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

• Status: Recruiting
• Trial end date: 2029-02-15
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Cytarabine; Daunorubicin; Etoposide; Etoposide phosphate; Fludarabine; Fludarabine phosphate; Idarubicin; Midostaurin; Mitoxantrone; Staurosporine

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