Last updated on June 2019

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


Brief description of study

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 the Part 2 to evaluate efficacy and safety of midostaurin. All patients will follow the same treatment regimen consisting in 2 Induction blocks, 3 consolidation blocks, 12 cycles of post-consolidation consisting of continuous therapy with midostaurin, and a follow-up phase.

Detailed Study Description

This trial is an open label, multi center single arm study to evaluate twice daily oral midostaurin with standard induction, consolidation chemotherapy with sequential midostaurin therapy for 5 treatment blocks (2 induction blocks, 3 consolidation blocks, followed by single agent midostaurin post consolidation therapy for 12 cycles.

the total maximum planned duration on treatment is 17 cycles ( 5 blocks and 12 cycles). a block is defined as the time from start of study treatment to the time of hematopoietic recovery, at the latest at Day (D) 42, or determination of persistent disease, which occur first.

patient will receive the firs course of induction chemotherapy according to local standard and duration is from 8 to 12 days. Upon FLT3 mutation is confirmed, patient will receive midostaurin for 14 days. After determination of remission and hematopoietic recovery, patient will receive Block 2.

Block 2 FLADx treatment duration is D1 to D6, and midostaurin from D8 to D21. patient who achieve hematopoietic recovery at the latest at D42 from the first day of block 2 will receive block 3.

Block3 consolidation HAM treatment duration is D1 to D4, followed by midostaurin D8 to D21 .patient who achieve hematopoietic recovery at the latest at D42 from the first day of block 3 will receive block 4.

Block 4 HA3E treatment duration is D1 to D5 followed by midostaurin D8 to D21. Patient who achieve hematopoietic recovery at the latest at D42 from the first day of block 4will receive block 5.

Block 5 HiDAC treatment duration is D1 to D3 followed by midostaurin D8 to D21. Patient in continuous remission with hematopoietic recovery will receive continuous post consolidation therapy of midostaurin, during 12 cycles ( 28 days per cycle).

in Part 1 of the study, patients in cohort of 3 will receive sequential midostaurin administered at 30mg/m2bid. if the 30mg/m2 bid is well tolerated as measured by the Dose Limited Toxicity ( DLT) rate during block 1, additional patients in cohort of 3 will be treated with sequential midostaurin at 60mg/m2 bid.

when the recommended 2 dose (RP2D) is confirmed, subsequent patients will be treated in the part 2 of the study at the RP2D.

Clinical Study Identifier: NCT03591510

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Recruitment Status: Open


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