient has a 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

    15. postmenarche female patients 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 – see Section 4.5.1.1)

    16. 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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary timepoint is week 6 after treatment.
    E.5.2Secondary end point(s)
    There are several urodynamic secondary efficacy measures; a central reviewer will determine the final value.

    • MCC (mL)
    -the MCC will also be presented as a proportion of the expected bladder capacity (EBC), where EBC is calculated as: (30 + [age in years × 30]) for patients who have volumes below the adult volume of 500 mL (age is at the time of the assessment) (Nevéus et al, 2006)
    • presence or absence of an IDC
    • if an IDC is present, maximum detrusor pressure during the first IDC (PdetMax1stIDC) (cm H2O)
    • maximum detrusor pressure during the storage phase (PdetMax) (cm H2O)
    • detrusor leak point pressure (DLPP) (cm H2O)
    • urine volume at first morning catheterization (mL)
    • presence or absence of night time urinary incontinence

    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
    The secondary timepoint is 6 weeks.
    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 concerned3
    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
    Austria
    Belgium
    Canada
    Czech Republic
    France
    Italy
    Poland
    Russian Federation
    Turkey
    United States
    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 months8
    E.8.9.1In the Member State concerned days31
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days31
    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: 132
    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: 66
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 66
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 66
    F.4.2.2In the whole clinical trial 132
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-10-11
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri Apr 19 16:31:27 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA