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    Summary
    EudraCT Number:2012-002692-34
    Sponsor's Protocol Code Number:MitoFibrateCT1-2012
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-02-26
    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 ConcernedAustria - BASG
    A.2EudraCT number2012-002692-34
    A.3Full title of the trial
    Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies
    Klinische Studie zum Nachweis der Sicherheit und Wirksamkeit von
    Bezafibrat als Arzneimitteltherapie für Patienten mit mitochondrialen Muskelerkrankungen
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients suffering from an inborn muscular dysfunction characterized by intramuscular lack of energy supply
    Klinische Studie zum Nachweis der Sicherheit und Wirksamkeit von
    Bezafibrat als Arzneimitteltherapie für Patienten mit angeborener Muskelerkrankung, die zu einer gestörten Energiebereitstellung
    der Muskulatur führt
    A.3.2Name or abbreviated title of the trial where available
    MitoFibrate CT1
    MitoFibrate CT1
    A.4.1Sponsor's protocol code numberMitoFibrateCT1-2012
    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 SponsorUniv.-Klinik für Kinder- und Jugendheilkunde
    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 supportUniv.-Klinik für Kinder- und Jugendheilkunde
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInstitut für angeborene Stoffwechselerkrankungen
    B.5.2Functional name of contact pointClinical trial information
    B.5.3 Address:
    B.5.3.1Street AddressStrubergasse 21
    B.5.3.2Town/ citySalzburg
    B.5.3.3Post code5020
    B.5.3.4CountryAustria
    B.5.4Telephone number00436624420021224
    B.5.6E-mailflorian.lagler@pmu.ac.at
    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 Bezastad
    D.2.1.1.2Name of the Marketing Authorisation holderSTADA Arzneimittel GmbH, Wien
    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 nameBezastad
    D.3.4Pharmaceutical form Coated tablet
    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 INNBEZAFIBRATE
    D.3.9.1CAS number 41859-67-0
    D.3.9.3Other descriptive name2-[4-[2-(4-Chlorobenzamido)ethyl]phenoxy]isobutyric Acid
    D.3.9.4EV Substance CodeSUB05810MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 placeboTablet
    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
    mitochondrial disorder
    Genetisch bedingte mitochondriale Muskelerkrankungen
    E.1.1.1Medical condition in easily understood language
    Inborn mitochondrial diseases leading to an abberant energy supply in muscular tissue severly affecting muscular performance and sometimes leading to psycho-mental disability and breathing failure
    Genetische mitochondriale Erkrankung,die zu einer gestörten intramuskulären Energiebereitstellung führt. Folgen: Leistungsintoleranz bis zu psychomentaler Behinderung und Versagen der Atemmuskulatur
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    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
    Recent findings derived from animal and cell culture studies demonstrate an upregulation of synthesis and activity of mitochondrial enzymes under bezafibrat and thus point to a beneficial effect of this substance for the therapy of mitochondrial myopathies (H. Tyynismaa et al. 2010, T. Wenz 2008 und 2010). This findings are further substantiated by clinical observations in course of compassionate use of Bezafibrat for patinets with mitochondrial myopathies at the centres planned to participate in this trial (Reutlingen, München, Salzburg, Innsbruck). The main objective of the trial is therefore the systematic evaluation of efficacy and safety of Bezafibrat as a therapeutic option for patients with mitochondrial myopathies.
    Aktuelle Ergebnisse von Studien mit Tiermodellen und Patientenzelllinien weisen jedoch darauf hin, dass das Arzneimittel Bezafibrat zu einer vermehrte Synthese und Aktivierung von mitochondrialen Enzymen führt und so eine positive Wirkung bei mitochondrialen Muskelerkrankungen erzielt werden könnte (H. Tyynismaa et al. 2010, T. Wenz 2008 und 2010). Einzelne individuelle Heilversuche in den beteiligten Zentren(Reutlingen, München, Salzburg,Innsbruck) und anderen Zentren bekräftigen den Eindruck dass dies therapeutisch genutzt werden kann.Im Rahmen der geplanten klinischen Studie sollen die Wirksamkeit und Sicherheit von Bezafibrat in der Behandlung von mitochondrialen Muskelerkrankungen systematisch evaluiert werden. Nur so kann rational beurteilt werden, ob sich die erhoffte Wirksamkeit bestätigt und ob unter Abwägung relevanter Risiken eine generelle Therapieempfehlung gerechtfertigt ist.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Ability to walk
    Age: 2-50 years
    confirmed Complex-I-defect or PDHc-defect
    ability to cope with the requirements for study participation
    Gehfähigkeit
    Patienten im Alter von 2-50 Jahre mit nachgewiesenem Complex-I-Defekt oder PDHc-Defekt
    Patienten/Patienteneltern die in der Lage sind den Anforderungen der Studienteilnahme gerecht zu werden
    E.4Principal exclusion criteria
    previously known intolerance or other contra-indications for bezafibrat
    Patienten mit bekannter Unverträglichkeit oder anderer Kontraindikationen gegen die Einnahme von Bezafibrat
    E.5 End points
    E.5.1Primary end point(s)
    time intervall until pre-defined manifestation of disease progression measured by walking-distance in a 6-minutes-walking test
    disease progression is defined as a decline of >20% in performance
    Primärer Studienendpunkt ist die Zeit bis zum Auftreten einer definierten Krankheitsprogredienz gemessen an:
    Gehstrecke im 6-Minuten-Gehtest
    Krankheitsprogredienz entspricht per definitionem einer Verschlechterung im Gehtest um >20%
    E.5.1.1Timepoint(s) of evaluation of this end point
    The subjective disease progression is measured every 4 weeks by the patients or their parents by Newcastle Scale Section 4. Once the score points to a disease progression, patients visit the study site where the primary endpoint is evaluated. Elsewise, the primary endpoint is routinely evaluated 3 and 6 months post randomization. A decline of >20% in the 6-minutes-walking test leads to termination of study participation. Elsewise, termination is routinely scheduled at study visit 6 months post randomization.
    alle 4 Wochen wird die subjektive Krankheitsprogredienz durch den Patienten oder dessen Eltern mittels Newcastle Scale Section 4 erhoben. Ergibt sich Hinweis auf Progredienz, so erfolgt außerdordentliche Visite und Evaluierung des Endpunkts. Andernfalls erfolgt die Bewertung des Endpunkts 3 Monate sowie 6 Monate nach der Randomisierung. Eine Verschlechterung um 20% des 6-Minuten-Gehtests führt zur Beendigung der Studienteilnahme. Allenfalls endet die Teilnahme bei der 6 Monats Visite ab Randomisierung.
    E.5.2Secondary end point(s)
    subjective disease progression: Newcastle Paediatric Mitochondrial Disease Rating Scale
    function of the autonomous nervous sytem: ANS-test
    Subjektive Krankheitsprogredienz: Newcastle Paediatric Mitochondrial Disease Rating Scale
    Autonome Nervensystem-Funktion: ANS-Testbatterie
    E.5.2.1Timepoint(s) of evaluation of this end point
    Newcastle Paediatric Mitochondrial Disease Rating Scale: every 4 weeks
    ANS-Test: every 3 months
    Newcastle Paediatric Mitochondrial Disease Rating Scale: alle 4 Wochen
    ANS-Testbatterie: alle 3 Monate während der 9 monatigen Gesamtdauer
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    „randomized withdrawal“-Studie mit einer Parallelgruppenphase nach einer offenen Phase
    „randomized withdrawal“-study with an open phase followed by a parallel-group phase
    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 trial2
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    LVLS
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 8
    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: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    children
    Kinder
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 16
    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)
    After the subject has ended the participation in the trial bezafibrate therapy can be continued on compassionate-use level.
    Die Studienteilnehmer können nach Beendigung der Studienteilnahme weiterhin auf Wunsch die Bezafibrat -Therapie auf Basis eines individuellen Heilversuchs fortführen
    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 Decision2013-05-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-26
    P. End of Trial
    P.End of Trial StatusOngoing
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