A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML
A Phase II, Open-label, Single Arm Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy and as a Single Agent Post-consolidation Therapy in Children With Untreated FLT3-mutated AML
ClinicalTrials.gov Identifier: NCT03591510
Novartis Reference Number: CPKC412A2218
Last Update: Jan 23, 2023
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
Study Description
This study will evaluate the safety, efficacy and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatrics patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia. The study has two parts: Part 1 to define the Recommended Phase 2 Dose, and Part 2 to evaluate safety and tolerability and efficacy of midostaurin. Both parts will consist of 2 induction blocks, 3 consolidation blocks, 12 cycles of post-consolidation consisting of continuous therapy with midostaurin, and a follow-up phase.
Interventions
Eligibility Criteria
Inclusion Criteria:
Documented Diagnosis of previously untreated de novo AML according to WHO 2016 criteria
Presence of a FLT3 mutation status 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.
Other protocol-defined inclusion/exclusion criteria may apply
Study Locations
Contacts
Have a question?
Call 1-888-669-6682 or email [email protected]