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Evaluating midostaurin with chemotherapy for pediatric 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

Phase 2 Interventional Novartis · NCT03591510

This study is testing if adding a new drug called midostaurin to standard chemotherapy can help children with FLT3-mutated Acute Myeloid Leukemia feel better and improve their treatment outcomes.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment24 (estimated)
Ages3 Months to 17 Years
SexAll
SponsorNovartis Industry-sponsored
Drugs / interventionschemotherapy
Locations35 sites (Aurora, Colorado and 34 other locations)
Trial IDNCT03591510 on ClinicalTrials.gov

What this trial studies

This clinical trial assesses the safety, efficacy, and pharmacokinetics of midostaurin when combined with standard chemotherapy in pediatric patients newly diagnosed with FLT3-mutated Acute Myeloid Leukemia (AML). The study is divided into two parts: the first part aims to determine the recommended dose of midostaurin, while the second part evaluates its safety and effectiveness. Participants will undergo multiple treatment blocks consisting of induction and consolidation chemotherapy, followed by a prolonged phase of midostaurin therapy. The total treatment duration may extend up to 17 cycles, depending on patient response and recovery.

Who should consider this trial

Good fit: Ideal candidates include pediatric patients with newly diagnosed FLT3-mutated AML who meet specific health criteria.

Not a fit: Patients with concurrent malignancies or specific types of leukemia, such as acute promyelocytic leukemia, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve outcomes for children with FLT3-mutated AML, potentially leading to higher remission rates.

How similar studies have performed: Previous studies have shown promise in using midostaurin for FLT3-mutated AML, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Aurora, Colorado and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions FLT3-mutated Acute Myeloid LeukemiaPKC412Acute Myeloid LeukemiaAMLFLT3-mutatedpediatric populationmidostaurinmidostaurin combined with standard chemotherapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.