ML-DS 2018: Trial Investigates CPX-351 for Down Syndrome Patients with Leukemia or MDS

A new trial, ML-DS 2018, has been launched in March 2024, with a restart in October 2024 after modifications for safety reasons. This trial will investigate CPX-351 in the first 2 treatment cycles and a dose reduction of cytarabine in the 4th treatment cycle in Down Syndrome patients with Myeloid Leukemia (ML) or Myelodysplastic Syndrome (MDS).

Children with Down syndrome and myeloid leukemia (ML-DS) have a superior outcome compared to non-DS patients but suffer from higher constitutional susceptibility to cytotoxic drugs.

The primary objective of this study is to improve the outcome (in terms of event-free survival (EFS)) in Down Syndrome patients with Myeloid Leukemia or Myelodysplastic Syndrome, with as secondary objective the reduction of severe adverse events.

CPX-351 is a chemotherapeutic agent that combines the two active ingredients: daunorubicin and cytarabine contained in a fixed ratio into a fat droplet envelope. The drug is already being used with promising results and has been approved to treat acute myeloid leukemia in adults. The drug has not yet been approved for use in children, although it has already been successfully used in studies with children with leukemia. In addition, the trial will investigate if a reduction of treatment intensity in course 4 for patients with a good response (<0.1% blasts in the bone marrow after course 1) does not result in inferior event-free survival.

The international sponsor of the ML-DS 2018 is the German Pediatric Oncology Group (GPOH gGmbH, Germany). In Belgium the study is coordinated by the University Hospital Ghent (UZ Gent), with Prof. dr. Barbara De Moerloose acting as the national coordinating investigator. 4 Belgian paediatric haematology-oncology centres are participating in this trial (UZ Gent, UZ Leuven, CUSL and CHC Montlégia). For Belgium we expect to recruit 4 patients over a period of 3 years.

This trial is open in Belgium thanks to the financial support of KickCancer.