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    Summary
    EudraCT Number:2012-000062-38
    Sponsor's Protocol Code Number:191622-101
    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:2018-03-09
    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 number2012-000062-38
    A.3Full title of the trial
    BOTOX® Treatment in Pediatric Upper Limb Spasticity: Double-blind Study
    Trattamento con BOTOX in pazienti pediatrici affetti da spasticita dell'arto superiore: 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 upper limb in children
    Trattamento della spasticita dell'arto superiore nei bambini
    A.4.1Sponsor's protocol code number191622-101
    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.3.4Pharmaceutical 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
    Upper Limb Spasticity.
    Spasticita dell'arto superiore
    E.1.1.1Medical condition in easily understood language
    Upper Limb Spasticity.
    Spasticita dell'arto superiore
    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 10048970
    E.1.2Term Arm 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 (3 U/kg and 6 U/kg) of BOTOX with standardized occupational therapy (OT) in pediatric patients with upper limb spasticity.
    Valutare la sicurezza e l'efficacia di un singolo trattamento di 2
    dosi (3 U/kg e 6 U/kg) di BOTOX in associazione con terapia occupazionale (TO)
    standardizzata in pazienti pediatrici affetti da spasticità dell'arto superiore
    E.2.2Secondary objectives of the trial
    None
    Non applicabile
    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
    • Upper limb monoplegic, hemiplegic, or triplegic spasticity (spasticity confirmed by Hypertonia Assessment Tool [HAT]) with single-arm sparing (only 1 arm requiring botulinum toxin treatment for spasticity during the study) resulting from cerebral palsy, or post-stroke with the stroke onset prior to age 2 and at least 12 months prior to the day 1 visit
    • Patient’s spasticity meets at least one of the conditions below in the study limb at the screening and day 1 visits:
    o Elbow flexor tone of 2 or greater as measured by the MAS-B AND elbow flexor muscle contracture of no more than 30 degrees, and/or
    o Wrist flexor tone of 2 or greater with finger flexor tone of 1+ or greater as measured by the MAS-B
    AND at least neutral position for wrist with fingers at maximum extension
    • Gross Motor Function Classification System Expanded and Revised (GMFCS-E&R) level I to IV at the screening visit
    • Manual Ability Classification System (MACS) 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
    • Patients who are on an intrathecal baclofen pump must be on a continuous infusion at a stable dose and regimen (ie, not on bolus infusions) 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; Spasticità monoplegica, emiplegica o triplegica dell'artosuperiore (spasticità confermata con HAT [Hypertonia Assessment Tool]) confinata a un solo
    braccio (solo 1 braccio necessita del trattamento con tossina botulinica per la spasticità
    durante lo studio), dovuta a paralisi cerebrale o post-ictus con l'insorgenza dell'ictus
    precedente ai 2 anni di età e risalente ad almeno 12 mesi prima della visita del giorno 1; - La
    spasticità del paziente deve soddisfare almeno una delle condizioni indicate di seguito
    nell'arto oggetto di studio alle visite di screening e del giorno 1: Tono dei muscoli flessori del
    gomito pari o superiore a 2 misurato con il punteggio MAS-B E contrattura dei muscoli
    flessori del gomito non superiore a 30 gradi e/o Tono dei muscoli flessori del polso pari o
    superiore a 2 con tono dei muscoli flessori delle dita pari o superiore a 1, misurati con il
    punteggio MAS-B E posizione almeno neutra del polso con le dita alla massima estensione; -
    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; Livello da I a V
    secondo il sistema di classificazione MACS (Manual Ability Classification System) alla visita
    di screening; - I pazienti trattati con farmaci antispastici o miorilassanti (ad es. 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 stabili 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 stabili per
    almeno 30 giorni prima della visita del giorno 1; - I pazienti trattati con pompa di infusione
    intratecale di baclofen devono essere stati sottoposti a infusione continua a una dose e a un
    regime di somministrazione stabili (ovvero non infusioni in bolo) 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 principal muscle group (elbow or wrist) for the study limb
    • 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
    • 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
    • Patient has had constraint-induced movement therapy within 2 months prior to the day 1 visit
    • History of surgical intervention 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 lower limb(s), or planned casting or splinting with a dynamic splint (eg, Dynasplint or UltraFlex) for spasticity of the study limb or affected lower limb(s) 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 del gruppo muscolare principale (gomito
    o polso) nell'arto oggetto di studio; - Epilessia non controllata, definita 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 necessitino di una
    benzodiazepina di salvataggio (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; - 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; -
    Pazienti sottoposti a terapia del movimento vincolo-indotta nei 2 mesi precedenti alla visita
    del giorno 1; - Anamnesi di intervento chirurgico nell'arto oggetto di studio (fatta eccezione
    per allungamento del tendine più di 12 mesi prima della visita del giorno 1) o intervento
    chirurgico pianificato in qualsiasi arto durante lo studio; - Ingessatura nei 6 mesi precedenti
    alla visita del giorno 1 o impianto di splint dinamico (ad es. Dynasplint o UltraFlex) nei 3
    mesi precedenti alla visita del giorno 1 per la spasticità dell'arto oggetto di studio o dell'arto
    o degli arti inferiori interessati oppure previsione di ingessatura o impianto di splint
    dinamico (ad es. Dynasplint o UltraFlex) per la spasticità dell'arto oggetto di studio o
    dell'arto o degli arti inferiori interessati durante lo studio; - Soggetti di sesso femminile in
    gravidanza, che allattino al seno o intendano iniziare una gravidanza durante lo studio; -
    Soggetti di sesso femminile potenzialmente fertili (dopo il menarca) che non utilizzino metodi
    contraccettivi affidabili (vedere Sezione 4.5.1.1 per i metodi contraccettivi accettabili)
    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, weeks 4 and 6.
    Baseline, Settimane 4 e 6
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints include the average grade change from baseline in MAS-B for finger flexor muscle group at weeks 4 and 6, functional goals (active and passive) using GAS by Physician evaluated separately relative to baseline at weeks 8 and 12, and MTS including the difference between slow (R2) and fast (R1) range of motion (R2 - R1) as well as respective change from baseline on the MTS of the principal muscle group 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 la variazione media del punteggio MAS B relativo
    al gruppo di muscoli flessori delle dita dal Baseline alle Settimane 4 e 6, 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. Please refer to protocol for details.
    Baseline, Settimane 2, 4, 6, 8, 12. Si prega di fare anche riferimento al protocollo per
    maggiori dettagli.
    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 concerned3
    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
    Poland
    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 Yes
    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)
    Patients who successfully complete this study without major protocol deviations will be given the option of enrolling in the open-label extension Study 191622-105 if they meet eligibility criteria. Otherwise patients would return to the standard of care treatment.
    I pazienti che completeranno con successo lo studio senza gravi deviazioni al protocollo
    avranno l'opportunita di essere arruolati nello studio di estensione in aperto 191622-105, 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 trial2017-07-06
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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
    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.

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