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    EudraCT Number:2013-003842-18
    Sponsor's Protocol Code Number:A301223
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-01-06
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2013-003842-18
    A.3Full title of the trial
    Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study.
    Preventie van nier- en blaasschade bij kinderen met spina bifida door middel van vroegtijdige injecties met Botulinum-Toxin-A (Botox): een pilotstudie.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study.
    Preventie van nier- en blaasschade bij kinderen met spina bifida door middel van vroegtijdige injecties met Botulinum-Toxin-A (Botox): een pilotstudie.
    A.3.2Name or abbreviated title of the trial where available
    Early Botox in SB patients.
    Vroegtijdige Botox bij SB patiënten.
    A.4.1Sponsor's protocol code numberA301223
    A.5.4Other Identifiers
    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 SponsorErasmus MC
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportJaap Schouten Foundation
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationErasmus MC
    B.5.2Functional name of contact pointP.I. (Dr. J.R. Scheepe)
    B.5.3 Address:
    B.5.3.1Street Address's-Gravendijkwal 230
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 CE
    B.5.4Telephone number+31107036898n.a.
    B.5.5Fax number+31107033968n.a.
    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. name BOTOX
    D. of the Marketing Authorisation holderAllergan Pharmaceuticals Ireland
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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.3.4Pharmaceutical form Intravesical solution/solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravesical use
    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 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
    Neurogenic bladder function disease in spina bifida patients.
    Neurogene blaasfunctiestoornis bij spina bifida patiënten.
    E.1.1.1Medical condition in easily understood language
    Spastic bladder in spina bifida patients.
    Spastische blaas bij patiënten met spina bifida.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10030685
    E.1.2Term Open spina bifida
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10041531
    E.1.2Term Spina bifida without mention of hydrocephalus
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10029280
    E.1.2Term Neurogenic bladder, not otherwise specified
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?
    - Kunnen vroegtijdige Botox-injecties in de blaas bij meningomyelocèle (MMC) patiënten verdere achteruitgang van de blaas- en nierfunctie voorkomen?
    E.2.2Secondary objectives of the trial
    - Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an operation (ileocystoplastic with appendicovesicostoma)?
    - Kunnen vroegtijdige Botox-injecties in de blaas bij MMC patiënten de noodzaak van een grote operatieve ingreep (ileocystoplastiek met appendicovesicostoma) verminderen?
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The study group exists of newborns with MMC who are treated with the standard treatment (CIC and anticholinergics). The minimal age at inclusion is 3 months. This is because of the minimal diameter of the urethra which is necessary for cystoscopy.

    The historical control group exists of MMC patients who have not been treated with Botox and who were born between 1998 and 2008.
    De studiegroep bestaat uit nieuwgeboren kinderen met MMC waarvoor de standaardbehandeling (CIC + anticholinergica) werd gestart. De minimale leeftijd voor inclusie is 3 maanden. Dit in verband met de vereiste minimale diameter van de urethra voor cystoscopie.

    De historische controlegroep bestaat uit MMC patiënten die niet zijn behandeld met Botox en die zijn geboren tussen 1998 en 2008.
    E.4Principal exclusion criteria
    The exclusion criteria for the study group are:
    - Patients with swallowing or breathing problems for which medical treatment is or was necessary.
    - Muscle diseases such as myasthenia gravis, amyotrophic lateral sclerosis or the Syndrome of Eaton-Lambert.

    For the historical group the database of (parents of) patients who object against the use of their medical records for research purposes.
    De exclusiecriteria voor de studiegroep zijn:
    - Patiënten met problemen met slikken of ademhaling waarvoor medische behandeling nodig is of was.
    - Spierziekten zoals myasthenia gravis, amyotrofe laterale sclerose of het Eaton-Lambertsyndroom.

    Voor de historische controlegroep zal worden gecontroleerd of (de ouders van) de patiënten bezwaar hebben gemaakt tegen het gebruik van gegevens voor medisch wetenschappelijk onderzoek.
    E.5 End points
    E.5.1Primary end point(s)
    End filling pressure of the bladder (determined by means of urodynamic testing) after 5 years of follow-up.
    Eindvullingsdruk en capaciteit van de blaas (vastgesteld door middel van een urodynamisch onderzoek) na 5 jaar follow-up.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 5 years of follow-up.
    Na 5 jaar follow-up.
    E.5.2Secondary end point(s)
    - Clinical parameters:
    * Number of urinary tract infections
    * Frequency of intermittent catheterisation
    * Bladder volumes (with intermittent catheterisation)
    - Urodynamic parameters
    * Bladder capacity
    * Bladder overactivities
    * Compliance of the bladder
    - Number and nature of operative interventions to the urinary tract.
    - Klinische parameters:
     * aantal urineweginfecties
     * frequentie van intermitterende katheterisatie
     * blaasvolumen (bij intermitterende katheterisatie)
    - Urodynamische parameters:
     * blaascapaciteit
     * blaasoveractiviteiten
     * compliantie van de blaas
    - Aantal en aard van de operatieve ingrepen aan de urinewegen
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Clinical parameters: continuous
    - Urodynamic parameters: at visits with urodynamic testing (age 2, 8, 17, 26, 35, 44, 53, and 62 months)
    - Number and nature of operative interventions to the urinary tract: continuous.
    - Klinische parameters: continu
    - Urodynamische parameters: tijdens visites met urodynamisch onderzoek (leeftijd 2, 8, 17, 26, 35, 44, 53, en 62 maanden)
    - Aantal en aard van de operatieve ingrepen aan de urinewegen: continu
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    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. trial design description
    historische controlegroep
    historic control group
    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. description
    geen behandeling met BOTOX (historische controlegroep)
    no treatment with BOTOX (historic control group)
    E.8.2.4Number of treatment arms in the trial1
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    Last visit of the last participant.
    Laatste visite van de laatste deelnemer.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years8
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned 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: 10
    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: 10
    F.1.1.5Children (2-11years) No
    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 No
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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 who have benefit from the study treatment can continu the 9-monthly BOTOX-injections after study participation.
    Patiënten die baat hebben bij de studiebehandeling met BOTOX kunnen de 9-maandelijkse BOTOX-injecties ook na afloop van hun studiedeelname ontvangen.
    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 Decision2014-01-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-04-24
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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