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    Summary
    EudraCT Number:2010-021817-22
    Sponsor's Protocol Code Number:Y-52-52120-153
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-02-07
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2010-021817-22
    A.3Full title of the trial
    A PHASE III, MULTICENTRE, DOUBLE BLIND, PROSPECTIVE, RANDOMISED, CONTROLLED, MULTIPLE TREATMENT STUDY ASSESSING EFFICACY AND SAFETY OF DYSPORT USED IN THE TREATMENT OF UPPER LIMB SPASTICITY IN CHILDREN
    ESTUDIO DE FASE III, MULTICENTRICO, DOBLE CIEGO, PROSPECTIVO, ALEATORIZADO, CONTROLADO, DE TRATAMIENTO MULTIPLE PARA EVALUAR LA EFICACIA Y LA SEGURIDAD DE DYSPORT EN EL TRATAMIENTO PEDIATRICO DE LA ESPASTICIDAD DE LAS EXTREMIDADES SUPERIORES
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3, blinded, multicentre study assessing efficacy and safety of Dysport for treatment of upper limb spasticity (altered skeletal muscle performance) in children
    Estudio de fase 3, multicentrico, cegado para evaluar la eficacia y seguridad de Dysport para el tratamiento de la espasticidad en miembros superiores (alteracion en el funcionamiento de los musculos esqueleticos) en niños.
    A.4.1Sponsor's protocol code numberY-52-52120-153
    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 SponsorIPSEN INNOVATION
    B.1.3.4CountryFrance
    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 supportIPSEN INNOVATION
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIPSEN PHARMA, S.A.
    B.5.2Functional name of contact pointDepart. Médico/Elena Hernández
    B.5.3 Address:
    B.5.3.1Street AddressTorre Realia-Plaza Europa 41-43 Planta 7
    B.5.3.2Town/ cityL´Hospitalet de Llobregat-Barcelona
    B.5.3.3Post code08908
    B.5.3.4CountrySpain
    B.5.4Telephone number34936-858-177
    B.5.5Fax number34936-851-011
    B.5.6E-mailelena.hernandez@ipsen.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 Dysport
    D.2.1.1.2Name of the Marketing Authorisation holderIpsen Biopharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 INNCLOSTRIDIUM BOTULINUM TOXIN TYPE A
    D.3.9.1CAS number 93384-43-1
    D.3.9.3Other descriptive nameBOTULINUM TOXIN TYPE A
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 No
    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 Yes
    D.3.11.13.1Other medicinal product typeToxina
    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
    Upper limb spasticity in children
    Espasticidad de la extremidad superior en niños
    E.1.1.1Medical condition in easily understood language
    Upper limb spasticity (altered skeletal muscle performance) in children
    Espasticidad del miembro superior (alteracion del funcionamiento del musculo esqueletico) en niños
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10048970
    E.1.2Term Arm spasticity
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary study objective is to assess the efficacy of two doses of Dysport (8 U/kg and 16 U/kg) compared to Dysport 2 U/kg used in the treatment of upper limb spasticity in children with CP following a single treatment.
    El objetivo principal del estudio es evaluar la eficacia de dos dosis de Dysport (8 unidades U/kg y 16 U/kg), en comparacion con Dysport 2 U/kg, en el tratamiento de la espasticidad de las extremidades superiores en niños con paralisis cerebral (PC) despues de un unico tratamiento.
    E.2.2Secondary objectives of the trial
    The secondary study objective is to assess the long term safety of multiple treatments of Dysport used in this study population.

    The tertiary study objectives will evaluate the long term efficacy of Dysport, its effect on pain in the study limb and quality of life (QoL) following multiple treatments.
    El objetivo secundario del estudio es evaluar la seguridad a largo plazo de multiples tratamientos con Dysport en la poblacion del estudio.

    Los objetivos terciarios del estudio evaluaran la eficacia a largo plazo de Dysport, su efecto sobre el dolor de la extremidad evaluada en el estudio y la calidad de vida (CdV) despues de multiples tratamientos.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    (1) Signed informed consent obtained from the child?s parent(s)/guardian(s) and, if applicable, a signed assent from the child.
    (2) Be from 2 to 17 years of age, inclusive.
    (3) Body weight of 10 kg or more at the baseline visit.
    (4) Have a diagnosis of CP, as defined by Rosenbaum.
    (5) Have increased muscle tone/spasticity in at least one upper limb.
    (6) Have a MAS score ?2 in the upper limb primary targeted muscle group (elbow flexors or wrist flexors) of the study limb (the limb to be injected in the first treatment) at the baseline visit.
    (7) Be classified as Gross Motor Function Classification System Level 1 to 4.
    (8) If undergoing therapy, such as physiotherapy, occupational therapy, or use of splints and/or orthoses, therapy must have been initiated at least 30 days prior to the baseline visit and agreed to continue the therapy throughout the study and at the minimum, up to Week 16 following the first treatment administration.
    (1) Consentimiento informado firmado de los padres/tutores del menor y, si procede, el asentimiento firmado del propio menor.
    (2) Edad de 2 a 17 años, ambos inclusive.
    (3) Peso corporal de 10 kg o más en la visita basal.
    (4) Diagnostico de PC, segun los criterios de Rosenbaum.
    (5) Hipertonia muscular/espasticidad como minimo en una extremidad superior.
    (6) Puntuacion MAS ?2 en la visita basal para el grupo muscular principal de referencia de la extremidad superior (flexores del codo o flexores de la muñeca) evaluada en el estudio (la extremidad que recibira la inyección en el primer tratamiento).
    (7) Nivel 1 a 4 en la escala GMFCS.
    (8) Si el sujeto recibe algun tipo de terapia, como fisioterapia, terapia ocupacional o uso de ferulas u ortesis, el mismo debera haberse instaurado como minimo 30 dias antes de la visita basal y tendra que acordarse la continuacion de dicha terapia durante todo el estudio y al menos hasta la semana 16 despues de la primera administracion del tratamiento.
    E.4Principal exclusion criteria
    (1) Fixed myocontracture in the primary targeted muscle group (elbow flexors or wrist flexors) of the study limb defined as having an available range of motion angle of <40°, regardless of the starting and finishing angles, measured at the Tardieu Scale (TS) slow (XV1) speed at the baseline visit. (Available range for wrist flexors should be measured without holding the fingers and allowing free finger flexion).
    (2) Subjects likely to be treated with BTX in the lower limb(s) and/or the nonstudy upper limb before Treatment 2 of the study.
    (3) Inadequate washout from previous BTX injection of any serotype for any condition:
    ? Within 6 months prior to the baseline visit in the study limb,
    or
    ? Within 3 months prior to the baseline visit in any other part of the body.
    (4) Subjects who require BTX treatment in a single muscle group in the study limb in Treatment 1.
    (5) Severe athetoid or dystonic movements in the study limb.
    (6) Previous or planned surgery for spasticity in the primary targeted muscle group(s) of the study limb.
    (7) Previous injection of alcohol and/or phenol within 1 year prior to the baseline visit affecting the study limb wrist flexors and/or elbow flexors selected for injection in Treatment 1.
    (8) Treatment with any drug that interferes either directly or indirectly with neuromuscular function (e.g. aminoglycoside antibiotics) or neuroblocking agents used during surgery (e.g. curare) within the last 30 days prior to study treatment.
    (9) Are pregnant and/or lactating.
    (10) Female subjects not willing to use contraceptive measures throughout the course of the study if postpubertal and sexually active.
    (11) Inability or unwillingness to comply with the protocol.
    (12) Subjects with any clinical (or sub-clinical) evidence of marked defective neuromuscular transmission (e.g. Lambert-Eaton syndrome or myasthenia gravis) or persistent clinically significant neuromuscular disorders.
    (13) Known sensitivity to BTX or to any of the components in the formulation or allergy to cow?s milk protein.
    (14) An infection at the injection site(s).
    (15) Previous rhizotomy less than 6 months prior to the baseline visit or rhizotomy planned/anticipated during the course of the study.
    (16) Subjects treated or likely to be treated with intrathecal baclofen within 30 days prior to the baseline visit or during the course of the study.
    (17) Treatment with a new investigational drug within 30 days prior to the baseline visit or scheduled to receive such a drug during the course of the study.
    (18) Subjects with a history of aspiration or conditions which put them at risk of aspiration, such as severe dysphagia.
    (19) Concurrent or history of frequent lower respiratory tract infections, aspiration pneumonia, or, as judged by the Investigator, has compromised respiratory function.
    (20) Any known medical condition, laboratory or diagnostic procedure finding, which might compromise compliance with the objectives and procedures of this protocol or preclude administration of botulinum toxin type A (BTX-A), as judged by the Investigator.
    (21) Any uncontrolled clinically significant medical condition other than CP.
    (1) Contractura muscular fija en el grupo muscular principal de referencia (flexores del codo o flexores de la muñeca) de la extremidad evaluada en el estudio, definida como un angulo de amplitud de movimiento <40°, independientemente de los angulos inicial y final, determinado a velocidad lenta (XV1) segun la escala Tardieu en la visita basal. (El intervalo de los flexores de la muñeca debera medirse sin sujetar los dedos y permitiendo la flexion libre de los mismos).
    (2) Sujetos que precisen tratamiento con BTX en las extremidades inferiores y/o en la extremidad superior no evaluada en el estudio antes del Tratamiento 2.
    (3) Periodo de lavado insuficiente de una inyeccion previa de BTX de cualquier serotipo utilizada para cualquier trastorno:
    ?En los 6 meses previos a la visita basal en la extremidad evaluada en el estudio
    o
    ?En los 3 meses previos a la visita basal en cualquier otra parte del cuerpo.
    (4) Sujetos que precisen en el Tratamiento 1 BTX en un solo grupo muscular de la extremidad evaluada en el estudio.
    (5) Movimientos atetoides o distonicos intensos en la extremidad evaluada en el estudio.
    (6) Intervencion quirurgica previa o programada por espasticidad en el grupo muscular principal de referencia de la extremidad evaluada en el estudio.
    (7) Inyección de alcohol y/o fenol en el año previo a la visita basal en los musculos flexores de la muñeca y/o del codo de la extremidad evaluada en el estudio elegidos para ser inyectados en el Tratamiento 1.
    (8) Tratamiento con cualquier farmaco que afecte directa o indirectamente a la funcion neuromuscular (p. ej., antibioticos aminoglucosidos) o uso de neurobloqueantes durante una intervención quirurgica (p. ej., curare) en los 30 dias previos al tratamiento del estudio.
    (9) Embarazo y/o lactancia.
    (10) Pacientes de sexo femenino que no deseen utilizar medidas anticonceptivas durante el transcurso del estudio si son pospuberales y sexualmente activas.
    (11) Incapacidad o poca disposicion para cumplir el protocolo.
    (12) Sujetos con evidencia clinica (o subclinica) de transmision neuromuscular defectuosa marcada (p. ej., sindrome de Lambert-Eaton o miastenia grave) o con trastornos neuromusculares persistentes clinicamente significativos.
    (13) Sensibilidad conocida a BTX o a alguno de los componentes de la formulacion o alergia a la proteina de la leche de vaca.
    (14) Infeccion en el punto de inyeccion.
    (15) Rizotomia en los 6 meses previos a la visita basal o programada/anticipada durante el transcurso del estudio.
    (16) Sujetos tratados con baclofeno intratecal en los 30 dias previos a la visita basal o con probabilidad de ser tratados durante el transcurso del estudio.
    (17) Tratamiento con un nuevo farmaco en investigacion en los 30 días previos a la visita basal o prevision de tal tratamiento durante el transcurso del estudio.
    (18) Sujetos con antecedentes de aspiracion u otro trastorno que suponga un riesgo de aspiracion, como disfagia.
    (19) Presencia o antecedente de infecciones frecuentes de las vias respiratorias inferiores, neumonia por aspiracion o que, a juicio del investigador, pueda afectar la funcion respiratoria.
    (20) Cualquier enfermedad conocida o resultados analiticos o de un procedimiento diagnostico que, a juicio del investigador, puedan poner en peligro el cumplimiento de los objetivos y los procedimientos de este protocolo o impedir la administracion de BTX-A.
    (21) Cualquier condicion medica no controlada clinicamente significativa, a excepcion de la PC.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    Mean change from Baseline to Treatment 1, Week 6 in MAS score in the Treatment 1 primary targeted muscle group (elbow flexors or wrist flexors).
    Objetivo principal de eficacia:
    Cambio medio de la puntuacion MAS en el grupo muscular principal de referencia para el Tratamiento 1 (flexores del codo o de la muñeca) entre la visita basal y la semana 6 del Tratamiento 1.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 6
    semana 6
    E.5.2Secondary end point(s)
    Mean PGA score at Treatment 1, Week 6.
    Mean GAS score at Treatment 1, Week 6.
    Puntuacion PGA media en la semana 6 del Tratamiento 1.
    Puntuacion GAS media en la semana 6 del Tratamiento 1.
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 6
    semana 6
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    dos dosis de Dysport (8 U/Kg y 16 U/Kg) frente a Dysport 2 U/Kg
    two doses of Dysport (8 units (U)/kg and 16 U/kg) compared to Dysport 2 U/kg
    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 EEA12
    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
    Bulgaria
    Chile
    Colombia
    Czech Republic
    France
    Georgia
    Israel
    Lithuania
    Mexico
    Poland
    Portugal
    Russian Federation
    South Africa
    Spain
    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
    Last visit last subject
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months3
    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: 210
    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: 105
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 105
    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
    A signed assent is obtained from the child when and where applicable (children from 2-year old can be included). Signed informed consent is always obtained from the child?s parent(s)/guardian(s).
    Se obtendra un asentimiento firmado de los niños cuando aplique (se pueden incluir niños desde 2 años). Siempre se obtendra el consentimiento informado firmado de los padres/tutores legales.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 210
    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 will return to standart medical care.
    Los pacientes retomarán su tratamiento médico habitual
    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 Decision2014-04-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-03-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-09-04
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