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

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.

Conditions

FLT3-mutated Acute Myeloid Leukemia

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
FLT3-mutated Acute Myeloid Leukemia
Intervention / Treatment

-

Contacts and Locations

Aurora

Novartis Investigative Site, Aurora, Colorado, United States, 80045

Miami

Novartis Investigative Site, Miami, Florida, United States, 33155

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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.

Ages Eligible for Study

3 Months to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novartis Pharmaceuticals,

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

2029-02-15