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    Summary
    EudraCT Number:2013-003259-39
    Sponsor's Protocol Code Number:NeuroClin02
    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 number2013-003259-39
    A.3Full title of the trial
    A Phase II dose ranging study of Bumetanide solution in children and adolescents with autism spectrum disorders.
    Estudio de Fase II para determinar la dosis de solución oral de bumetanida en niños y adolescentes con Trastorno del Espectro del Autismoc
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study in children and adolescents with autism.
    Estudio en los niños y adolescentes con autismo.
    A.4.1Sponsor's protocol code numberNeuroClin02
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/110/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNeurochlore
    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 supportNeurochlore
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNeurochlore
    B.5.2Functional name of contact pointClinical Trial Department
    B.5.3 Address:
    B.5.3.1Street AddressINMED, Parc Scientifique de Luminy
    B.5.3.2Town/ cityMARSEILLE Cedex 09
    B.5.3.3Post codeMARSEILLE Cedex
    B.5.3.4CountryFrance
    B.5.4Telephone number+33(0)680 11 74 94
    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 No
    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 nameBumetanide
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameBUMETANIDE
    D.3.9.4EV Substance CodeSUB05971MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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 placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Diseases and Related Health Problems, tenth revision (ICD-10) and with a Childhood Autism Rating Scale (CARS) score > 34.
    Niños y adolescentes (varones y mujeres) de ≥2 a ≤18 años de edad que pesan ≥11 kg, correspondiente a F84.0 (Autismo Infantil) o F84.5 (Síndrome de Asperger) de acuerdo a la Clasificación Estadística Internacional sobre Enfermedades y Problemas de Salud Relacionados, décima revisión (International Statistical Classification of Diseases and Related Health Problems, Tenth revision - ICD-10) y con una puntuación CARS > 34.
    E.1.1.1Medical condition in easily understood language
    Autism and/or Asperger's Syndrome
    Autismo Infantil/Síndrome de Asperger
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Behaviours [F01]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the optimal dose strength for the pivotal Phase III study
    Niños y adolescentes con TEA confirmada según orientaciones internacionales.
    E.2.2Secondary objectives of the trial
    To evaluate the pharmacokinetics (PK) of Bumetanide in children and adolescents with ASD;
    To confirm the efficacy of Bumetanide in the treatment of children and adolescents with autistic spectrum Disorder (ASD).
    Evaluar la farmacocinética (PK) de bumetanida en niños y adolescentes con trastorno del espectro del autismo (TEA);
    Confirmar la eficacia de bumetanida en el tratamiento de niños y adolescentes con TEA.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female patients aged 2 to 18 years, inclusive;
    2.F84.0 (Childhood Autism) or F84.5 (Asperger's Syndrome) scores according to the International Statistical Classification of Diseases and Related Health Problems, 10th revision (ICD-10);
    3.Patients meeting criteria for autism on Autism Diagnosis Interview-Revised (ADI-R).
    4.CARS score > 34 points;
    5.Weight ≥11 kg;
    6. The patient is able to comply with the protocol for the duration of the study, including treatment, blood sampling and scheduled follow-up visits and examinations;
    7.The patient's parent/guardian has given written informed consent, prior to any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to their future medical care.
    1. Varones o mujeres de ≥2 años a ≤18 años;
    2. Puntuaciones de F84.0 (Autismo infantil) o F84.5 (Síndrome de Asperger), según la ICD 10;
    3. Cumplir con los criterios de autismo de la Escala de Observación Diagnóstica del Autismo - Genérica (ADOS G) y ADI-R (si está disponible la ADI R retrospectiva para niños >5 años de edad, no es necesario repetir la evaluación ADI R);
    4. Puntuación CARS >34 puntos;
    5. Peso ≥11 kg;
    6. Capacidad para cumplir con los procedimientos especificados en el protocolo durante todo el estudio, incluyendo tratamiento, toma de muestras, y visitas y exámenes de seguimiento planificados;
    7. Consentimiento informado por escrito (y aceptado, si corresponde según las leyes y regulaciones locales), antes de cualquier procedimiento relacionado con el estudio y que no sea parte de los cuidados médicos normales, en el entendimiento de que el consentimiento puede ser retirado por el paciente en cualquier momento, sin perjudicar su atención médica futura.
    E.4Principal exclusion criteria
    1.Serious, unstable illnesses including, gastroenterologic, respiratory, cardiovascular (QT interval lengthening), endocrinologic, immunologic, or hematologic disease;
    2.Renal or hepatic dysfunction that would interfere with excretion or metabolism of Bumetanide;
    3.Patients with any specific neurological disorders like seizures, microcephaly;
    4.Patients taking psychoactive medications except melatonin;
    5.Documented history of hypersensitivity reaction to sulfonamide derivatives.
    1. Enfermedad grave e inestable, incluyendo enfermedades gastroenterológicas, respiratoratorias, cardiovasculares (alargamiento del intervalo QT), endocrinológicas, inmunológicas o hematológicas;
    2. Disfunción renal o hepática que pueda interferir con la excreción o el metabolismo de bumetanida;
    3. Trastornos neurológicos tales como convulsiones y microcefalia;
    4. Tratamiento con medicamentos psicoactivos, excepto melatonina;
    5. Historia documentada de reacción de hipersensibilidad a los derivados de la sulfamida.
    E.5 End points
    E.5.1Primary end point(s)
    Change in CARS from baseline (Day 1) to Day 90.
    The treatment groups will be compared using a non-parametric test as specified in the Statistical Analysis Plan.
    Cambio en la CARS desde el valor inicial (Día 1) al Día 90.
    Los grupos de tratamiento serán comparados utilizando una prueba no paramétrica, tal como se especifica en el SAP.
    E.5.1.1Timepoint(s) of evaluation of this end point
    N/A
    n/a
    E.5.2Secondary end point(s)
    By way of further confirmatory measures of efficacy, the following endpoints will be analyzed:
    - CGI-I scale;
    - The Vineland Adaptive Behavior Scales (Vineland-II) (personal and social skills);
    - The Social Responsiveness Scale (SRS) (focuses on the child?s reciprocal social interactions);
    - QOL
    Utilizando mediciones de confirmación adicionales de eficacia, se analizarán los siguientes objetivos finales:
    • CGI-I;
    • Escalas de Comportamiento Adaptativo de Vineland (Vineland-II);
    • SRS (enfocada en las interacciones sociales recíprocas del niño);
    • QoL.
    E.5.2.1Timepoint(s) of evaluation of this end point
    n/a
    n/a
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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 Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 80
    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: 40
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2014-02-07. Yes
    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
    If a parent/guardian is willing for the patient to participate in the study, they will be requested to give written informed consent. The informed consent will be signed and personally dated by both a parent/guardian and the Investigator.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 80
    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)
    None
    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 Decision2014-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-12-26
    P. End of Trial
    P.End of Trial StatusCompleted
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