MyeChild 01: Treating children with acute myeloid leukaemia

Phase 3

• Conditions: Acute myeloid leukaemia; Cancer; Acute megakaryoblastic leukaemia

• Registration Number: ISRCTN12389567
• Lead Sponsor: Cancer Research Clinical Trials Unit (CRCTU)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-02-03
• Last Update Posted: 28 November 2023
• Study Completion: 2032-05-31

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 700

Inclusion Criteria

Current participant inclusion criteria as of 02/09/2021:
Inclusion criteria for trial entry:
1. A diagnosis of AML/high risk myelodysplastic syndrome (MDS)/isolated myeloid sarcoma (either de novo or secondary)
2. Aged <18 years
3. No prior chemotherapy or biological therapy for AML other than that permitted in the protocol
4. Normal cardiac function (fractional shortening =28% or ejection fraction of 55%)
5. Fit for protocol chemotherapy
6. Documented negative pregnancy test for patients of childbearing potential
7. Patient agrees to use effective contraception (patients of childbearing potential)
8. 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 take part in the embedded dose escalation study (please contact the trial office for further information).
1. Patient meets the inclusion criteria for trial entry
2. Aged either:
2.1. =12 months for the major dose finding study
2.2. =12 weeks and <12 months
3. Normal renal function defined as calculated creatinine clearance =90 ml/min/1.73m² (calculated using the BNFc formula, or that in use locally)
4. Normal hepatic function defined as total bilirubin =2.5 x upper limit of normal (ULN) for age unless it is caused by leukaemic involvement, Gilbert’s syndrome, or a similar disorder
5. ALT or AST =10 x ULN for age
6. 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:
Centres must be formally activated to be able to deliver treatment with gemtuzumab ozogamicin (please contact the trial office for further information).
1. Patient meets the inclusion criteria for trial entry
2. Aged either:
2.1. =12 months
2.2. =12 weeks and <12 months
2.3. =28 days and and <12 weeks
3. Normal renal function defined as calculated creatinine clearance =90 ml/min/1.73m² (calculated using the BNFc formula, or that in use locally)
4. Normal hepatic function defined as total bilirubin =2.5 x upper limit of normal (ULN) for age unless it is caused by leukaemic involvement, Gilbert’s syndrome, or a similar disorder
5. ALT or AST =10 x ULN for age
6. 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):
1. Patient meets the inclusion criteria for trial entry
2. Aged either:
2.1. =12 months
2.2. =12 weeks and <12 months (once R2 open in patients aged =12 weeks and <12 months)
3. Normal renal function defined as calculated creatinine clearance =90 ml/min/1.73m² (calculated using the BNFc formula, or that in use locally)
4. Normal hepatic function defined as total bilirubin =2.5 x upper limit of normal (ULN) for age unless it is caused by leukaemic involvement, Gilbert’s syndrome, or a similar disorder
5. ALT or AST =10 x ULN for age
6. Written informed consent from the patient and/or parent/legal guardian

Inclusion criteria for participation in R4:
1. Patient meets the inclusion criteria for trial entry
2. Induction treatment as per MyeChild 01 protocol or treated with 1 or 2 courses of mitoxantrone & cytarabine ± treatment intensification with FLA-Ida off trial
3. Patient is in CR or CRi defined as <5% blasts confirmed by flow cytometry//molecular/FISH in a bone marrow aspirate taken

Exclusion Criteria

Current participant exclusion criteria as of 02/09/2021:
1. Acute promyelocytic leukaemia (APL)
2. Myeloid leukaemia of Down Syndrome (ML DS)
3. Blast crisis of chronic myeloid leukaemia
4. Relapsed or refractory AML
5. Bone marrow failure syndromes
6. Prior anthracycline exposure which would inhibit the delivery of study anthracyclines
7. Concurrent treatment or administration of any other experimental drug or with any other biological therapy for AML/high risk MDS/isolated MS
8. Pregnant or lactating

Previous participant exclusion criteria:
1. Acute promyelocytic leukaemia (APL)
2. Myeloid leukaemia of Down Syndrome (ML DS)
3. Blast crisis of chronic myeloid leukaemia
4. Relapsed or refractory AML
5. Bone marrow failure syndromes
6. Prior anthracycline exposure which would inhibit the delivery of study anthracyclines
7. Concurrent treatment or administration of any other experimental drug or with any other biological therapy for AML
8. Pregnant or lactating females

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Gemtuzumab ozogamicin dose finding study:<br>Incidence of dose limiting toxicities (DLTs) are evaluated up to day 45 post course 1 and course 2 of induction chemotherapy.