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Acute Myeloid LeukaemiaPhase 3Myechild01NCT02724163

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

A Phase 3 study testing Gemtuzumab ozogamicin, Liposomal daunorubicin and Mitoxantrone in people with Acute Myeloid Leukaemia, run by University of Birmingham at 68 sites in 6 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages Up to 17 years
Conditions the study is for
Acute Myeloid Leukaemia
What is being tested?
Gemtuzumab ozogamicin, Liposomal daunorubicin and Mitoxantrone
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
700 participants
Where
68 sitesin 6 countries
Study dates
Apr 2016to Dec 2032
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
Show all 52Show fewer
  • 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.
  • 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.
  • 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.

Who cannot join

  • 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.

Trial locations and contacts

68 sites in 6 countries.

68 sites in 6 countries

France28 sites

  • Centre Hospitalier Universitaire Amiens - Picardie

    Amiens

  • Centre Hospitalier Universitaire D'angers

    Angers

  • Centre Hospitalier Regional Universitaire Besancon - Hopital Jean Minjoz

    Besançon

  • Centre Hospitalier Universitaire De Bordeaux - Hopital Pellegrin

    Bordeaux

  • Centre Hospitalier Regional Universitaire Brest - Hopital Morvan

    Brest

  • Centre Hospitalier Universitaire De Caen

    Caen

  • Centre Hospitalier Universitaire De Clermont-ferrand

    Clermont-Ferrand

  • Centre Hospitalier Universitaire Dijon Bourgogne - Hopital D'enfants

    Dijon

  • Centre Hospitalier Universitaire De Grenoble

    Grenoble

  • Hopital Jeanne Dr Flandre

    Lille

  • Centre Hospitalier Universitaire De Limoges

    Limoges

  • Centre Leon Berard

    Lyon

  • Hopital De La Timone

    Marseille

  • Centre Hospitalier Regional Universitaire Montpellier - Hopital Arnaud-de-villeneuve

    Montpellier

  • Centre Hospitalier Universitaire De Nancy

    Nancy

  • Centre Hospitalier Universitaire De Nantes

    Nantes

  • Centre Hospitalier Universitaire De NICE

    Nice

  • Hopital Armand Trousseau

    Paris

  • Hopital Robert Debre

    Paris

  • Hopital Saint Louis

    Paris

  • Centre Hospitalier Universitaire De Poitiers

    Poitiers

  • Chu De Reims

    Reims

  • Centre Hospitalier Universitaire De Rennes - Hopital Sud

    Rennes

  • Centre Hospitalier Universitaire De Rouen

    Rouen

  • Centre Hospitalier Universitaire Saint-etienne

    Saint-Etienne

  • Strasbourg Hautepierre

    Strasbourg

  • Centre Hospitalier Universitaire De Toulouse - Hopital Des Enfants

    Toulouse

  • Centre Hospitalier Regional Universitaire De Tours - Hopital Clocheville

    Tours

Other trials in France

United Kingdom20 sites

  • Royal Aberdeen Children's Hospital

    Aberdeen, AB25 2ZG

  • Aberdeen Royal Infirmary, NHS Grampian

    Aberdeen, AB25 2ZN

  • Royal Belfast Hospital for Sick Children

    Belfast, County Antrim, BT12 6BE

  • Birmingham Children's Hospital NHS Foundation Trust

    Birmingham, B4 6NH

  • University Hospitals Bristol NHS Foundation Trust

    Bristol, BS1 3NU

  • Addenbrookes Hospital, Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ

  • Cardiff and Vale University Health Board, Noah's Ark Children's Hospital for Wales

    Cardiff, CF14 4XW

  • NHS Lothian, Royal Hospital for Sick Children

    Edinburgh, EH9 1LF

  • NHS Greater Glasgow and Clyde, The Royal Hospital for Children

    Glasgow, G51 4TF

  • Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust

    Leeds, LS9 7TF

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, L12 2AP

  • University College London Hospitals NHS Foundation Trust

    London, NW1 2PG

  • The Royal Marsden NHS Foundation Trust

    London, SW3 6JJ

  • Great Ormond Street Hospital For Children NHS Trust

    London, WC1N 3JH

  • Royal Manchester Childrens' Hospital , Central Manchester University Hospitals NHS Foundation Trust

    Manchester, M13 9WL

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    Newcastle, NE7 7DN

  • Nottingham University Hospitals NHS Trust

    Nottingham, NG7 2UH

  • John Radcliffe Hospital, Oxford Radcliffe Hospitals NHS Trust

    Oxford, OX3 9DU

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH

  • Southampton University Hospitals NHS Trust

    Southampton, SO16 6YD

Other trials in United Kingdom

Switzerland9 sites

  • Kantonsspital Aarau

    Aarau

  • Universitäts-Kinderspital beider

    Basel

  • Ospedale San Giovanni

    Bellinzona

  • Inselspital Bern

    Bern

  • Hug Hopitaux Universitaires De Geneve

    Geneva

  • Centre Hospitalier Universitaire Vaudois Chuv Lausanne

    Lausanne

  • Luzerner Kantonspital - Kinderspital Luzern

    Lucerne

  • Ostschweizer Kinderspital

    Sankt Gallen

  • University Children's Hospital Zurich

    Zurich

Other trials in Switzerland

Australia8 sites

  • Women and Children's Hospital Adelaide

    Adelaide

  • Queensland Children's Hospital

    Brisbane

  • Monash Children's Hospital

    Melbourne

  • Royal Childrens Hospital

    Melbourne

  • John Hunter Children's Hopsital

    New Lambton Heights

  • Perth Children's Hospital

    Perth

  • Sydney Children's Hospital

    Sydney

  • The Childrens Hospital At Westmead

    Westmead

Other trials in Australia

New Zealand2 sites

  • Starship Childrens Hospital

    Auckland

  • Christchurch Hospital

    Christchurch

Other trials in New Zealand

Ireland1 site

  • Our Lady's Hospital for Sick Children

    Dublin

Other trials in Ireland

About this study

Official title: International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia - Incorporating an Embedded Dose Finding Study for Gemtuzumab Ozogamicin in Combination With Induction Chemotherapy

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.
Read the detailed description

MyeChild 01 is an international phase III clinical trial in children with acute myeloid leukaemia (AML); a disease with significant mortality. It will compare two induction chemotherapy regimens: mitoxantrone and cytarabine (current standard treatment) with liposomal daunorubicin and cytarabine. This will test liposomal daunorubicin, which is believed to be less cardiotoxic than similar conventional drugs, although this is unproven. (Randomisation 1 (R1) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug.)

Patients responding well to induction chemotherapy are eligible for a randomisation of two consolidation regimens: high dose cytarabine (current standard treatment) or fludarabine and cytarabine (FLA); a regimen commonly used in patients with relapsed disease, testing whether FLA is more effective in front line therapy than standard consolidation treatment. Patients with cytogenetic features associated with a higher risk of relapse and those responding sub-optimally to induction treatment are candidates for haemopoietic stem cell transplant (HSCT) and are eligible for a randomisation comparing two HSCT conditioning regimens: myeloablative conditioning (MAC) (current UNited Kingdom (UK) standard) or reduced intensity conditioning (RIC). HSCT has not consistently shown benefit in high risk patients because the mortality associated with the procedure has outweighed the advantage from a reduction in relapse risk. This will test whether reducing the intensity of conditioning improves survival by reducing transplant related deaths without increasing the relapse rate. The trial incorporates a dose finding study for gemtuzumab ozogamicin. The aim is to identify the optimum tolerated number of doses of gemtuzumab ozogamicin (up to a total of 3 doses), which can be safely combined with either of the induction chemotherapy regimens and then to compare this number of doses with one dose of gemtuzumab ozogamicin. The intensity of treatment will be directed by cytogenetics/molecular genetics and response assessed by minimal residual disease (MRD) levels measured by flow cytometry and molecular methodology.

Being tested
Gemtuzumab ozogamicin, Liposomal daunorubicin, Mitoxantrone, Fludarabine, Cytarabine
Keywords
Acute Myeloid Leukaemia, Children, Gemtuzumab ozogamicin, Liposomal daunorubicin, Mitoxantrone, Randomised controlled trial, Risk stratification, Minimal residual disease, Stem cell transplant
Medical subject headings
Leukemia, Myeloid, Acute, Neoplasm, Residual

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Gemtuzumab ozogamicinDrug

    Also called Mylotarg

    Antibody-conjugated chemotherapy agent.

  • Liposomal daunorubicinDrug

    Anthracycline

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

  • MitoxantroneDrug

    DNA-reactive agent

  • FludarabineDrug

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

  • CytarabineDrug

    Pyrimidine nucleoside analogue, an antineoplastic agent.

  • BusulfanDrug

    Alkylsulfonate

  • CyclophosphamideDrug

    A nitrogen mustard alkylating agent from the oxazaphosphorine group

Study groups in NCT02724163
Study groupRoleWhat they receive
MitoxantroneActive comparatorCourse 1 * Mitoxantrone: 12 mg/m2 daily by IV infusion over 1 hour on days 1, 2, 3 and 4 (total 4 doses). * Cytarabine:100 mg/m2 12 hourly by IV bolus on days 1-10 inclusive (total 20 doses). Course 2 * Mitoxantrone: 12 mg/m2 daily by IV infusion over 1 hour on days 1, 2 and 3 (total 3 doses). * Cytarabine: 100 mg/m2 12 hourly by IV bolus on days 1-8 inclusive (total 16 doses).
Receives: Drug: Mitoxantrone; Drug: Cytarabine
Liposomal daunorubicinExperimentalRandomisation 1 (R1)) closed early to recruitment on 8th September 2017, due to liposomal daunorubicin manufacturing issues resulting in unavailability of the drug. Course 1 * Liposomal daunorubicin: 80 mg/m2 daily by 1 hour IV infusion on days 1, 3 and 5 (total 3 doses). * Cytarabine: 100 mg/m2 12 hourly by IV bolus on days 1-10 inclusive (total 20 doses). Course 2 * Liposomal daunorubicin: 60 mg/m2 daily by 1 hour IV infusion on days 1, 3 and 5 (total 3 doses). * Cytarabine: 100 mg/m2 12 hourly by IV bolus on days 1-8 inclusive (total 16 doses).
Receives: Drug: Liposomal daunorubicin; Drug: Cytarabine
Gemtuzumab Ozogamicin Dose Finding StudyExperimental* Cohort 1: 1x3mg/m2 IV infusion over 2hours on day 4. * Cohort 2: 2x3mg/m2 IV infusion over 2hours on day 4 and day 7. * Cohort 3: 3x3mg/m2 IV infusion over 2hours on days 4, 7 and 10.
Receives: Drug: Gemtuzumab ozogamicin
High dose cytarabineActive comparatorTwo courses of Cytarabine: 3 g/m2 12 hourly by IV infusion over 4 hours on days 1, 3 and 5 (total 6 doses).
Receives: Drug: Cytarabine
Fludarabine & cytarabineExperimentalTwo courses of: * Fludarabine: 30 mg/m2 daily by IV infusion over 30 minutes on days 1-5 inclusive (total 5 doses). * Cytarabine: 2 g/m2 daily by IV infusion over 4 hours on days 1-5 inclusive (total 5 doses).The cytarabine infusion should be started 4 hours after the start of the fludarabine infusion
Receives: Drug: Fludarabine; Drug: Cytarabine
Myeloablative conditioningActive comparator* Busulfan Area Under the Curve (AUC) 70-100mg/L x hr by IV infusion over 3 hours, given 12 hourly on days -10 to -7 (8 doses). * Cyclophosphamide 50mg/kg/day by IV infusion over 1 hour, on days -5 to -2 (4 doses).
Receives: Drug: Busulfan; Drug: Cyclophosphamide
Reduced intensity conditioningExperimental* Busulfan AUC60-65mg/L X hr by IV infusion over 3 hours, given 12 hourly on days -5 to -2 (8 doses). * Fludarabine 30mg/m2/day by IV infusion over 30 minutes on days -8 to -3 (6 doses).
Receives: Drug: Fludarabine; Drug: Busulfan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of dose limiting toxicities (DLTs).

    Measured: Incidence of DLTs will be evaluated up to day 45 post course 1 and course 2 of induction chemotherapy.

  2. Event Free Survival (EFS).

    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.

    Measured: Event free survival (EFS) will be evaluated as the time from randomisation one to the first event, up to 16 years.

  3. Event Free Survival (EFS).

    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.

    Measured: Event free survival (EFS) will be evaluated as the time from randomisation two to the first event, up to 16 years..

  4. Relapse free survival (RFS).

    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.

    Measured: 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.

  5. Early treatment related adverse reactions.

    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).

    Measured: Early treatment related adverse reactions will be evaluated at day 100 post-transplant.

  6. Relapse free survival (RFS).

    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.

    Measured: 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.

Secondary outcomes (20)

  1. The nature, incidence and severity of adverse events (AEs) (gemtuzumab ozogamicin dose finding study).

    Measured: Evaluated by day 45 post course 1 and course 2.

  2. Response measured by bone marrow assessment using morphology and minimal residual disease (MRD) assessment (gemtuzumab ozogamicin dose finding study).

    Response is assessed by morphology confirmed by MRD levels measured by flow cytometry, molecular methods or fluorescence in situ hybridisation (FISH) as defined in the protocol, in combination with platelet and neutrophil counts. These results of these assessments will be combined to determine the patient's disease response using the response criteria defined in the protocol.

    Measured: Evaluated by day 45 post course 1 and course 2.

  3. Serum pharmacokinetic (PK) parameters of gemtuzumab ozogamicin: Clearance (CL) (gemtuzumab ozogamicin dose finding study)

    Serum PK parameters will be measured using serial samples taken at multiple timepoints during course 1 and at 1 month post first dose of gemtuzumab ozogamicin as defined in the protocol by dose cohort.

    Measured: Evaluated up to one month after the first dose of gemtuzumab ozogamicin.

  4. Serum pharmacokinetic (PK) parameters of gemtuzumab ozogamicin: Volume of distribution (Vd) (gemtuzumab ozogamicin dose finding study)

    Serum PK parameters will be measured using serial samples taken at multiple timepoints during course 1 and at 1 month post first dose of gemtuzumab ozogamicin as defined in the protocol by dose cohort.

    Measured: Evaluated up to one month after the first dose of gemtuzumab ozogamicin.

  5. Complete remission (CR) (R1 & R2).

    Evaluated using remission status at completion of course 1 and course 2.

    Measured: Evaluated and presented at the completion of course 1 and 2 of treatment up to a maximum of 45 days post each course of treatment

  6. Reasons for failure to achieve CR (R1 & R2).

    Evaluated as resistant disease, induction death or not evaluable.This will be evaluated at completion of course 1 and 2 of treatment, once patient's blood counts have recovered or reason for non-recovery has been determined.

    Measured: Evaluated and presented at the completion of course 1 and 2 of treatment, up to a maximum of 45 days post each course of treatment.

  7. Cumulative Incidence of Relapse (CIR) (all randomisations).

    The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. CIR estimates will be presented at 24 months along with 95% confidence intervals for randomisations 1, 2 and 3, and at 12 months for randomisation 4.

    Measured: Evaluated as time from randomisation to the relevant question to relapse, up to 16 years.

  8. Death in CR (DCR) (R1, R2 & R3).

    The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. DCR estimates will be presented at 24 months along with 95% confidence intervals.

    Measured: Evaluated as time from randomisation to relevant question to date of death from any cause in patients who have achieved CR, up to 16 years.

  9. Event Free Survival (EFS) (R1, R2 & R3).

    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.

    Measured: Evaluated as time from randomisation to the relevant question to the first of failure to achieve CR (recorded as an event on day 1), relapse, secondary malignancy or death from any cause, up to 16 years.

  10. Overall Survival (OS) (all randomisations).

    The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. OS estimates will be presented at 24 months along with 95% confidence intervals for randomisations 1, 2 and 3, and at 12 months for randomisation 4.

    Measured: Evaluated as time from randomisation to the relevant question to death from any cause or date last seen for patients who are alive at the end of the trial, up to 16 years.

  11. Incidence of toxicities (all randomisations).

    Measured: Evaluated 30 days after end of trial treatment.

  12. Incidence of cardiotoxicity (R1, R2 & R4 only).

    Measured: Evaluated 30 days after end of trial treatment.

  13. Incidence of bilirubin of grade 3 of higher (R2 & R4 only).

    Measured: Evaluated 30 days after end of trial treatment.

  14. Incidence of Veno-Occlusive Disease (R2 & R4 only).

    Measured: Evaluated 30 days after end of trial treatment.

  15. Minimal Residual Disease (MRD) clearance after course 1 & 2 (R1 & R2 only).

    Evaluated using MRD result at completion of course 1 and 2 once patient's blood counts have recovered or reason for non-recovery has been determined.

    Measured: Evaluated and presented at completion of course 1 and 2 of treatment, up to a maximum of 45 days post each course of treatment.

  16. Time to haematological recovery (all randomisations).

    Evaluated using the date of haematological recovery (platelets to >=80 x 10^9/L, and neutrophils to >=1.0 x 10^9/L). The primary analysis will be carried out once the last patient has a minimum of 1 year follow up. Time to haematological recovery estimates will be presented at 45 days post course 1 and course 2 of treatment along with 95% confidence intervals.

    Measured: Evaluated by day 45 post course 1 and course 2.

  17. Days in hospital after each course of treatment (all randomisations).

    Total number of days spent in hospital for each course of treatment, collected from date of randomisation until count recovery after final course of treatment, up to a maximum of 45 days post the final course of treatment. This will be summarised per course of treatment.

    Measured: Evaluated once all patients have completed trial treatment.

  18. Incidence of mixed chimerism at day 100 post-transplant (R4 only).

    Measured: Evaluated at day 100 post-transplant.

  19. Treatment Related Mortality (TRM) (R4 only).

    The primary analysis will be carried out once the last patient has a minimum of 1 year follow up which is estimated to be 7 years after the start of recruitment. TRM estimates will be presented at 12 months along with 95% confidence intervals.

    Measured: Evaluated as time in days between randomisation to R4 and death which is unrelated to the underlying disease and considered related to the transplant procedure.

  20. Gonadal function (R4 only).

    The method of assessment will be by scale (Tanner scale) and physiological parameters. This will be evaluated at 1 year post-transplant and at the end of study follow-up.

    Measured: Evaluated at 1 year post-transplant and at the end of follow-up, which is estimated to be through to study completion, an average timeframe of 10 years.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Mar 2016 (estimated)
  2. Study startApr 2016
  3. Primary completionDec 2031 (estimated)
  4. Study completionDec 2032 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
700 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02724163
Sponsor's protocol ID
RG_14-088
Other IDs
2014-005066-30, 2014
Acronym
Myechild01
Status last verified
Jun 2026
First submitted
8 Jan 2016
Last updated
29 Jun 2026
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Gemtuzumab ozogamicin, Liposomal daunorubicin and Mitoxantrone in people with Acute Myeloid Leukaemia, run by University of Birmingham at 68 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 52 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 68 sites in France, United Kingdom, Switzerland, Australia, New Zealand and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of dose limiting toxicities (DLTs). (time frame: Incidence of DLTs will be evaluated up to day 45 post course 1 and course 2 of induction chemotherapy.). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02724163.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2026.

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