Clinical trial · Interventional
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| FLT3-mutated Acute Myeloid Leukemia | — | UNRESOLVED | — |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cytarabine | Drug | Cytarabine | ALIAS |
| Daunorubicin or idarubicin | Drug | — | UNRESOLVED |
| Etoposide | Drug | Etoposide | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Midostaurin | Drug | Midostaurin | ALIAS |
| Mitoxantrone | Drug | Mitoxantrone | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Chemotherapy followed by Midostaurin
- description
- In Part 1, midostaurin with standard induction (Block 1 induction according to local practice, Block 2 induction containing fludarabine, cytarabine, daunorubicin/idarubicin) and consolidation (Block 3: cytarabine + mitoxantrone, Block 4: cytarabine + etoposide, Block 5: cytarabine) followed by single agent midostaurin post-consolidation therapy. In Part 2, midostaurin with standard induction (Block 1 induction according to local practice, Block 2 induction containing cytarabine + mitoxantrone) and consolidation (Block 3: cytarabine + etoposide, Block 4: cytarabine + mitoxantrone, Block 5: cytarabine) followed by single agent midostaurin post-consolidation therapy.
- interventionNames
- Drug: Midostaurin
- Drug: Fludarabine
- Drug: Cytarabine
- Drug: Daunorubicin or idarubicin
- Drug: Mitoxantrone
- Drug: Etoposide
Primary outcomes (3)
- measure
- Part 1 of the study: Occurence of dose limiting toxicities (DLT)
- timeFrame
- From the start of midostaurin treatment in Block 1 to the end of Block 2, from Day 1 to Day 84
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
- Maximum age
- 17 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available