Global Study on Midostaurin with Chemotherapy for Newly Diagnosed Children with FLT3 Mutated Acute Myeloid Leukemia (AML)

Sponsor: Novartis Pharmaceuticals

Sponsor score: 60
Inactive

ClinicalTrials ID:NCT03591510

Provider View

This clinical trial is testing the safety and effectiveness of a drug called midostaurin, combined with standard chemotherapy, in children newly diagnosed with a specific type of leukemia called FLT3-mutated Acute Myeloid Leukemia (AML).

Patient Parameters

Program Overview

The study aims to find out if midostaurin, when used with standard chemotherapy, is safe and effective for children with newly diagnosed FLT3-mutated Acute Myeloid Leukemia (AML). The trial has two parts: Part 1 will determine the best dose of midostaurin, and Part 2 will evaluate its safety and effectiveness. Both parts include two initial treatment phases, three follow-up treatment phases, and a year of ongoing midostaurin therapy, followed by a monitoring phase.

Description

This study is testing a treatment plan that includes the drug midostaurin, taken by mouth twice a day, along with standard chemotherapy for children with a specific type of leukemia called FLT3-mutated Acute Myeloid Leukemia (AML). The treatment is divided into several phases:

  • Induction Phase: Patients will first receive chemotherapy for 8 to 12 days. If they have the FLT3 mutation, they will also take midostaurin for 14 days. If the leukemia goes into remission and blood counts recover, they will move to the next phase.

  • Consolidation Phases: There are three blocks of treatment, each with chemotherapy and midostaurin. Patients will continue to the next block if their leukemia stays in remission and blood counts recover.

  • Post-Consolidation Therapy: If patients remain in remission, they will take midostaurin alone for 12 cycles, each lasting 28 days.

In Part 1, small groups of patients will receive different doses of midostaurin to find the safest and most effective dose. In Part 2, all patients will receive the dose determined in Part 1.

The study will monitor patients for side effects and how well the treatment works in controlling the leukemia.

Eligibility Criteria

Inclusion Criteria

  • Newly diagnosed with a specific type of leukemia called FLT3-mutated Acute Myeloid Leukemia (AML).
  • Must have a confirmed FLT3 mutation before starting midostaurin.
  • Must have a performance status score of 60 or higher, indicating they are able to carry out normal activities.
  • Must have liver function tests (AST and ALT) no more than 3 times the normal limit.
  • Must have a total bilirubin level no more than 1.5 times the normal limit.
  • Must have a creatinine clearance of at least 30 ml/min, indicating adequate kidney function.

Exclusion Criteria

  • Have another type of cancer or specific types of leukemia, such as AML with Philadelphia Chromosome or Acute Promyelocytic Leukemia.
  • Have leukemia that has spread to the central nervous system and is causing symptoms.
  • Have leukemia that is only outside the bone marrow or is secondary AML or MDS.
  • Have received prior treatment with a FLT3 inhibitor, except for up to one week before joining the study (excluding midostaurin).

Other specific criteria may apply as defined by the study protocol.

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