HEM iSMART-D: Trametinib + Dexamethasone + Chemotherapy in Children With Relapsed or Refractory Hematological Malignancies

This study is designed for children and young adults. To be considered, you must have been diagnosed with acute lymphoblastic leukaemia (ALL) or lymphoblastic lymphoma (LBL) when you were between 1 and 18 years old. You can join the study up until your 21st birthday. Additionally, your cancer must have either returned after previous treatment or not responded to it.

An important requirement is that doctors need to have already checked your cancer cells and found specific genetic changes in what's called the RAS-RAF-MAPK pathway. This study is specially designed for cancers with these particular changes. You also need to be in reasonably good health overall, meaning your important organs like kidneys and liver are working well enough, and you meet certain weight requirements (e.g., at least 7kg if under 6 years old, or at least 10kg if older than 6).

If you are interested, your parents or legal guardian will need to give their permission, and if you're old enough, you'll also be asked to agree. Before joining, doctors will check to make sure you meet all the necessary health and cancer-specific criteria for your safety and to ensure the study is right for you.

If you join this study, you will receive a combination of medicines: Trametinib, Dexamethasone, Cyclophosphamide, Cytarabine, and some chemotherapy directly into your spinal fluid (called intrathecal chemotherapy). The specific dose and how often you get these will be determined by the study doctors.

You will have regular hospital visits for treatments, blood tests, and other checks to see how you are responding and to monitor any side effects. These checks might include imaging scans like X-rays, CT scans, or MRI scans. Doctors will also take samples of your bone marrow or spinal fluid at specific times to see how the cancer is reacting to the treatment.

The total duration of your participation will depend on how you respond to the treatment and the study plan, but there will be a follow-up period even after treatment ends to continue monitoring your health. The medical team will provide a detailed schedule of visits and procedures if you consider joining.