Active, not recruiting
Phase 3
International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia
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
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)
- Incidence of dose limiting toxicities (DLTs). — Incidence of DLTs will be evaluated up to day 45 post course 1 and course 2 of induction chemotherapy.
- Event Free Survival (EFS). — Event free survival (EFS) will be evaluated as the time from randomisation one to the first event, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. EFS estimates will be presented at 24 months along with 95% confidence intervals.
- Event Free Survival (EFS). — Event free survival (EFS) will be evaluated as the time from randomisation two to the first event, up to 16 years..
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. EFS estimates will be presented at 24 months along with 95% confidence intervals.
- Relapse free survival (RFS). — Relapse free survival (RFS) will be evaluated as the time of randomisation three to the first relapse or death from any cause, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. RFS estimates will be presented at 24 months along with 95% confidence intervals.
- Early treatment related adverse reactions. — Early treatment related adverse reactions will be evaluated at day 100 post-transplant.
Early treatment related adverse reactions defined as the incidence by day 100 post-transplant of grade 3-5 toxicity for the following systems using the National Cancer Institute (NCI) Common Terminology Criteria v4:
* Cardiac (pericardial effusion/Left ventricular systolic dysfunction).
* Respiratory, thoracic and mediastinal (hypoxia/pneumonitis).
* Gastrointestinal (GI) (diarrhoea/typhlitis/upper and lower GI haemorrhage).
* Investigations (bilirubin).
* Renal and Urinary (acute kidney injury/haematuria).
* Nervous system (seizure).
- Relapse free survival (RFS). — Relapse free survival (RFS) will be evaluated as the time of randomisation four to the first relapse or death from any cause, up to 16 years.
The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. RFS estimates will be presented at 12 months along with 95% confidence intervals.
Trial sites (68)
| Facility | City | Region | Status |
| 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.