Status:

COMPLETED

A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML

Lead Sponsor:

Novartis Pharmaceuticals

Conditions:

Acute Myeloid Leukemia

Eligibility:

All Genders

18-70 years

Phase:

PHASE2

Brief Summary

This study evaluated the efficacy and safety of midostaurin in combination with daunorubicin/cytarabine induction, high dose cytarabine consolidation and midostaurin single agent continuation therapy ...

Detailed Description

This was a Phase II, multi-center trial consisting of two parts; Part 1: an open label, safety evaluation part in Japan only (minimum of three evaluable subjects) and Part 2: a double-blind, randomize...

Eligibility Criteria

Inclusion

  • Diagnosis of AML (≥ 20% blasts in the bone marrow based on WHO 2016 classification). Patients with APL (acute promyelocytic leukemia) with PML-RARA are not eligible
  • Documented presence of an ITD and/or TKD activating mutation in the FLT3 gene, as determined by analysis in a Novartis designated laboratory An exception will be patients who are enrolled into the part 1 in Japan, who may be treated with midostaurin irrespective of AML FLT3 genotype.
  • Patients must meet the following laboratory value criteria that indicate adequate organ function at the screening visit:
  • Estimated creatinine clearance ≥ 30 ml/min
  • Total bilirubin ≤ 1.5 x ULN, except in the setting of isolated Gilbert syndrome
  • Aspartate transaminase (AST) ≤ 3.0 x ULN
  • Alanine transaminase (ALT) ≤ 3.0 x ULN
  • Suitability for intensive chemotherapy in the judgment of the investigator

Exclusion

  • Neurologic symptoms suggestive of CNS leukemia unless CNS leukemia has been excluded by a lumbar puncture. Patients with CSF fluid positive for AML blasts are not eligible
  • Developed therapy-related AML after prior radiotherapy (RT) or chemotherapy for another cancer or disorder
  • Known hypersensitivity to midostaurin, cytarabine or daunorubicin or to any of the excipients of midostaurin/placebo, cytarabine or daunorubicin
  • Abnormal chest X-ray unless the abnormality represents a non-active, or non-clinically significant finding, such as scarring (subjects with controlled non active lung infection are eligible)
  • Known impairment of gastrointestinal (GI) function or GI disease that might alter significantly the absorption of midostaurin
  • Cardiac or cardiac repolarization abnormality
  • Pregnant or nursing (lactating) women
  • Women of child-bearing potential, unless they are using highly effective methods of contraception during dosing and for 4 months after stopping medication

Key Trial Info

Start Date :

April 6 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

November 14 2022

Estimated Enrollment :

67 Patients enrolled

Trial Details

Trial ID

NCT03280030

Start Date

April 6 2018

End Date

November 14 2022

Last Update

March 20 2025

Active Locations (34)

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Page 1 of 9 (34 locations)

1

Novartis Investigative Site

Pokfulam, Hong Kong

2

Novartis Investigative Site

Nagoya, Aichi-ken, Japan, 466-8650

3

Novartis Investigative Site

Toyoake, Aichi-ken, Japan, 470 1192

4

Novartis Investigative Site

Kashiwa, Chiba, Japan, 277 8577