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    Summary
    EudraCT Number:2010-022235-10
    Sponsor's Protocol Code Number:ITCC-015/EWOG-MDS-Azacytidine-2010
    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:2013-04-16
    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 number2010-022235-10
    A.3Full title of the trial
    A Phase I/II study of Azacitidine (Vidaza®) in pediatric patients with newly diagnosed or relapsed high-grade pediatric MDS or JMML
    Eine Phase I/II Studie mit Azacitidin (Vidaza®) an Kindern und Jugendlichen mit der Neudiagnose oder Rezidivdiagnose eines Hochrisiko-MDS oder einer JMML
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Azacitidine in children with MDS or JMML
    Azacitidin bei Kindern und Jugendlichen mit MDS oder JMML
    A.3.2Name or abbreviated title of the trial where available
    Azacitidine in high grade MDS and JMML pediatric patients
    A.4.1Sponsor's protocol code numberITCC-015/EWOG-MDS-Azacytidine-2010
    A.5.4Other Identifiers
    Name:NTR 2578Number:Nederlands Trial Register
    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 SponsorErasmus MC
    B.1.3.4CountryNetherlands
    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 supportGo4Children Foundation
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportCelgene
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTrial Office
    B.5.2Functional name of contact pointS. Ramnarain
    B.5.3 Address:
    B.5.3.1Street AddressDr. Molewaterplein 60
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 GJ
    B.5.3.4CountryNetherlands
    B.5.4Telephone number31107036325
    B.5.5Fax number31107036681
    B.5.6E-mailresearch-kocr@erasmusmc.nl
    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 vidaza
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe Ltd
    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 numberORPHA86359
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Powder for suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAZACITIDINE
    D.3.9.1CAS number 320672
    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
    Advanced Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
    fortgeschrittenes Myelodysplastisches Syndrom (MDS) und Juvenile Myelomonozytäre Leukämie (JMML)
    E.1.1.1Medical condition in easily understood language
    MDS und JMML
    Präleukämie und JMML
    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 17.0
    E.1.2Level LLT
    E.1.2Classification code 10054439
    E.1.2Term Juvenile chronic myelomonocytic leukemia
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10068361
    E.1.2Term MDS
    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 establish the recommended dose and preliminary efficacy of azacitidine in children with newly diagnosed and relapsed advanced MDS or JMML.
    Festlegen einer Dosisempfehlung und Erhebung von vorläufigen Daten zur Wirksamkeit von Azacitidine bei Kindern mit der Diagnose eines Hochrisiko-MDS oder eines MDS-Rezidivs oder JMML
    E.2.2Secondary objectives of the trial
    -To determine the safety and tolerability of azacitidine in newly diagnosed and relapsed advanced MDS and JMML
    -To determine (preliminary) the hematological remission rate in these patients
    -To describe the durability of response and long-term follow-up, including that of patients undergoing stem-cell transplant after treatment with azacitidine
    -To determine the plasma pharmacokinetic parameters of azacitidine
    -To study the pharmacodynamic effects of azacitidine in pediatric advanced MDS or JMML
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In this study 4 subgroups of patients are eligible, which will be enrolled in 4 different strata:

    •stratum 1: newly diagnosed patients with advanced MDS (RAEB or RAEB-t) in a ‘pre stem cell transplantation window’.
    •stratum 2: relapsed patients with advanced MDS in a ‘re-transplantation window’. At relapse azacitidine may also be continued when a 2nd transplant is not feasible, as long as the patient benefits from treatment.
    •stratum 3: newly diagnosed patients with JMML in a ‘pre-stem cell transplantation window’.
    •stratum 4: relapsed patients with JMML in a ‘re-transplantation window’. Azacitidine may also be continued when a 2nd transplant is not feasible and as long as the patient benefits from treatment.
    •straum 5:newly diagnosed or relapsed patients with secondary advanced MDS, occurring after
    chemotherapy, radiotherapy and or stem-cell transplantation, or secondary cases after
    prior treatment for aplastic anemia. Note: secondary MDS cases after bone-marrow failures or familial cases are not eligible


    General conditions:
    •Advanced primary or secondary MDS or JMML confirmed by the diagnostic criteria as specified in the EWOG-MDS 2006 protocol (see appendix 1)
    •1 month to ≤ 18 years old
    •Lansky play score ≥ 60; or Karnofsky performance status ≥ 60 (appendix 2)
    •Life expectancy ≥ 3 months
    •Normal renal function defined as less than or equal to NCI-CTCAE grade 1 (max 1.5 x ULN).
    •Normal liver function defined as less than or equal to NCI-CTCAE grade 1 (max 2.5 x ULN for transaminases and bilirubin)
    •No chemotherapy within 3 weeks of start of study medication. For 6-MP or low-dose cytarabine in JMML patients 1 week wash-out time is sufficient.
    •For JMML patients: saturation >92% without additional supply of oxygen
    •For JMML patients: peripheral blood monocyte count > 1.0x109/l
    •For relapsed patients following HSCT: recovery of all acute toxic effects of prior chemotherapy/stem-cell transplantation.
    •Able to comply with scheduled follow-up and with management of toxicity.
    •For patients with childbearing potential, a negative test for pregnancy is to be considered before entry on study. If applicable, use of an effective contraceptive method.
    •Written informed consent from patients or from parents or legal guardians for minor patients, according to local law and regulations.
    E.4Principal exclusion criteria
    Prior or current history:
    •Other serious illnesses or medical conditions
    •Genetic abnormalities indicative of AML
    •JMML patients in whom a diagnosis of Noonan syndrome is suspected based on clinical history and/or presenting symptoms
    •Patients with secondary MDS with underlying bone-marrow failure syndromes or with familial MDS
    •Isolated extramedullary disease
    •Symptomatic CNS-involvement
    •Current uncontrolled infection
    •Cardiac toxicity (shortening fraction below 28%)
    •Concurrent treatment with any other anti-cancer therapy is not allowed
    •Pregnant or lactating patients
    •Patients who cannot be regularly followed up for psychological, social, familial or geographic reasons
    •Patient with expected non compliance to toxicity management guidelines
    •Prior treatment with a demethylating agent
    •Allergy to azicitidine or mannitol.
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective of this study is to establish a recommended dose and preliminary efficacy of azacitidine in children with newly diagnosed or relapsed advanced MDS or JMML.

    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary endpoint may be limited by Dose Limiting toxicities in cycle 1 but otherwise full evaluation of the primary endpoint is possible after of treatment and after end of study.
    E.5.2Secondary end point(s)
    Other objectives concern:
    •To determine the safety and tolerability of azacitidine in newly diagnosed and relapsed MDS and/or JMML.
    •To determine (preliminary) the hematological remission rate in these patients.
    •To describe the durability of response and long-term follow-up (at least 1 year after end of treatment), including that of patients undergoing stem-cell transplant after treatment with azacitidine.
    •To determine the plasma pharmacokinetic parameters of azacitidine.
    •To study the pharmacodynamic effects of azacitidine in pediatric MDS or JMML.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For most endpoints after 3 cycles, and others after 1 year after HSCT or 1 year after last treatment with azacitidine when no HSCT is given
    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 Yes
    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 Yes
    E.7.1.3.1Other trial type description
    Phase I-II
    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 No
    E.8.1.2Open Yes
    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 Yes
    E.8.2.3.1Comparator description
    historical data when time to transplant was not bridged with chemotherapy
    E.8.2.4Number of treatment arms in the trial4
    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 EEA15
    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
    Last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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: 65
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    children 1 month of age until children 12 years of age
    Kinderen vanaf 1 maand tot 12 jaar
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 65
    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)
    follow up (safety, survival, and new therapy) until 1 year after HSCT or last treatment in case HSCT did not take place.
    Follow up (veligheid, overleving en nieuwe therapy) tot 1 jaar na stamceltransplantatie of laatste gift indien er geen stamceltransplantatie is gegeven.
    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 ITCC
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation DCOG-ECTC
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation EWOG-MDS
    G.4.3.4Network Country Netherlands
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-05-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-07
    P. End of Trial
    P.End of Trial StatusOngoing
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