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    The EU Clinical Trials Register currently displays   38927   clinical trials with a EudraCT protocol, of which   6396   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2011-002567-17
    Sponsor's Protocol Code Number:TUD-MIDOKI-052
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-09-12
    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 ConcernedGermany - BfArM
    A.2EudraCT number2011-002567-17
    A.3Full title of the trial
    A single-arm phase II trial to assess the efficacy of Midostaurin (PKC412) added to standard primary therapy in patients with newly diagnosed c-KIT or FLT3-ITD mutated t(8;21) AML
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Behandlung von Patienten mit einer Akuten Myeloischen Leukämie mit einer c-KIT oder FLT3-ITD Mutation in Verbindung mit einer t(8;21) Mutation mit Midostaurin zusätzlich zur Standard-Chemotherapie
    A.3.2Name or abbreviated title of the trial where available
    MIDOKIT
    A.4.1Sponsor's protocol code numberTUD-MIDOKI-052
    A.5.4Other Identifiers
    Name:Novartis Study codeNumber:CPKC412ADE01T
    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 SponsorTechnische Universität Dresden
    B.1.3.4CountryGermany
    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 supportNovartis Pharma GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTechnische Universität Dresden
    B.5.2Functional name of contact pointHead of Clinical Trial Dept.
    B.5.3 Address:
    B.5.3.1Street AddressFetscherstraße 74
    B.5.3.2Town/ cityDresden
    B.5.3.3Post code01307
    B.5.3.4CountryGermany
    B.5.4Telephone number+4903514583775
    B.5.6E-mailchristoph.roellig@uniklinikum-dresden.de
    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 Rydapt
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/04/214
    D.3 Description of the IMP
    D.3.1Product nameMidostaurin
    D.3.2Product code PKC412
    D.3.4Pharmaceutical form Capsule, soft
    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 INNMidostaurin
    D.3.9.1CAS number 120685-11-2
    D.3.9.3Other descriptive nameMIDOSTAURIN
    D.3.9.4EV Substance CodeSUB21040
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25mg/capsule
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with newly diagnosed c-KIT or FLT3-ITD mutated t(8;21) AML
    E.1.1.1Medical condition in easily understood language
    patients with newly diagnosed c-KIT or FLT3-ITD mutated t(8;21) Acute Myeloid Leukemia
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10060557
    E.1.2Term Acute myelocytic leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t(8;21) AML
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Diagnosis of c-KIT and/or FLT3-ITD mutated t(8;21) AML i.e.
    o >20% myeloid blasts in bone marrow and/or peripheral blood at initial diagnosis
    o Plus cytogenetic diagnosis of aberration t(8;21)/AML1-ETO
    o Plus mutation of c-KIT gene (mut-KIT17 or mut-KIT8) or FLT3-ITD mutation or both c-KIT and FLT3-ITD mutations
    • Chemo-responsive disease* as determined by early bone marrow assessment on day 14-16 after first cycle of standard induction therapy with seven-day continuous infusion of 100-200 mg/m2 cytarabine per day in combination with three doses of daunorubicine, or idarubicine, or mitoxantrone
    • Age 18 – 65 years
    • Fit for intensive chemotherapy as assessed by
    o Adequate liver and renal function as assessed by the following laboratory requirements to be conducted within 7 days prior to screening:
     Total bilirubin ≤ 1.5 times the upper limit of normal
     ALT and AST ≤ 2.5 times upper limit of normal
     Creatinine ≤ 1.5 times upper limit of normal
    o Adequate cardiac function, i.e. left ventricular ejection fraction (LVEF) of >= 50% as assessed by transthoracal twodimensional echocardiography (“M Mode”) or MUGA scan
    • ECOG performance status of 0-2
    • Life expectancy of at least 12 weeks
    • Subject's written informed consent has been obtained
    • Legal capacity (see also exclusion criterion)
    E.4Principal exclusion criteria
    - Primary refractory or previously relapsed AML
    - Non-eligibility for high-dose cytarabine based consolidation, e.g. intolerance to cytarabine
    - Inability to swallow oral medications
    - Symptomatic congestive heart failure as defined by left ventricular ejection fraction (LVEF) of ≤50% as assessed by transthoracal twodimensional echocardiography (“M Mode”) or MUGA scan
    - Subject without legal capacity who is unable to understand the nature, significance and consequences of the study
    - Investigational drug therapy outside of this trial during or within 4 weeks of study entry
    - Known or persistent abuse of medication, drugs or alcohol
    E.5 End points
    E.5.1Primary end point(s)
    2-year event-free Survival
    E.5.1.1Timepoint(s) of evaluation of this end point
    evaluation during study and in follow-up visits up to 2 year after study entry
    E.5.2Secondary end point(s)
    Time to relapse (TTR), Cumulative incidence of relapse (CIR), Overall survival (OS), Relapse-free survival (RFS), morphologic and molecular CR rate, incidence of AEs/SAEs, MRD kinetics
    E.5.2.1Timepoint(s) of evaluation of this end point
    evaluation during study and in follow-up visits up to 2 year after study entry
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 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 concerned12
    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 No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 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.1.2.1Number of subjects for this age range: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 state18
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 18
    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)
    same as normal treatment for that condition
    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 Decision2012-02-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-30
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