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

Launched by NOVARTIS PHARMACEUTICALS · Jul 7, 2018

Keywords

ClinConnect Summary

This clinical trial is exploring the use of a medication called midostaurin combined with standard chemotherapy to treat children and teens who have just been diagnosed with a specific type of blood cancer known as FLT3-mutated Acute Myeloid Leukemia (AML). The study aims to find out how safe this combination is, how well it works, and how the body processes midostaurin. The trial has two parts: the first part will determine the best dose of midostaurin to use, and the second part will look at how well the treatment is tolerated and its effectiveness over several treatment cycles.

To be eligible for this study, patients must be newly diagnosed with AML and have a confirmed FLT3 mutation, which is a specific genetic change associated with this type of cancer. They should also have a certain level of health and specific lab results. Participants in the trial will receive the combination treatment in blocks, followed by continuous therapy with midostaurin, and will be monitored closely throughout the process. It's important to know that patients with certain other health conditions or treatments will not be eligible to join. If you're considering this study for your child, it could be a valuable opportunity to access new treatment options while contributing to important medical research.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Documented Diagnosis of previously untreated de novo AML according to WHO 2016 criteria
• • Presence of a FLT3 mutation status as measured/confirmed by a designated lab with results available prior first dose of Midostaurin
• • Patients with Lansky or Karnofsky performance status equal or superior to 60
• • Patient with the following laboratory value : AST and ALT ≤ 3times ULN
• • Serum Total bilirubin ≤ 1.5times ULN
• • Estimated creatinine clearance ≥30ml/min
• Exclusion Criteria:
• • Any concurrent malignancy, AML with Philadelphia Chromosome, AML-DS, JMML
• • Symptomatic leukemic CNS involvement
• • Isolated extramedullary leukemia, secondary AML and MDS
• • Acute Promyelocytic Leukemia with the PML RARA rearrangement
• • Patient who have received prior treatment with a FLT3 inhibitor. However, up to 1 week of FLT3 inhibitor (except midostaurin) exposure prior to study enrollment is permissible.
• • Other protocol-defined inclusion/exclusion criteria may apply