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    The EU Clinical Trials Register currently displays   44043   clinical trials with a EudraCT protocol, of which   7319   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:2008-003360-19
    Sponsor's Protocol Code Number:P070123
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2008-08-21
    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 ConcernedFrance - ANSM
    A.2EudraCT number2008-003360-19
    A.3Full title of the trial
    Etude de l'effet de la pioglitazone dans l'ataxie de Friedreich : étude "preuve de concept"
    A.3.2Name or abbreviated title of the trial where available
    ACTFRIE
    A.4.1Sponsor's protocol code numberP070123
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    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 Actos
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameActos
    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 INNpioglitazone
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpioglitazone
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpioglitazone
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number45
    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
    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
    Ataxie de Friedreich
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level PT
    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
    L'objectif principal est d'évaluer l'efficacité de la Pioglitazone comparativement à un placebo sur l'atteinte neurologique des patients atteints de maladie de Friedreich. L'atteinte neurologique sera évaluée par l'échelle "International Cooperative Ataxia Rating Scale" (ICARS) mise au point sous l'égide de la Fédération Mondiale de Neurologie (Trouillas et al, 1997) et on considèrera que le traitement est efficace si sur 2 ans sur cette échelle l'augmentation est inférieure ou égale à 2 points, la stabilisation de l'état clinique étant déjà considérée comme une amélioration.
    E.2.2Secondary objectives of the trial
    -Evaluer la tolérance du traitement
    - Evaluer l'efficacité du traitement par Pioglitazone sur l'atteinte neurologique mesurée par : - " Friedreich's Ataxia Rating Scale " FARS validée plus récemment (Lynch et al, 2005 ; Subramony et al, 2005 ; Lynch et al, 2006). / - l'analyse de la marche / - l' évaluation de la consommation énergétique à la marche / - l'étude de la posture /- étude des mouvements des membres supérieurs /- l'évaluation de la force musculaire /- l'évaluation du handicap fonctionnel par l'échelle DSS (Disability Status Scale) et de la qualité de vie par le questionnaire MOS SF-36 (Medical Outcome Study Short Form 36 item health survey) / - l'étude de l'acoustique de la parole et du langage /- l'évaluation des troubles ophtalmologiques / - l'évaluation de l'audition
    - évaluation de l'efficacité de la pioglitazone sur le plan cardiologique
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Mise en place d'un protocole et d'outils pour quantifier les fonctions du membre supérieur chez des personnes atteintes par la maladie de Friedreich (40 patients+40 témoins)
    E.3Principal inclusion criteria
    -Ataxie de Friedreich avec confirmation par étude moléculaire. Patients porteurs à l'état homozygote de l'expansion GAA dans le gène de la frataxine (Il sera demandé au patient de fournir les résultats de l'analyse génétique de confirmation de l'ataxie de friedreich), avec des expansions supérieures à 300 répétitions ;
    -Age à l'inclusion compris entre 7 et 24 ans inclus
    -Patients capables de faire 2 pas (y compris avec l'aide d'un déambulateur) ou de tenir debout
    -Patients symptomatiques sur le plan neurologique
    -Absence d'insuffisance cardiaque clinique et fraction d'éjection à l'échographie cardiaque supérieure à 50%
    -Patients (ou titulaires de l'autorité parentale pour les mineurs) ayant donné leur consentement éclairé
    -Patientes d'accord pour utiliser une méthode contraceptive si elles sont sexuellement actives pendant la durée de l'essai
    -Patients n'ayant reçu aucune modification de leur traitement (idebenone) et du dosage donné dans les 6 mois précédents l'inclusion et d'accord pour n'avoir aucune modification de leur traitement tout au long de l'essai
    -Patient affilié à un régime de sécurité sociale ou CMU*
    CMU* : Couverture médicale universelle

    Groupe témoins pour l'étude ancillaire
    - Age à l'inclusion 7ans et 24 ans
    - Absence d'incapacité au niveau des membres supérieurs (centrale ou périphérique)
    - Absence de mouvements anormaux
    - Capacité intellectuelle, motrice et neuropsychologique normale
    - Réalisation d'un examen médical préalable
    - Consentement éclairé et signé
    E.4Principal exclusion criteria
    Groupe malade
    - Hétérozygotes composites
    - Sujets incapables de tenir debout même avec aide
    - Insuffisance cardiaque NYHA I à IV ou constatation à l'échographie cardiaque d'une fraction d'éjection inférieure à 50% ou diminution de la fraction d'éjection supérieure ou égale à 20%
    - Atteinte hépatique (ASAT, ALAT supérieures à 2, 5 fois la limite supérieure de la norme)
    - Patients diabétiques (glycémie supérieure à 1,26g/l à jeun ou supérieure à 2g à 120 minutes)
    - Modification du traitement habituel dans les 6 mois précédents l'étude et au cours de l'étude
    - Patiente enceinte ou allaitante

    Groupe témoins (pour l'étude ancillaire)
    - Non-affiliation à un régime de sécurité sociale ou CMU (bénéficiaire ou ayant droit)
    E.5 End points
    E.5.1Primary end point(s)
    Le critère de jugement principal choisi sera défini à partir de l'échelle ICARS " International Cooperative Ataxia Rating Scale " . L'efficacité de la pioglitazone sera définie par la prise au maximum de 2 points sur l'échelle ICARS au bout de 2 ans de traitement déterminant la stabilité / amélioration et définissant le succès. A contrario, l'échec sera défini par la prise de plus de 2 points sur l'échelle ICARS.

    Les critères de jugement secondaires sont (cf protocole p 30) : Echelle FARS / Etude de la marche et de la station debout / Evaluation du mouvement des membres supérieurs (cf étude ancillaire ) / Evaluation du handicap fonctionnel par l'échelle DSS et de la qualité de vie par le questionnaire MOS SF- 36 / Evaluation ophtalmologique / Analyse acoustique du contrôle moteur de la parole / Evaluation de l'audition / Evaluation de l'atteinte cardiaque / Evaluation de la tolérance à la Pioglitazone / Profil pharmacocinétique de la Pioglitazone
    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 Yes
    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) 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 Yes
    E.8.2.3Other No
    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 concerned6
    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 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
    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 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.1.1.1In 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.1.1.6Adolescents (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 Yes
    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 2008-08-21. 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state80
    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 Decision2008-09-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-11-17
    P. End of Trial
    P.End of Trial StatusOngoing
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