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    Summary
    EudraCT Number:2007-004001-96
    Sponsor's Protocol Code Number:ATFD07
    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:2010-03-08
    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 number2007-004001-96
    A.3Full title of the trial
    Efectos del tratamiento con deferiprone sobre la hipertrofia miocárdica y los parámetros de función cardiaca en pacientes afectos de Ataxia de Friedreich
    A.4.1Sponsor's protocol code numberATFD07
    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 SponsorHospital Sant Joan de Déu
    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 Yes
    D.2.1.1.1Trade name Ferriprox
    D.2.1.1.2Name of the Marketing Authorisation holderApotex Europe Ltd
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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 Cachet
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    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
    La Ataxia de Friedreich es una enfermedad causada por un déficit de la proteína frataxina. Su déficit produce acúmulo de hierro libre intramitocondrial, un incremento de la producción de radicales libres y al daño celular por estrés oxidativo. Evaluaremos la evolución cardiaca y neurológica con los datos del seguimiento de los cinco años pasados en tratamiento exclusivo con idebenona y a partir del nuevo tratamiento con deferiprone asociado a idebenona.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10017374
    E.1.2Term Friedreich's ataxia
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Monitorizar prospectivamente el efecto del tratamiento con idebenona conjuntamente con deferiprone sobre el corazón de los paciente con AF mediante el seguimiento del grado de hipertrofia miocárdica y los parámetros de función cardiaca.
    E.2.2Secondary objectives of the trial
    1.Comparar el efecto del tratamiento con idebenona frente al tratamiento conjunto de deferiprone e idebenona en los pacientes afectos de AF sobre la función cardiaca y la hipertrofia miocárdica.

    2. Medir prospectivamente el efecto del tratamiento con Idebenona conjuntamente con deferiprone sobre la sintomatología neurológica de los pacientes afectos de AF mediante el seguimiento con escalas que cuantifican el grado de afectación clínica de estos pacientes.

    3. Monitorizar el efecto del deferiprone sobre los depósitos de hierro cerebrales mediante secuencias T2 de RM cerebral.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Diagnostico genético confirmado de ataxia de Friedreich mediante la demostración de expansiones repetitivas GAA en el gen de la frataxina.
    - Pacientes en tratamiento previo con idebenona.
    - Las participantes de sexo femenino deberán presentar un test de gestación negativo y deberán confirmar el uso un método anticonceptivo efectivo previo al inicio del estudio.
    - Firma del consentimiento informado del paciente ante testigo antes del inicio del estudio.
    E.4Principal exclusion criteria
    - Alergia conocida al Ferriprox® o a alguno de sus componentes.
    - Déficit de hierro definido por niveles de ferritina inferiores a los niveles normales para edad y sexo.
    - Elevación anomála de ALT superior a 2 veces el límite superior de la normalidad en 2 controles sucesivos.
    - Creatinina sérica por encima de los niveles normales de referencia.
    - Historia o evidencia de neutropenia definida por un recuento absoluto de neutrófilos inferior a 1500/mm3 o trombocitopenia definida como un recuento de plaquetas inferior a 150.000/mm3.
    - Recibir o estar recibiendo alguna droga en proceso de investigación durante la realización del estudio o en los 30 días previos al inicio de éste.
    - Embarazo o administración de lactancia materna durante la realización del estudio.
    - Antecedentes de enfermedad cancerosa.
    - Abuso o antecedentes de abuso de drogas.
    E.5 End points
    E.5.1Primary end point(s)
    Inclusión en alguno de los criterios de exclusión durante la realización del ensayo
    Aparición de agranulocitosis o efectos secundarios intratables durante le ensayo
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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
    se comparará la situación basal de los paciente sin el tratamineto y tras su administración
    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 establece una duración de 12 meses a partir del inicio del tratamiento con deferiprone y tras haber relalizado los controles clínico-analíticos y exploraciones complementarias especificadas en en protocolo.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months12
    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.1.1.1In 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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    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)
    Los controles seguirán siendo los mismo que reciben habitualmente:
    Seguimiento ecocardiográfico cada 12 meses
    Seguimineto neurológico (escala ICARS): cada 6 meses
    Analítica sanguínea con pérfil bioquímico: cada 6 meses
    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 Decision2007-12-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-07-31
    P. End of Trial
    P.End of Trial StatusOngoing
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