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Midostaurin Access Program for Newly Diagnosed FLT3 (ITD or TKD) Mutated AML Adult Patients Eligible for Standard Induction and Consolidation Chemotherapy

Conditions
FLT3 Mutation, Internal Tandem Duplication (ITD) or Tyrosine Kinase Domain (TKD)
Acute Myeloid Leukemia (AML) With
Registration Number
NCT02624570
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

The purpose of this study is to provide access to Midostaurin and gather additional safety data on the combination of Midostaurin and standard of care for adult patients with newly diagnosed Fms-like tyrosine kinase receptor (FLT3) mutated Acute Myeloid Leukemia (AML) who are eligible for standard induction and consolidation chemotherapy.

Detailed Description

The purpose of this study is to provide Midostaurin (study drug) treatment to newly diagnosed FLT3 mutated (ITD or TKD) AML adult patients (18 years or older) eligible for standard standard induction (cytarabine + daunorubicin/Idarubicin) and consolidation (cytarabine) chemotherapy.

Recruitment & Eligibility

Status
NO_LONGER_AVAILABLE
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

Written informed consent must be obtained prior to any screening procedures. Patients must meet all Inclusion Criteria:

  • Patient is able to communicate well with the investigator, to understand and comply with the requirements of the study.
  • Patients must be 18 years of age or older; elderly patients must be fit to receive intensive induction and consolidation chemotherapy
  • Patients must have a documented FLT3 mutation (ITD or TKD)
  • Patients must have an ECOG Performance Status of ≤ 2
  • Patients must have a documented unequivocal diagnosis of AML according to WHO 2008 classification (>20% blasts in the bone marrow and/or peripheral blood), excluding M3 (acute promyelocytic leukemia).
  • Patients requiring intrathecal chemotherapy must have a minimum washout of 48 hours prior to the first dose of midostaurin
  • AML patients with a history of antecedent treatment for myelodysplasia (MDS), e.g. azacitidine or decitabine, remain eligible for treatment on this study. These agents must have been discontinued for a period of at least 30 days or 5 half-lives of the drug (whichever is greater) before midostaurin can be administered.
  • Secondary AML, e.g. patients with antecedent history of treatment for prior malignancy Patients must have the following laboratory values (Direct Bilirubin ≤ 2.5 x ULN, Serum Creatinine ≤ 2.5 x ULN)
Exclusion Criteria

Patients eligible for this study must not meet any of the following criteria:

  • Prior therapy for AML with the following exceptions: (emergency leukapheresis, emergency treatment for hyperleukocytosis with hydroxyurea for ≤ 5 days, cranial RT for CNS leukostasis (one dose only), growth factor/cytokine support)
  • Patients with LVEF less than 45% (by echocardiogram or MUGA) or symptomatic congestive heart failure, Class III or IV according to New York Heart Association (NYHA) classification
  • Patients with any uncontrolled illness, including, but not limited to, acute or chronic pancreatitis or uncontrolled infection
  • QTc >500 msec on screening ECG. History of hypersensitivity to any drugs or metabolites of similar chemical classes as the IMP.
  • Participation in a prior investigational interventional (drug) study with administration of the investigational product within 30 days or 5 half-lives of the investigational product, whichever is longer.
  • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods.
  • Sexually active males should not father a child during this study and for upto 5 months following.

Study & Design

Study Type
EXPANDED_ACCESS
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (44)

Mayo Clinic Arizona

🇺🇸

Phoenix, Arizona, United States

The Jordan Research and Education Center ABSMC Comprehensive Cancer Ctr

🇺🇸

Berkeley, California, United States

City of Hope National Medical Center Department of Hematology & HCT

🇺🇸

Duarte, California, United States

St. Judes Medical Center

🇺🇸

Fullerton, California, United States

University of Calif Irvine Medical Center Family Comp Cancer Cntr

🇺🇸

Orange, California, United States

Kaiser Permanente Northwest Kaiser

🇺🇸

Denver, Colorado, United States

Poudre Valley Hospital Poudre Valley Hospital -U of C

🇺🇸

Fort Collins, Colorado, United States

Memorial Healthcare System Memorial Healthcare System

🇺🇸

Hollywood, Florida, United States

UF Health Cancer Center at Orlando Health Orlando Health

🇺🇸

Orlando, Florida, United States

H Lee Moffitt Cancer Center and Research Institute SC - 5

🇺🇸

Tampa, Florida, United States

Scroll for more (34 remaining)
Mayo Clinic Arizona
🇺🇸Phoenix, Arizona, United States

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