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    Summary
    EudraCT Number:2013-002154-67
    Sponsor's Protocol Code Number:A-MANECE-2013
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-09-16
    Trial 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 number2013-002154-67
    A.3Full title of the trial
    RANDOMIZED CLINICAL TRIAL, PLACEBO COMPARED TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
    ENSAYO CLINICO DOBLE CIEGO, RANDOMIZADO, COMPARADO CON PLACEBO PARA EVALUAR LA EFICACIA Y SEGURIDAD DE MINOCICLINA EN PACIENTES CON SINDROME DE ANGELMAN (ESTUDIO A-MANECE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    STUDY TO EVALUATE THE EFFICACY AND SAFETY OF MINOCYCLINE IN ANGELMAN SYNDROME (A-MANECE STUDY)
    ESTUDIO PARA EVALUAR LA EFICACIA Y SEGURIDAD DE LA MINOCICLINA EN PACIENTES CON SINDROME DE ANGELMAN (ESTUDIO A-MANECE)
    A.3.2Name or abbreviated title of the trial where available
    A-MANECE
    A-MANECE
    A.4.1Sponsor's protocol code numberA-MANECE-2013
    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 SponsorDra. Cristina Avendaño Solá
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPrivate donation
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNo
    B.5.2Functional name of contact pointDra. Belén Ruiz Antorán
    B.5.3 Address:
    B.5.3.1Street AddressJoaquín Rodrigo
    B.5.3.2Town/ cityMajadahonda
    B.5.3.3Post codeNo
    B.5.3.4CountrySpain
    B.5.4Telephone number34911917479
    B.5.5Fax number34911917650
    B.5.6E-mailmariabelen.ruiz@salud.madrid.org
    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 Aknemin 50
    D.2.1.1.2Name of the Marketing Authorisation holderAlmirall Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 Capsule
    D.3.4.1Specific paediatric formulation No
    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 INNMINOCYCLINE
    D.3.9.1CAS number 13614-98-7
    D.3.9.2Current sponsor codePL 33016/0005
    D.3.9.3Other descriptive nameMINOCYCLINE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB03304MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 placeboCapsule
    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
    Angelman Syndrome
    Síndrome de Angelman
    E.1.1.1Medical condition in easily understood language
    Angelman Syndrome
    Síndrome de Angelman
    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 16.0
    E.1.2Level PT
    E.1.2Classification code 10049004
    E.1.2Term Angelman's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To explore the efficacy of Mynocicline in the treatment of patients affected of Angelman Syndrome, in terms of increase in the age of development
    Explorar la eficacia de minociclina en el tratamiento de pacientes con Síndrome de Angelman, en términos de incremento en la edad de desarrollo equivalente obtenida a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), tras 8 y/o 16 semanas de tratamiento.
    E.2.2Secondary objectives of the trial
    1) To compare mynocicline efficay vs placebo in terms of:
    -specific improvement of cognitive development, language and comunication, motor development, social-emotional development and adaptative conduct.
    - improvement of adaptive behaviour, , the ability to adapt to changes in their environment, learn new skills everyday and the level of independence
    2) To evaluate the safety of mynociclne
    1) Comparar la eficacia de minociclina frente a placebo en términos de:
    - Mejoría especifica del desarrollo cognitivo, lenguaje y comunicación, desarrollo motor, desarrollo socio-emocional y conducta adaptativa obtenidos a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), en la 8, 16 y 24 semanas.
    - Mejora en los comportamientos de adaptación, la capacidad de adaptarse a los cambios en su entorno, aprender nuevas habilidades cotidianas y el nivel de independencia en la 8, 16 y 24 semanas obtenidos a través de la Escala de Comportamiento Vineland Adaptive, cuarta edición (Vin-II).
    - Mejora de los EEG. Medida en base a los cambios en la actividad de fondo, tipo, número y duración de las crisis, tendencia a la generalización de las crisis, tipos de anomalías paroxísticas registradas y la evaluación global del neurofisiologo clínico, en la 8, 16 y 24 semanas
    2) Evaluar la seguridad de minociclina
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    (1) Male or female between 6 and 30 years old.
    (2) Clinical diagnosis of Angelman Syndrome and molecular confirmation of diagnosis.
    (3) The participant has an acceptable guardian can give consent on behalf of the participant.
    (1) Hombre o mujer entre 6 y 30 años de edad.
    (2) Diagnostico clínico de Sindrome de Angelman y confirmación molecular del diagnóstico.
    (3) El participante tiene un tutor aceptable capaz de dar su consentimiento en nombre del participante,
    E.4Principal exclusion criteria
    (1) Patients with hypersensitivity to tetracyclines.
    (2) Patients with impaired hepatic or renal function and in those with mainly drug allergy history.
    (3) Any other condition that in the opinion of the investigator is considered clinically relevant and that administration of minocycline contraindicated ..
    (1) Pacientes con hipersensibilidad a las tetraciclinas.
    (2) Pacientes con las funciones hepática o renal alteradas y en aquellos con historial alérgico fundamentalmente medicamentoso.
    (3) Cualquier otra patología que a criterio del investigador se considere clínicamente relevante y que contraindique la administración de minociclina..
    E.5 End points
    E.5.1Primary end point(s)
    Increased on the equivalent age of development, obtained through Development Scale R Merrill-Palmer (MP-R)
    Incremento en la edad de desarrollo equivalente, obtenida a través de la Escala de Desarrollo Merrill-Palmer R (MP-R)
    E.5.1.1Timepoint(s) of evaluation of this end point
    8, 16 and 24 weeks
    SEMANA 8, 16 y 24
    E.5.2Secondary end point(s)
    - Improved specific cognitive, language and communication, motor development, social-emotional and adaptive behavior obtained through Development Scale R Merrill-Palmer (MP-R), at 8, 16 and 24 weeks.
    - Improved adaptive behaviors, the ability to adapt to changes in their environment, learn new life skills and level of independence in the 8, 16 and 24 weeks obtained through the Vineland Adaptive Behavior Scale, Fourth Edition ( Vin-II).
    - Improvement of EEG. Measure based on changes in the background activity, type, number and duration of crises, widespread tendency to crises, paroxysmal abnormalities recorded types and the overall evaluation of clinical neurophysiologist, at 8, 16 and 24 weeks
    - Safety and tolerability.
    a) Physical Examination
    b) Vital signs
    c) Laboratory Tests
    d) Adverse effects (AEs) list for treatment, laboratory values, values ??outside the reference range and descriptive statistics.
    Variables secundarias:
    - Mejoría especifica del desarrollo cognitivo, lenguaje y comunicación, desarrollo motor, desarrollo socio-emocional y conducta adaptativa obtenidos a través de la Escala de Desarrollo Merrill-Palmer R (MP-R), en la 8, 16 y 24 semanas.
    - Mejora en los comportamientos de adaptación, la capacidad de adaptarse a los cambios en su entorno, aprender nuevas habilidades cotidianas y el nivel de independencia en la 8, 16 y 24 semanas obtenidos a través de la Escala de Comportamiento Vineland Adaptive, cuarta edición (Vin-II).
    - Mejora de los EEG. Medida en base a los cambios en la actividad de fondo, tipo, número y duración de las crisis, tendencia a la generalización de las crisis, tipos de anomalías paroxísticas registradas y la evaluación global del neurofisiologo clínico, en la 8, 16 y 24 semanas
    - Seguridad y tolerancia.
    a) Examen físico
    b) Signos vitales
    c) Pruebas de laboratorio
    d) Efectos adversos (EA): listado por tratamiento, valores de laboratorio, valores fuera del rango de referencia y estadísticas descriptivas.
    E.5.2.1Timepoint(s) of evaluation of this end point
    8, 16 and 24 weeks
    SEMANA 8, 16 y 24
    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) 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 No
    E.8.1.6Cross over Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Patients may stop participating in the trial at the time they wish.
    Also, in his judgment and discretion, the researcher may decide to withdraw the patient from the trial, does not meet the rules of the protocol.
    Los pacientes podrán suspender su participación en el ensayo en el momento en que lo deseen.
    Asimismo, a su juicio y criterio, el investigador podrá decidir la retirada del paciente del ensayo, si no cumple las normas del protocolo.
    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 months0
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 32
    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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    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 No
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Angelman Syndrome patients had a severe mental retardation is not expected to provide your child assent.
    Dadas las características de estos pacientes (retraso mental severo) no esta previsto que el niño proporcione su asentimiento,
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state32
    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 Decision2013-12-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-22
    P. End of Trial
    P.End of Trial StatusOngoing
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