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Clinical Trials in Ireland / NCT02724163
Active, not recruiting Phase 3

International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia

NCT02724163 · tracked via the Priya Life Science Ireland tracker
Sponsor
University of Birmingham
Phase
Phase 3
Started
2016-04
Last updated
2026-06-29

Condition(s) studied

Acute Myeloid Leukaemia

Investigational drug(s) / intervention(s)

Gemtuzumab ozogamicinLiposomal daunorubicinMitoxantroneFludarabineCytarabineBusulfanCyclophosphamide

Gemtuzumab ozogamicin: Antibody-conjugated chemotherapy agent.

Liposomal daunorubicin: Anthracycline (Randomisation 1 (R1)) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug.

Mitoxantrone: DNA-reactive agent

Fludarabine: A water-soluble fluorinated nucleotide analogue of the antiviral agent vidarabine.

Cytarabine: Pyrimidine nucleoside analogue, an antineoplastic agent.

Busulfan: Alkylsulfonate

Cyclophosphamide: A nitrogen mustard alkylating agent from the oxazaphosphorine group

Study summary

The main purpose of this study is :

1. To establish which number of doses of gemtuzumab ozogamicin (up to a maximum of 3 doses) is tolerated and can be safety delivered in combination with cytarabine plus mitoxantrone or liposomal daunorubicin in induction
2. To compare mitoxantrone (anthracenedione) \& cytarabine with liposomal daunorubicin (anthracycline) \& cytarabine as induction therapy. (Randomisation 1 (R1) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug.)
3. To compare a single dose of gemtuzumab ozogamicin with the optimum tolerated number of doses of gemtuzumab ozogamicin (identified by the dose-finding study) when combined with induction chemotherapy.
4. To compare two consolidation regimens: high dose cytarabine (HD Ara-C) and fludarabine \& cytarabine (FLA) in standard risk patients.
5. To compare the toxicity and effectiveness of two haemopoietic stem cell transplant (HSCT) conditioning regimens of different intensity: conventional myeloablative conditioning (MAC) with busulfan/cyclophosphamide and reduced intensity conditioning (RIC) with fludarabine/busulfan.

Eligibility

Sex
ALL
Min age
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: Inclusion criteria for trial entry * Diagnosis of acute myeloid leukaemia (AML) /high risk Myelodysplastic syndrome (MDS) (\>10% blasts in the bone marrow)/isolated myeloid sarcoma (MS) (either de novo or secondary). * Age \<18 years at trial entry. * No prior chemotherapy or biological therapy for AML/high risk MDS/isolated MS other than that permitted in the protocol. * Normal cardiac function defined as fractional shortening ≥28% or ejection fraction ≥55%. * Fit for protocol chemotherapy. * Documented negative pregnancy test for female patients of childbearing potential. * Patient agrees to use effective contraception (patients of child bearing potential). * Written informed consent from the patient and/or parent/legal guardian. Inclusion criteria for participation in the gemtuzumab ozogamicin dose finding study: Centres must be formally activated in order to be take part in the embedded dose escalation study. Please contact the trial office for further information. * Patient meets the inclusion criteria for trial entry. * Age: * ≥12 months for the major dose finding study * ≥ 12 weeks and \<12 months for the minor dose finding study * Normal renal function defined as calculated creatinine clearance ≥90ml/min/1.73m2. * Normal hepatic function defined as total bilirubin ≤2.5 upper limit of normal (ULN) for age unless it is caused by leukaemic involvement or Gilbert's syndrome or similar disorder. * Alanine transaminase (ALT) or aspartate transaminase (AST) ≤10 x ULN for age. * Written informed consent from the patient and/or parent/legal guardian. Inclusion criteria for treatment with gemtuzumab ozogamicin for patients not participating in the gemtuzumab ozogamicin dose finding study or R2. * Patient meets the inclusion criteria for trial entry (section 4.1.1) * Age: * ≥12 months * ≥ 12 weeks * ≥28 days and \<12 weeks (patients will receive a maximum of one dose of gemtuzumab ozogamicin) * Normal renal function, defined as calculated creatinine clearance ≥90 ml/min/1.73m2 * Normal hepatic function, defined as total bilirubin ≤2.5 upper limit of normal (ULN) for age and not due to leukaemic involvement or Gilbert's syndrome or similar disorder * ALT or AST ≤10 x ULN for age * Written informed consent from the patient and/or parent/legal guardian Inclusion criteria for participation in R2.(once open to randomisation in the applicable age group) • Patient meets the inclusion criteria for trial entry Patient age: * ≥12 months * ≥12 weeks (once R2 open in patients aged ≥12 weeks and \<12 months) * Normal renal function defined as calculated creatinine clearance ≥90ml/min/1.73m2. * Normal hepatic function defined as total bilirubin ≤2.5 ULN for age and not due to leukaemic involvement or Gilbert's syndrome or similar disorder. * ALT or AST ≤10 x ULN for age. * Written informed consent from the patient and/or parent/legal guardian. Inclusion criteria for participation in R3. * Patient meets the inclusion criteria for trial entry * Induction treatment as per MyeChild 01 protocol or treated with 2 courses of mitoxantrone \& cytarabine off trial. * Minimal residual disease (MRD) response (performed in MyeChild 01 centralised laboratories, see national MyeChild 01 Laboratory Manual): * Patients with good risk cytogenetics/molecular genetics and a MRD level \<0.1% by flow after course 2, or a decrease in transcript levels of \>3 logs after course 2 for those with an informative molecular marker, but without an informative marker of sufficient sensitivity for flow MRD monitoring or * Patients with intermediate risk cytogenetics/molecular genetics with a MRD level \<0.1% by flow after course 1 and course 2, or a decrease in transcript levels of \>3 logs after course 1 and course 2 for those with an informative molecular marker, but without an informative marker of sufficient sensitivity for flow MRD monitoring. * Written informed consent from the patient and/or parent/legal guardian. Inclusion criteria for participation in R4. * Patient meets the inclusion criteria for trial entry * Induction treatment as per MyeChild 01 protocol or treated with 1 or 2 courses of mitoxantrone \& cytarabine ± treatment intensification with fludarabine, cytarabine \& idarubicin (FLA-Ida) off trial. * Patient is in complete remission (CR) or CR with incomplete blood count recovery (CRi) defined as \<5% blasts confirmed by flow cytometry/ molecular/FISH in a bone marrow aspirate taken within 6 weeks prior to randomisation to R4. * Patient meets one of the following criteria and is a candidate for HSCT as per the protocol: * High risk after course 1 (all patients with poor risk cytogenetics and patients with intermediate risk cytogenetics who fail to achieve CR/CRi). * Intermediate risk cytogenetics with MRD \>0.1% after course 1 and 2 measured by flow. If no flow MRD marker of sufficient sensitivity is identified, a molecular MRD marker with a sensitivity of \>0.1% may be used. * Good risk cytogenetics with flow MRD \>0.1% confirmed by a decrease in molecular MRD of \<3 logs or rising transcript levels after course 3 despite treatment intensification (FLA-Ida) and after discussion with the Clinical Co-ordinators. * Availability of a 9-10/10 human leukocyte antigen (HLA) matched family or unrelated donor or 5-8/8 matched cord blood unit with an adequate cell dose as defined by the protocol section 17.1. * Written informed consent from the patient and/or parent/legal guardian. Exclusion Criteria: Exclusion criteria for all randomisations * Acute Promyelocytic Leukaemia. * Myeloid Leukaemia of Down Syndrome. * Blast crisis of chronic myeloid leukaemia. * Relapsed or refractory AML. * Bone marrow failure syndromes. * Prior anthracycline exposure which would inhibit the delivery of study anthracyclines. * Concurrent treatment or administration of any other experimental drug or with any other biological therapy for AML/high risk MDS/isolated MS. * Pregnant or lactating females.

Primary outcome measure(s)

Trial sites (68)

FacilityCityRegionStatus
Women and Children's Hospital Adelaide Adelaide Australia
Queensland Children's Hospital Brisbane Australia
Monash Children's Hospital Melbourne Australia
Royal Childrens Hospital Melbourne Australia
John Hunter Children's Hopsital New Lambton Heights Australia
Perth Children's Hospital Perth Australia
Sydney Children's Hospital Sydney Australia
The Childrens Hospital At Westmead Westmead Australia
Centre Hospitalier Universitaire Amiens - Picardie Amiens France
Centre Hospitalier Universitaire D'angers Angers France
Centre Hospitalier Regional Universitaire Besancon - Hopital Jean Minjoz Besançon France
Centre Hospitalier Universitaire De Bordeaux - Hopital Pellegrin Bordeaux France
Centre Hospitalier Regional Universitaire Brest - Hopital Morvan Brest France
Centre Hospitalier Universitaire De Caen Caen France
Centre Hospitalier Universitaire De Clermont-ferrand Clermont-Ferrand France
Centre Hospitalier Universitaire Dijon Bourgogne - Hopital D'enfants Dijon France
Centre Hospitalier Universitaire De Grenoble Grenoble France
Hopital Jeanne Dr Flandre Lille France
Centre Hospitalier Universitaire De Limoges Limoges France
Centre Leon Berard Lyon France
Hopital De La Timone Marseille France
Centre Hospitalier Regional Universitaire Montpellier - Hopital Arnaud-de-villeneuve Montpellier France
Centre Hospitalier Universitaire De Nancy Nancy France
Centre Hospitalier Universitaire De Nantes Nantes France
Centre Hospitalier Universitaire De NICE Nice France
Hopital Armand Trousseau Paris France
Hopital Robert Debre Paris France
Hopital Saint Louis Paris France
Centre Hospitalier Universitaire De Poitiers Poitiers France
Chu De Reims Reims France
Centre Hospitalier Universitaire De Rennes - Hopital Sud Rennes France
Centre Hospitalier Universitaire De Rouen Rouen France
Centre Hospitalier Universitaire Saint-etienne Saint-Etienne France
Strasbourg Hautepierre Strasbourg France
Centre Hospitalier Universitaire De Toulouse - Hopital Des Enfants Toulouse France
Centre Hospitalier Regional Universitaire De Tours - Hopital Clocheville Tours France
Our Lady's Hospital for Sick Children Dublin Ireland
Starship Childrens Hospital Auckland New Zealand
Christchurch Hospital Christchurch New Zealand
Kantonsspital Aarau Aarau Switzerland

+ 28 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02724163 on ClinicalTrials.gov ↗ ← All trials in Ireland