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    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   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).

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    Summary
    EudraCT Number:2010-024084-40
    Sponsor's Protocol Code Number:XAMNANTIOXAP2010
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2011-01-07
    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 number2010-024084-40
    A.3Full title of the trial
    Ensayo clínico en adrenomieloneuropatía (AMN): validación de biomarcadores de estrés oxidativo, eficacia y tolerancia de la combinación de antioxidantes N-acetilcisteína, ácido lipoico y vitamina E
    A.4.1Sponsor's protocol code numberXAMNANTIOXAP2010
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAurora Pujol Onofre
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Information not present in EudraCT
    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 nameN-acetilcisteína (NAC)
    D.3.4Pharmaceutical form Powder for oral solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNAcetilcisteína
    D.3.9.1CAS number 616-91-1
    D.3.9.3Other descriptive nameN-acetilcisteína, N-acetil-L-cisteína
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number600
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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.IMP: 2
    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 Information not present in EudraCT
    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 namevitamina E
    D.3.4Pharmaceutical form Capsule, soft
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNacetato de dl-alfa-tocoferol
    D.3.9.3Other descriptive namealfa tocoferol, vitamina E
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number150
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 typevitamina
    D.IMP: 3
    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 nameacido lipoico
    D.3.4Pharmaceutical form Capsule*
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNacido tióctico
    D.3.9.2Current sponsor codeacido lipoico
    D.3.9.3Other descriptive nameacido alfa lipoico
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number300
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Adrenoleucodistrofia ligada al X (XALD), fenotipo Adrenomieloneuropatía (AMN).
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 13
    E.1.2Level LLT
    E.1.2Classification code 10051260
    E.1.2Term Adrenoleucodistrofia
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    El objetivo principal del ensayo clínico es la validación e identificación de marcadores y evaluación de los efectos biológicos en estos marcadores así como la tolerancia y seguridad de una combinación de antioxidantes (NAC, ácido lipoico y vitamina E), en pacientes de adrenomieloneuropatía (AMN). Se trata del estudio de una combinación de fármacos en una nueva indicación terapéutica.
    E.2.2Secondary objectives of the trial
    I) Evaluar la seguridad y tolerancia de la combinación de fármacos en un tratamiento crónico en este tipo de pacientes.
    II) Mejorar la atención clínica y el consejo genético de los pacientes, especialmente en los aspectos reproductivos en los que hemos detectado frecuentemente falta de información en las portadoras.
    III) Recopilar información sobre epidemiología, historia natural de la enfermedad, generar nuevos datos clínicos y familiares de la población afecta en forma de base de datos. Esto facilitará la inclusión de esta población española en estudios internacionales que pretenden una mejor comprensión de la historia natural de la enfermedad, y su acceso a los últimos ensayos terapéuticos experimentales. Este objetivo es factible ya que Aurora Pujol participa en el proyecto europeo Leukotreat como co-coordinador.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Pacientes sintomáticos, mayores de 18 años, afectos de AMN, ambos sexos, diagnosticados clínica y bioquímicamente, tengan o no su mutación identificada en el caso de los varones; las mujeres deben tener su mutación identificada o ser portadoras obligadas.
    E.4Principal exclusion criteria
    Enfermedad inflamatoria cerebral avanzada con trastorno cognitivo, y/o necesitar la ayuda de 2 bastones para caminar. Crisis epilépticas, hipersensibilidad a compuestos relacionados con cisteína, úlcera gastro-duodenal, asmáticos, insuficiencia respiratoria grave. Alteración de la función hepática (transaminasas >2 veces del normal), u otra alteración en la analítica básica. Mujeres en edad fértil, embarazadas o lactantes. Presentar alteraciones en los controles periódicos de niveles de transaminasas, bioquímica general y hematocrito que se realizarán paralelos a los controles que realizaremos o cualquier otro efecto secundario.
    E.5 End points
    E.5.1Primary end point(s)
    VP1=Niveles de lesión oxidativa en proteínas y peroxidación lipídica. Se cuantificarán en plasma, MNC y LCR los siguientes marcadores de lesión oxidativa en proteínas: GSA y AASA, CML y CEL más MDAL. Son cinco marcadores de lesión que pueden variar independientemente. Los análisis se realizarán por cromatografía de gases/espectrometría de masas. La cuantificación se realizará por estandarización externa, usando curvas patrón construidas con los marcadores deuterados y no deuterados de síntesis propia. Las cantidades de productos se expresarán como micromoles de GSA, AASA, CML, CEL y MDAL por mol de lisina.
    *Estos análisis se realizarán en el Departamento de Medicina Experimental, Facultad de Medicina, Universidad de Lleida.
    VP2=Niveles de marcadores de oxidación en lípidos: Cuantificaremos los niveles de 10-hidroxi-docosahexanoico, 17-hidroxi-docosahexanoico, 8-isoprostaglandina F2&#945; (en orina), ácido hidroxioctadecadienoico, isomeros 13 y 9 ácido 15 hidroxieicosatetraenoico, en ionización negativa y 1-palmitoil-2-araquidonoil-3-glicerofosfocolina, 1-palmitoil-2-linoleoil-3-glicerofosfocolina, 4 hidroxinonenal, nitrooleato y hidroxioctadienoato de colesterol en ionización positiva. Se realiza su medición por estandarización externa en un sistema LC-QTOF, en las mismas muestras.
    *Estos análisis se realizarán en el Departamento de Medicina Experimental, Facultad de Medicina, Universidad de Lleida.
    VP3=Niveles de lesión oxidativa en DNA. Cuantificaremos los niveles de 8-oxodG en plasma, orina y células mononucleares, mediante HPLC acoplada a un detector electroquímico (20, 21, 48) que compraremos si se nos concede el proyecto presentado en la última convocatoria del MSPSI para ensayos clínicos de iniciativa independiente. Los resultados se expresarán como cociente 8-oxodG/creatinine. La concentración de creatinina se medirá con el kit 555-A de Sigma Diagnostics, según el método de Slot (49, 50). También cuantificaremos las deleciones en DNA por PCR cuantitativa, técnica ya establecida en nuestro laboratorio y extremadamente rápida (resultados en 24 h).
    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 Yes
    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 Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Se considera final del ensayo el momento de la ultima vista de seguimiento del ultimo paciente incluido en el ensayo
    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 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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 Decision2011-06-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-03-10
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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