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   43974   clinical trials with a EudraCT protocol, of which   7312   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:2012-000042-35
    Sponsor's Protocol Code Number:191622-111
    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:2018-03-12
    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 number2012-000042-35
    A.3Full title of the trial
    BOTOX® Treatment in Pediatric Lower Limb Spasticity: Double-blind Study
    Trattamento con BOTOX in pazienti pediatrici
    affetti da spasticita dell'arto inferiore: Studio in doppio cieco
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of Spasticity in the lower limb in children
    Trattamento della spasticità dell'arto inferiore nei bambini
    A.3.2Name or abbreviated title of the trial where available
    ND
    ND
    A.4.1Sponsor's protocol code number191622-111
    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 Limited EU Regulatory Dept
    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.3.4Pharmaceutical 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.3.10 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for solution for injection
    D.8.4Route of administration of the placeboIntramuscular use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Lower Limb Spasticity
    Spasticità dell'arto inferiore
    E.1.1.1Medical condition in easily understood language
    Lower Limb Spasticity
    Spasticità dell'arto
    inferiore
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10028335
    E.1.2Term Muscle spasticity
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10024132
    E.1.2Term Leg spasticity
    E.1.2System Organ Class 10029205 - Nervous system 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 a single treatment of 2 doses (4 U/kg and 8 U/kg) of BOTOX with standardized PT in pediatric patients with lower limb spasticity.
    Valutare la sicurezza e l'efficacia di un singolo trattamento di 2
    dosi (4 U/kg e 8 U/kg) di BOTOX associato a terapia fisica (TF) standardizzata in pazienti
    pediatrici affetti da spasticità dell'arto inferiore
    E.2.2Secondary objectives of the trial
    None
    Non presenti
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female, 2 to 16 years and 11 months of age (prior to 17th birthday) at the day 1 visit
    • Minimum weight of 10 kg at the screening and day 1 visits
    • Monoplegic or hemiplegic patients with cerebral palsy who have dynamic muscle contracture (spasticity confirmed by Hypertonia Assessment Tool [HAT]) of the ankle plantar flexors associated with true equinus foot deformity as described by Rodda and Graham (2001; see Figure 1 in exclusion criteria). Equinovarus or equinovalgus deformities are acceptable.
    • MAS-B score ≥ 2 for the ankle plantar flexors and minimum range of dorsiflexion of 0 degrees of the ankle with knee fully extended in the study limb at screening and day 1 visits
    • Patients for whom study treatment in the 3 ankle plantar flexors (namely gastrocnemius, soleus, and tibialis posterior) of the study leg is appropriate
    • Gross Motor Function Classification System – Expanded and Revised (GMFCS-E&R) level I to IV at the screening visit
    • Patients who are on anti-spastic medications or muscle relaxants (eg, oral baclofen, tizanidine, dantrolene, scopolamine [oral or patch], vigabatrin, or benzodiazepine therapy) must be on a stable dose and regimen for at least 30 days prior to the day 1 visit
    • Patients who are on anti-epileptic medications must be on a stable dose and regimen for at least 30 days prior to the day 1 visit
    • Written informed consent has been obtained from parent/legally authorized representative
    • Written minor assent has been obtained in accordance with local laws and institutional review board (IRB)/independent ethics committee (IEC) requirements
    • 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 for United States [US] sites and written Data Protection consent for European Union [EU] sites)
    • Negative urine pregnancy test at the screening and day 1 visits (for females of childbearing potential, defined as females post menarche)
    - Soggetti di entrambi i sessi, di età compresa tra 2 e 16 anni e 11 mesi (prima del
    compimento del 17° compleanno) alla visita del giorno 1; - Peso minimo di 10 kg alle visite
    di screening e del giorno 1; Pazienti pediatrici monoplegici o emiplegici affetti da paralisi cerebrale che presentano contrattura muscolare dinamica (spasticità confermata con HAT
    [Hypertonia Assessment Tool]) dei flessori plantari della caviglia associata a reale deformità
    del piede equino come descritto da Rodda e Graham (2001; vedere la Figura 1 nei criteri di
    esclusione). Le deformità da piede equino-varo o equino-valgo sono accettabili; - Punteggio
    MAS-B 2 per i flessori plantari della caviglia e intervallo minimo di dorsiflessione della
    caviglia pari a 0 gradi con ginocchio dell'arto oggetto dello studio completamente esteso alle
    visite di screening e del giorno 1; - Pazienti per i quali il trattamento dello studio nei 3
    flessori plantari della caviglia (ovvero gastrocnemio, soleo e tibiale posteriore) della gamba
    oggetto dello studio è appropriato; - Livello da I a IV secondo il sistema di classificazione
    GMFCS-E&R (Gross Motor Function Classification System Expanded and Revised) alla
    visita di screening; - I pazienti trattati con farmaci antispastici o miorilassanti (ad esempio
    baclofen orale, tizanidina, dantrolene, scopolamina [orale o cerotto], vigabatrin o
    benzodiazepine) devono aver assunto la terapia a una dose e a un regime di
    somministrazione stabile per almeno 30 giorni prima della visita del giorno 1; - I pazienti
    trattati con farmaci antiepilettici devono aver assunto la terapia a una dose e a un regime di
    somministrazione stabile per almeno 30 giorni prima della visita del giorno 1; - Ottenimento
    del consenso informato scritto di un genitore/rappresentante legalmente autorizzato; - Il
    consenso scritto dei minori deve essere stato ottenuto in conformità alle normative locali e ai
    requisiti della Commissione di revisione dell'istituzione/Comitato etico indipendente; - Dove
    applicabile, la documentazione scritta deve essere stata ottenuta in conformità ai requisiti
    nazionali e locali vigenti in materia di privacy (ad es. autorizzazione scritta all'uso e al
    rilascio delle informazioni sanitarie e dello studio di ricerca per i centri negli Stati Uniti
    [USA] e consenso scritto al trattamento dei dati per i centri nell'Unione Europea [UE]); -
    Risultato negativo del test di gravidanza sulle urine alle visite di screening e del giorno 1
    (per i soggetti di sesso femminile potenzialmente fertili, ovvero che abbiano avuto il
    menarca)
    E.4Principal exclusion criteria
    • Any uncontrolled clinically significant medical condition other than the one under study
    • Any medical condition that may put the patient at increased risk with exposure to Botulinum Toxin Type A Purified Neurotoxin Complex, including diagnosed muscular dystrophy (eg, Duchenne’s muscular dystrophy), myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis, mitochondrial disease, or any other significant disease that might interfere with neuromuscular function
    • Fixed contracture of the ankle for the study limb
    • Patients with clinically significant spasticity of the knee flexors in the study limb (defined as either MAS-B knee score higher than MAS-B ankle score [with knee extended] or MAS-B knee score > 2 at the screening or day 1 visit) and require BOTOX injection to the knee flexors during the study
    • Patients with the following gait patterns in the study limb: Type 1 “drop foot” or Type IV “equinus with jump knee, or pelvic rotation, hip flexed, adducted, or internal rotation” (see Figure 1)
    • Uncontrolled epilepsy defined as more than 1 generalized seizure per month within 3 months prior to the day 1 visit 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 the day 1 visit
    • Patients with presence or history of aspiration pneumonia, recurrent lower respiratory tract infections, uncontrolled asthma, or compromised respiratory function within 12 months prior to the day 1 visit that may indicate a vulnerable respiratory state per the investigator’s clinical judgment
    • Patients with presence or history (within 12 months prior to the day 1 visit) of aspiration or a condition(s) that, in the investigator’s opinion, may put the patient at an increased risk for aspiration (eg, significant drooling, chronic dysphagia [difficulty swallowing] requiring changes in diet, or clinically significant gastroesophageal reflux disease)
    • Patients previously exposed to botulinum toxin therapy of any serotype who have a history of allergy or sensitivity to the toxin or its components or other significant adverse experiences that, in the investigator’s opinion, may put the patient at an unacceptable risk
    • Botulinum toxin therapy of any serotype for any condition within 6 months prior to the day 1 visit
    • Patients who are on an active intrathecal baclofen pump therapy within 7 days prior to the day 1 visit or plan to receive such therapy during the study
    • History of treatment with phenol or alcohol block in the study limb within 12 months prior to the day 1 visit or planned treatment with phenol or alcohol block in any limb(s) during the study
    • History of or planned treatment during the study with selective dorsal rhizotomy or deep brain stimulation
    • History of surgical intervention of the lower leg (the knee and below of the study limb; except for tendon lengthening more than 12 months prior to the day 1 visit) or planned surgical intervention of any limb(s) during the study
    • Previous casting within 6 months prior to the day 1 visit or splinting with a dynamic splint (eg, Dynasplint® or UltraFlex) within 3 months prior to the day 1 visit for spasticity of the study limb or the affected upper limb, or planned casting or splinting with a dynamic splint (eg, Dynasplint or UltraFlex)
    for spasticity of the study limb or affected upper limb during the study
    • Females who are pregnant, nursing, or planning a pregnancy during the study
    • Females of childbearing potential (defined as females post menarche) not using reliable means of contraception (see Section 4.5.1.1 for acceptable contraceptive methods)
    - Qualsiasi condizione medica non controllata, clinicamente significativa, oltre a quella
    oggetto di studio; - Qualsiasi condizione medica che potrebbe esporre il paziente a un
    maggior rischio con l'esposizione alla tossina botulinica di tipo A, complesso di neurotossine
    purificato, compresa diagnosi di distrofia muscolare (ad es. distrofia muscolare di
    Duchenne), miastenia gravis, sindrome di Eaton-Lambert, sclerosi laterale amiotrofica,
    malattia mitocondriale o qualsiasi altra patologia significativa che potrebbe interferire con
    la funzionalità neuromuscolare; - Contrattura fissa della caviglia nell'arto oggetto dello
    studio; - Pazienti affetti da spasticità dei flessori del ginocchio clinicamente significativa
    nell'arto oggetto dello studio (punteggio MAS-B del ginocchio maggiore del punteggio
    MAS-B della caviglia [con ginocchio esteso] o punteggio MAS-B del ginocchio >2 alla visita
    di screening o del giorno 1) che necessitano di iniezione di BOTOX nei flessori del ginocchio
    durante lo studio; - Pazienti che presentano i seguenti modelli di andatura nell'arto oggetto
    dello studio: Tipo 1 "piede cadente" o Tipo IV "piede equino con ginocchio del saltatore o
    rotazione pelvica, flessione, adduzione o rotazione interna dell'anca" (vedere la Figura 1); -
    Epilessia non controllata, caratterizzata da più di 1 crisi convulsiva generalizzata al mese nei
    3 mesi precedenti alla visita del giorno 1 o anamnesi di crisi convulsive prolungate o attività
    convulsive ripetitive che necessitano la somministrazione di una benzodiazepina di
    salvataggio (per via orale, rettale, ecc.) più di una volta al mese, crisi convulsive di durata
    superiore a 10 minuti, stato epilettico o epilessia con interessamento autonomico nei 9 mesi
    precedenti alla visita del giorno 1; - Pazienti con presenza o anamnesi di polmonite da
    aspirazione, infezioni ricorrenti delle vie respiratorie inferiori, asma non controllata o
    compromissione della funzionalità respiratoria nei 12 mesi precedenti alla visita del giorno
    1, che, nel giudizio clinico dello sperimentatore, potrebbe essere indicativa di uno stato
    respiratorio vulnerabile; - Pazienti con presenza o anamnesi (nei 12 mesi precedenti alla
    visita del giorno 1) di aspirazione o di una o più condizioni che, secondo il parere dello
    sperimentatore, potrebbero esporre il paziente a un rischio maggiore di aspirazione (ad es.
    salivazione eccessiva, disfagia cronica [difficoltà di deglutizione] che necessiti di
    cambiamenti dell'alimentazione o malattia da reflusso gastroesofageo clinicamente
    significativa); - Pazienti già sottoposti a terapia con tossina botulinica di qualsiasi sierotipo,che presentino anamnesi di allergia o sensibilità alla tossina o ai suoi componenti o altre
    esperienze avverse significative che, secondo il parere dello sperimentatore, potrebbero
    esporre il paziente a un rischio inaccettabile; - Terapia con tossina botulinica di qualsiasi
    sierotipo per qualsiasi condizione nei 6 mesi precedenti alla visita del giorno 1; - Pazienti
    trattati con pompa di infusione intratecale di baclofen nei 7 giorni precedenti la visita del
    giorno 1 o che si prevede vengano sottoposti a tale terapia nel corso dello studio; - Anamnesi
    di trattamento con blocco fenolico o alcolico nell'arto oggetto di studio nei 12 mesi
    precedenti alla visita del giorno 1 o trattamento pianificato con blocco fenolico o alcolico in
    qualsiasi arto durante lo studio; - Anamnesi o previsione di trattamento durante lo studio con
    rizotomia dorsale selettiva o stimolazione cerebrale profonda [fine caratteri disponibili – per
    la lista completa dei criteri di esclusione si prega di fare riferimento al protocollo, paragrafo
    4.4]
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoints will be the average grade change from baseline to weeks 4 and 6 in MAS-B and the average CGI by Physician score at weeks 4 and 6.
    L'endpoint di efficacia primario sarà la variazione media del punteggio MAS B dal Baseline
    alle Settimane 4 e 6 e del punteggio CGI valutato dal medico alle Settimane 4 e 6
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, week 4 and week 6.
    Baseline, Settimane 4 e 6
    E.5.2Secondary end point(s)
    Functional goals, active and passive, using GAS by Physician will be evaluated separately relative to baseline at weeks 8 and 12.

    Secondary efficacy variables of the Modified Tardieu Scale (MTS) include the difference between slow (R2) and fast (R1) range of motion (R2 - R1) as well as respective change from baseline on the MTS for the ankle plantar flexors at weeks 2, 4, 6, 8, and 12.

    Additionally changes from baseline for each R1 and R2 at each assessment point will be evaluated.
    L'endpoint di efficacia secondario include obiettivi funzionali (attivi e passivi) come il
    punteggio GAS (Goal Attainment Scale) valutato dal medico e relativo alla variazione dal
    Baseline alle Settimane 8 e 12 e il punteggio MTS (Modified Tardieu Scale), che ricomprende
    la differenza tra l'intervallo di movimento lento (R2) e veloce (R1) in gradi (R2 R1) così come
    la variazione dal Baseline alle Settimane 2, 4, 6, 8 e 12 del MTS relativo al gruppo muscolare
    principale. Inoltre, verranno valutate le modifiche dell angolo di movimento (R2 e R1) a
    partire dal Baseline e per ogni punto predeterminato di valutazione
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, weeks 2, 4, 6, 8, 12.
    Baseline, Settimane 2, 4, 6, 8, 12.
    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 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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    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
    Brazil
    Canada
    India
    Israel
    Korea, Republic of
    Philippines
    Russian Federation
    South Africa
    Turkey
    Ukraine
    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
    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 months1
    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 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: 412
    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: 206
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 206
    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
    The following may be enrolled but may not be able to give their written consent:
    - very young children
    - subjects with non-severe cognitive impairment related to their cerebral palsy (severe cognitive impairment is excluded by the protocol)
    I seguenti soggetti
    potranno essere arruolati
    senza essere in grado di
    fornire personalmente il
    loro consenso scritto: -
    bambini molto piccoli; -
    soggetti con
    menomazione cognitiva
    non grave relativa a
    paralisi cerebrale
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 104
    F.4.2.2In the whole clinical trial 412
    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)
    Subjects who successfully complete this study without major protocol deviations (eg, noncompliance to protocol-required procedures) will be given the option of enrolling in the open-label extension Study 191622-112 if they meet eligibility criteria. Otherwise subjects would return to the standard of care treatment.
    I soggetti che completeranno con successo lo studio senza gravi deviazioni al protocollo
    avranno l'opportunita di essere arruolati nello studio di estensione in aperto 191622-112, in
    caso rispettino i criteri di inclusione. Diversamente, torneranno alla terapia standard
    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 Decision2013-05-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-06-28
    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 © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA