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    Summary
    EudraCT Number:2013-000018-39
    Sponsor's Protocol Code Number:AMLBFM2012
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-03-10
    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 number2013-000018-39
    A.3Full title of the trial
    Clinical trial for the treatment of acute myeloid leukemia in children and adolescents
    Klinische Prüfung zur Behandlung der akuten myeloischen Leukämien bei Kindern und Jugendlichen
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trials for the treatment of acute myeloid leukemia in children and adolescents
    Klinische Prüfung zur Behandlung der akuten myeloischen Leukämien bei Kindern und Jugendlichen
    A.3.2Name or abbreviated title of the trial where available
    AML-BFM 2012
    A.4.1Sponsor's protocol code numberAMLBFM2012
    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 SponsorGesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH gGmbH)
    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 supportDeutsche Krebshilfe e. V.
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversitätsklinikum Essen, Kinderklinik III
    B.5.2Functional name of contact pointAML-BFM
    B.5.3 Address:
    B.5.3.1Street AddressHufelandstraße 55
    B.5.3.2Town/ cityEssen
    B.5.3.3Post code45122
    B.5.3.4CountryGermany
    B.5.4Telephone number00492017233755
    B.5.5Fax number00492017235386
    B.5.6E-mailaml-bfm@uk-essen.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 Evoltra
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme
    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 numberORPHA72855
    D.3 Description of the IMP
    D.3.1Product nameEvoltra
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCLOFARABINE
    D.3.9.1CAS number 123318-82-1
    D.3.9.4EV Substance CodeSUB21902
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Etopophos
    D.2.1.1.2Name of the Marketing Authorisation holderBistol-Myers Squibb
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameEtopophos
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNETOPOSIDE PHOSPHATE
    D.3.9.3Other descriptive nameETOPOSIDE PHOSPHATE
    D.3.9.4EV Substance CodeSUB13772MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    AML
    AML
    E.1.1.1Medical condition in easily understood language
    acute myeloid leukemia
    Akute Myeloische Leukämie
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10000886
    E.1.2Term Acute myeloid leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. improvement of event-free survival of children and adolescents with AML by clofarabine in combination with cytarabine and liposomal daunorubicin in the induction therapy.
    2. reduction of therapy toxicity and improve quality of life by a shortened maintenance therapy without worsening the prognosis.
    1. Verbesserung des ereignisfreien Überlebens von Kindern und Jugendlichen mit AML durch Clofarabin in Kombination mit Cytarabin und liposomalem Daunorubicin in der Induktionstherapie.
    2. Verringerung der Therapietoxizität und Verbesserung der Lebensqualität durch eine verkürzte Erhaltungstherapie, ohne die Prognose zu verschlechtern.
    E.2.2Secondary objectives of the trial
    3. improvement of event-free survival by a zyto-/molekulargenetisch and MRD-based risk classification. Comprehensive biological characterization to improve treatment stratification and as a basis of molecular therapeutic options.
    4. Measurement of the side effects of the combined therapy of molecular active substances in combination with conventional chemotherapy.
    5. detection of molecular relapse after completion of intensive chemotherapy followed by MRD monitoring in peripheral blood (up to 18 months from diagnosis)
    3. Verbesserung des ereignisfreien Überlebens durch eine zyto-/molekulargenetisch und MRD basierte Risikoklassifikation. Umfassende biologische Charakterisierung zur Verbesserung der Therapiestratifizierung und als Grundlage molekularer Therapieoptionen.
    4. Erfassung der Nebenwirkungen der kombinierten Therapie molekular-wirksamer Substanzen in Kombination mit konventioneller Chemotherapie.
    5. Erfassung eines molekularen Rezidivs nach Abschluss der intensiven Chemotherapie durch MRD-Monitoring im peripheren Blut (bis Monat 18 ab Diagnose)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • diagnosis of AML (according to WHO classification 2008)
    • Acute leukemia with mixed line assignment and myeloid dominance (MPAL; according to WHO classification 2008: included dominant myeloid biphenotypic leukemia, and acute undifferentiated leukemia (AUL))
    • Age 0 to 18 years old
    • Consent for study participation and for data processing and data dissemination
    • Diagnose einer AML (lt. WHO Klassifikation 2008)
    • Akute Leukämie mit gemischter Linienzuordnung und myeloischer Dominanz (MPAL; lt. WHO-Klassifikation 2008: eingeschlossen dominant myeloisch biphänotypische Leukämien und akute undifferenzierte Leukämien (AUL))
    • Alter 0 bis 18 Jahre
    • Einwilligungserklärung zur Studienteilnahme und zur Datenverarbeitung und Datenweitergabe
    E.4Principal exclusion criteria
    • Patients with acute promyelocytic leukemia / AML with t (15, 17); PML / RARA
    • pre-existing conditions / syndromes which preclude treatment
    • Patients with trisomy 21 and ML-DS and / or transient myeloproliferative syndrome (reference to the TMD-prevention study or the ML-DS 2006 Study)
    • Acute leukemias bilineäre
    • Secondary acute myeloid leukemia observation patients
    • refusal of treatment
    • Participation in another clinical trial, the Invention could affect the questions of this study
    • Pregnancy
    • Lactation
    • Hypersensitivity to clofarabine
    • patients of reproductive age who reject an effective pregnancy prevention
    • previous treatment with cytostatic drugs for more than 14 days
    • Patienten mit einer akuten Promyelozytenleukämie / AML mit t(15;17); PML/RARA
    • Vorerkrankung/Syndrome, die eine Behandlung ausschließen
    • Patienten mit Trisomie 21 und ML-DS und/oder transientem myeloproliferativem Syndrom (Verweis an die TMD-Präventionsstudie oder die ML-DS 2006 Studie)
    • Akute bilineäre Leukämien
    • Sekundäre akute myeloische Leukämien, Beobachtungspatienten
    • Ablehnung der Therapie
    • Teilnahme an einer anderen klinischen Prüfung, deren Invention die Fragestellungen dieser Studie beeinträchtigen könnten
    • Schwangerschaft
    • Stillzeit
    • Überempfindlichkeit gegen Clofarabin
    • Patienten im fortpflanzungsfähigen Alter, die eine effektive Schwangerschaftsverhütung ablehnen
    • Vortherapie mit zytostatischen Medikamenten von mehr als 14 Tagen
    E.5 End points
    E.5.1Primary end point(s)
    1: Event-free survival of patients randomized
    2: Disease-free survival from randomization 2
    1: Ereignisfreies Überleben der randomisierten Patienten
    2: Erkrankungsfreies Überleben ab Randomisierung 2
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 33% have occurred or 66% of the expected under the null hypothesis events, an interim analysis of randomization 1 is performed. Four years after the end of the patients receiving the final evaluation (including final report and publication) is provided.
    Nachdem 33% bzw 66% der unter der Nullhypothese erwarteten Ereignisse aufgetreten sind, wird eine Zwischenauswertung der Randomisierung 1 durchgeführt. Vier Jahre nach dem Ende der Patientenaufnahme ist die endgültige Auswertung (einschließlich Abschlußbericht und Publikation) vorgesehen.
    E.5.2Secondary end point(s)
    Leukemia-free survival and overall survival
    Disease-free survival correlated to minimal residual disease
    Detection of molecular relapse
    Ansprechkinetik of minimal residual disease: AML t (8, 21); inv (16), MLL rearrangement, Flt3-ITD/TDK, c-KIT mutation, NPM1, WT1 mutation
    relapse incidence
    Measure quality of life through toxicity monitoring
    Leukämiefreies Überleben und Gesamtüberleben
    Erkrankungsfreies Überleben in Korrelation zur minimalen Resterkrankung
    Erfassung des molekularen Rezidivs
    Ansprechkinetik der minimalen Resterkrankung: AML t(8;21); inv(16), MLL-Rearrangement, Flt3-ITD/TDK, c-kit-Mutation, NPM1-, WT1-Mutation
    Rezidivinzidenz
    Erfassung der Lebensqualität durch Toxizitätsmonitoring
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 33% have occurred or 66% of the expected under the null hypothesis events, an interim analysis of randomization 1 is performed. Four years after the end of the patients receiving the final evaluation (including final report and publication) is provided.
    Nachdem 33% bzw 66% der unter der Nullhypothese erwarteten Ereignisse aufgetreten sind, wird eine Zwischenauswertung der Randomisierung 1 durchgeführt. Vier Jahre nach dem Ende der Patientenaufnahme ist die endgültige Auswertung (einschließlich Abschlußbericht und Publikation) vorgesehen.
    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 Yes
    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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA63
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Czech Republic
    Germany
    Slovakia
    Switzerland
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years9
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years9
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    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: 625
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 625
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 625
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 625
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 625
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 625
    F.1.2Adults (18-64 years) No
    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 state45
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 600
    F.4.2.2In the whole clinical trial 625
    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)
    Depending on the disease, the study participants are to participate in the clinical trial, healed the corresponding relapse therapy are supplied as part of recurrence in accordance with or died. If further treatment is required, this is carried out regularly by the treating physician according to the specifications of the current guidelines.
    Abhängig vom Krankheitsverlauf sind die Studienteilnehmer nach Teilnahme an der klinischen Prüfung, geheilt, werden im Rahmen eines Rezidivs nach Maßgabe der entsprechenden Rezidivtherapie versorgt oder sind verstorben. Sollte eine Weiterbehandlung erforderlich sein, erfolgt sie regulär durch den behandelnden Arzt nach Vorgaben der aktuellen Leitlinien.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation AML-BFM Study Group
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-05-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2019-03-01
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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