MyeChild 01


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

Trial Overview and Summary

  • MyeChild-01 Trial Synopsis v2.0 16-Jun-2020 (PDF 388KB)

AML is a rare disease in children and teenagers (70 cases per annum in children less than 16 years in the UK), but is a significant cause of childhood cancer mortality. This trial is an International collaboration and will recruit patients with newly diagnosed AML, high risk myelodysplastic syndrome (MDS) defined as greater than 10% blasts in the bone marrow, and isolated myeloid sarcoma (MS) up to their 18th birthday. Children with myeloid leukaemia of Down syndrome (ML DS) and acute promyelocytic leukaemia (APL) are excluded. The expected number of patients recruited per year is 120-150 or up to 700 cases in 5-6 years and is sufficient to address the four randomised questions proposed.

All major national groups report similar outcomes for childhood AML: overall survival (OS) 65-70%, event-free survival (EFS) 50-60% and cumulative incidence of relapse (CIR) 35-40% (consensus of the International Berlin Frankfurt Munster (I-BFM) AML group, 2011). Whilst advances in supportive care and better salvage therapy after relapse have led to a moderate improvement in OS, the CIR remains unacceptably high with relapsed disease the commonest cause of death. This study plans to build on experience gained from previous UK, French and international trials and to test a number of strategies with the potential to improve outcome: 1) intensive anthracycline or anthracenedione therapy combined with cytarabine in induction, 2) induction intensification with a higher dose of gemtuzumab ozogamicin, 3) assessment of fludarabine, a purine analogue, in consolidation and 4) evaluation of reduced intensity conditioning (RIC) in allogeneic haemopoietic stem cell transplantation (HSCT) in 1st complete remission (CR1). In addition, risk group stratification will direct therapy and will include cytogenetic/molecular genetic characteristics, morphological response to induction therapy and minimal residual disease (MRD) assessment of treatment response. Different MRD methodologies will be studied for their predictive value. The treatment choices and risk stratification are now discussed with relevance to previous UK and French studies and the literature.

Please note that the trials team cannot give individuals clinical advice. Patients and their families should contact their treating physician to discuss trials for which they may be eligible

Chief Investigator: Professor Brenda Gibson
Coordinating Sponsor: University of Birmingham
Funders: CRUK (Cancer Research UK)
Disease Site: Haematological
Trial Type: Clinical Trial of an Investigational Medical Product
Status: Open to recruitment
Open to new sites?  No
Recruitment start date: April 2016
Anticipated Recruitment end date August 2023
CRCTU Trial Management Team: Children's Cancer Trials Team
Trial E-mail Address:

Trial Protocol

Please Note:

Clinical trial protocols are complex technical documents which should only be used for the treatment of subjects taking part in the trial. Patients who are interested in taking part in the trial are advised to talk to their health care professional or refer to CancerHelp website see the link below.

Investigators please ensure you have R&D approval for this specific version of the protocol before using as a reference. 

MyeChild-01 Protocol v4.0 10-Nov-2021 (PDF 2714KB)


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