A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML

To join this study, a child or teenager must have been recently diagnosed with Acute Myeloid Leukemia (AML) and doctors must have confirmed that their cancer has the 'FLT3 mutation'. They should be between 3 months and 17 years of age. They also need to be generally well enough to take part, as measured by certain health assessments.

There are some conditions that would prevent a child from joining. For instance, if they have other types of cancer, certain specific AML types (like AML with Philadelphia Chromosome or Acute Promyelocytic Leukemia), or if the cancer has spread to their brain or spinal cord in a noticeable way. Children who have already been treated significantly for this specific type of mutation (FLT3 inhibitor) are also usually not able to join. The study also requires certain blood test results to be within a healthy range.

If your child joins this study, they will receive a combination of midostaurin and chemotherapy. The treatment schedule is divided into several 'blocks' – two initial intensive treatment blocks, followed by three further treatment blocks. After these, they will continue to receive midostaurin on its own for 12 more cycles. Each treatment block has a specific duration, typically lasting up to 42 days, or until initial recovery or if the disease is still present.

Throughout the study, your child will take midostaurin by mouth twice a day. The total treatment duration is planned for up to 17 cycles, which includes the five initial blocks and the 12 cycles of midostaurin only. There will be regular check-ups and tests to monitor their health and to see how the treatment is working. After completing the main treatment, there will also be a follow-up period where doctors will continue to monitor their health.