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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)
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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.
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Study & Design

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

Trial Locations

Locations (44)

City of Hope National Medical Center Department of Hematology & HCT

🇺🇸

Duarte, California, United States

The Jordan Research and Education Center ABSMC Comprehensive Cancer Ctr

🇺🇸

Berkeley, California, United States

St. Judes Medical Center

🇺🇸

Fullerton, California, United States

Memorial Healthcare System Memorial Healthcare System

🇺🇸

Hollywood, Florida, United States

University of Calif Irvine Medical Center Family Comp Cancer Cntr

🇺🇸

Orange, California, United States

Poudre Valley Hospital Poudre Valley Hospital -U of C

🇺🇸

Fort Collins, Colorado, United States

Augusta University Georgia Cancer Center Pharmacy

🇺🇸

Augusta, Georgia, United States

University of Chicago Medical Center

🇺🇸

Chicago, Illinois, United States

Ft Wayne Medical Oncology and Hematology Inc

🇺🇸

Fort Wayne, Indiana, United States

Rush University Medical Center Dept.of Rush UniversityMedCtr.

🇺🇸

Chicago, Illinois, United States

Kaiser Permanente NW Region Kaiser Permanente Northwest

🇺🇸

Clackamas, Oregon, United States

University of Rochester Medical Center Univ of Rochester (7)

🇺🇸

Rochester, New York, United States

Penn State University / Milton S. Hershey Medical Center Penn State Cancer Institute

🇺🇸

Hershey, Pennsylvania, United States

Weill Cornell Medical College NY Presbyterian Hospital

🇺🇸

New York, New York, United States

University of Pennsylvania Cancer Center University of Pen/Abr Canc ctr

🇺🇸

Philadelphia, Pennsylvania, United States

University of Texas Medical Branch University of Texas MB

🇺🇸

Galveston, Texas, United States

Intermountain Healthcare - Huntsman Cancer Clinics Intermountain Healthcare (2)

🇺🇸

Murray, Utah, United States

Virginia Oncology Associates Virginia Oncology Assoc. (6)

🇺🇸

Norfolk, Virginia, United States

Wenatchee Valley Hospital and Clinics

🇺🇸

Wenatchee, Washington, United States

Cheyenne Regional Medical Center

🇺🇸

Cheyenne, Wyoming, United States

Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

Karmanos Cancer Institute Harper Hosp. Invest. Pharmacy

🇺🇸

Detroit, Michigan, United States

Methodist Healthcare System

🇺🇸

San Antonio, Texas, United States

Huntsman Cancer Institute Univ of Utah

🇺🇸

Salt Lake City, Utah, United States

Mayo Clinic Arizona

🇺🇸

Phoenix, Arizona, United States

Henry Ford Hospital SC

🇺🇸

Detroit, Michigan, United States

Vanderbilt University Medical Center, Clinical Trials Center Division Hematology/Oncology

🇺🇸

Nashville, Tennessee, United States

McGovern Medical School at the University of Texas Health

🇺🇸

Houston, Texas, United States

Oncology Consultants Oncology Consultants

🇺🇸

Houston, Texas, United States

Swedish Cancer Institute Cancer Institute

🇺🇸

Seattle, Washington, United States

Veterans Affairs Puget Sound Health Care System VAMC Seattle, WA Divison

🇺🇸

Seattle, Washington, United States

Ochsner Clinic Foundation

🇺🇸

New Orleans, Louisiana, United States

Indiana Blood and Marrow Institute Indiana Blood and Marrow Trans

🇺🇸

Beech Grove, Indiana, United States

Norton Cancer Institute Norton Cancer Institute

🇺🇸

Louisville, Kentucky, United States

Nebraska Cancer Specialist/Missouri Valley Cancer Consortium

🇺🇸

Omaha, Nebraska, United States

Medical University of South Carolina Hematology-Oncology Division

🇺🇸

Charleston, South Carolina, United States

Mayo Clinic Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

University of Louisville / James Graham Brown Cancer Center Louisville 529-539

🇺🇸

Louisville, Kentucky, United States

Kaiser Permanente Northwest Kaiser

🇺🇸

Denver, Colorado, 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

Tulane University Medical Center

🇺🇸

New Orleans, Louisiana, United States

Healthcare Partners Medical Group

🇺🇸

Las Vegas, Nevada, United States

Erlanger Medical Center Erlanger Health System

🇺🇸

Chattanooga, Tennessee, United States

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