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    The EU Clinical Trials Register currently displays   44256   clinical trials with a EudraCT protocol, of which   7344   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:2005-005938-12
    Sponsor's Protocol Code Number:29-09-1963
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2006-04-05
    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 ConcernedAustria - BASG
    A.2EudraCT number2005-005938-12
    A.3Full title of the trial
    Eine offene, nicht randomisierte, einarmige Pilotstudie zur Beurteilung der Wirksamkeit von Erythropoetin bei Friedreich Ataxie.
    A.4.1Sponsor's protocol code number29-09-1963
    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 SponsorDr. Sylvia Boesch
    B.1.3.4CountryAustria
    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.2.1.1.1Trade name Neo-Recormon
    D.2.1.1.2Name of the Marketing Authorisation holderRoche
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    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 nameNeo-Recormon
    D.3.2Product code EU1/97/031/031-032
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    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
    Die Friedreich Ataxie (FRDA) ist eine autosomal-rezessiv vererbte, progressive neurodegenerative Erkrankung, die mit einer Inzidenz von rund 1:50.000 auftritt. Es handelt sich dabei um die am häufigsten vorkommende Ataxie beim Menschen. Sie manifestiert sich klinisch initial als spinozerebelläre Ataxie, im weiteren Krankheitsverlauf tritt meist eine hypertrophe Kardiomyopathie hinzu, deren Komplikationen häufig für den frühen Tod der Patienten verantwortlich sind.
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Basierend auf eigenen in-vitro Voruntersuchungen soll nun im Rahmen einer Pilotstudie die Sicherheit und Wirksamkeit von Erythropoetin in der Behandlung von Patienten mit Friedreich Ataxie untersucht werden. Diese Untersuchung stellt nach bisher frustranen Therapieversuchen (z.B. Desoxyferramin, Idebenone) einen gänzlich neuen Therapieansatz dar.
    HAUPTZIELKRITERIUM: Stabile Aufregulierung von FRATAXIN bei Patienten mit Friedreich Ataxie über 8 Wochen.
    E.2.2Secondary objectives of the trial
    NEBENZIELKRITERIEN: Klinische Evaluierung nach 12 Monaten mittels Rating Scales (Ataxie, Lebensqualität), klinische Surogatmarker (MRI, SSEP), stabile Frataxinaufregulierung über 1 Jahr.
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    5.2 Einschlusskriterien
    • Alter > 18. Lj.
    • Diagnose einer Friedreich Ataxie (genetisch + klinisch)
    • Einwilligungsfähigkeit muss gegeben sein.
    • Schriftliche Einverständniserklärung zur Teilnahme liegt vor
    E.4Principal exclusion criteria
    • Hb > 0,5 l/l zu Beginn der Studie
    • Thrombozytose
    • Jede maligne Erkrankung
    • Jede andere z.B. chronisch entzündliche Erkrankung
    • Chronische Alkoholerkrankung
    • Schwerer Diabetes mellitus Typ I und II (HbA1c >8%)
    • Chronische Leberinsuffizienz
    • Epilepsie
    • Herzinsuffizienz (NYHA > 2)
    • Vorgeschichte mit thrombotischen/thrombembolischen Ereignissen
    • Antikoagulation
    • Schwangerschaft oder Stillen
    • Eisenmangel
    • Vitamin B12, Folsäuremangel
    • Kardiovaskuläre Erkrankungen vor allem arterielle Hypertonie
    • Schwere psychiatrische Erkrankung
    • Bekannte Überempfindlichkeit gegen Erythropoetin
    • Teilnahme an anderen klinischen Studien
    E.5 End points
    E.5.1Primary end point(s)
    Kann die Gabe von Erythropoetin in einer Dosierung von 70 I.E./kg KG subkutan dreimal pro Woche zu einer stabilen Aufregulierung von FRATAXIN bei Patienten mit Friedreich Ataxie führen und die Progression der Friedreich Ataxie beeinflussen?

    4.1 Primäres Zielkriterium

    • Stabile Aufregulierung von FRATAXIN bei Patienten mit Friedreich Ataxie über 8 Wochen.

    Erläuterung zum Hauptzielkriterium
    • Bisher konnten nur bei Personen Untersuchungen dazu durchgeführt werden, die aus anderen Gründen Erythropoetin benötigen (Dialysepflichtige Niereninsuffizienz, u.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 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 Information not present in EudraCT
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Information not present in EudraCT
    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) Information not present in EudraCT
    E.7.4Therapeutic use (Phase IV) Information not present in EudraCT
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 Information not present in EudraCT
    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
    Veränderung der klinischen Progression nach 6 und 12 Monaten, gemessen mit der FRDA Rating Scale (Subramony 2002), ICARS, SARA
    Veränderung der Lebensqualität gemessen mit SF-12 und EQ-5D nach 12 Wochen, 3, 6, und 12 Monaten
    •Veränderungen/Verbesserung sogenannter Marker für „oxidative Stress“ Erfassung/Veränderung der zentralen Leitungszeiten der somatosensiblen evozierten Potentiale nach 6 und 12 Monaten
    Erfassung/Veränderung des Durchmessers des Myelons im craniocervikalen Übergang
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    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 Information not present in EudraCT
    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 2006-04-05. Yes
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    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.3.3.6.1Details of subjects incapable of giving consent
    Schwangerschaftstest vor und während der Studie, Verhütumg während der Studie, bisher bei Erythropeotin keine Teratogenität bekannt
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    F.4.2.2In the whole clinical trial 12
    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 Decision2006-06-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2006-06-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-11-01
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