Novartis Pharmaceuticals

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

Official Title: 
An Open-label, Multi-center, Expanded Treatment Protocol of Midostaurin (PKC412) in 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.
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.

EA Type
Treatment IND/Protocol
Acute Myeloid Leukemia (AML) With, FLT3 Mutation, Internal Tandem Duplication (ITD) or Tyrosine Kinase Domain (TKD)

Drug: Midostaurin
Midostaurin, also known as PKC412 is a multi-kinase inhibitor of FLT3 (fms-like tyrosine kinase-3)
Other Name: PKC412

Eligibility Criteria: 

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.

Eligibility Gender: 
Eligibility Age: 
Minimum: 18 Years
United States

Mayo Clinic Arizona
Phoenix, Arizona, 85054

Contact: 480-342-2545

Investigator: Lisa Sproat

The Jordan Research and Education Center ABSMC Comprehensive Cancer Ctr
Berkeley, California, 94705

Contact: 510-204-3428

Investigator: Oleg Krijanovski

City of Hope National Medical Center Department of Hematology & HCT
Duarte, California, 91010

Contact: +00 626 256 4673 x62405

Investigator: Al Malki Monzr

St. Judes Medical Center
Fullerton, California, 92835

Investigator: Steven Kim

University of Calif Irvine Medical Center Family Comp Cancer Cntr
Orange, California, 92868

Contact: 714-456-5153

Investigator: Deepa Jeyakumar

Kaiser Permanente Northwest Kaiser
Denver, Colorado, 80209

Contact: 303-861-3302

Investigator: Susan M Freeman

Poudre Valley Hospital Poudre Valley Hospital -U of C
Fort Collins, Colorado, 80528

Investigator: Steven R. Schuster

Memorial Healthcare System Memorial Healthcare System
Hollywood, Florida, 33021

Contact: 954-265-1846

Investigator: Yehuda Deutsch

UF Health Cancer Center at Orlando Health Orlando Health
Orlando, Florida, 32806

Investigator: Julio Hajdenberg

H Lee Moffitt Cancer Center and Research Institute SC - 5
Tampa, Florida, 33612

Contact: 813-745-3685

Investigator: Kendra Sweet

Augusta University Georgia Cancer Center Pharmacy
Augusta, Georgia, 30912

Contact: 706-721-2171

Investigator: Locke J Bryan

Rush University Medical Center Dept.of Rush UniversityMedCtr.
Chicago, Illinois, 60612

Contact: 312-563-2540

Investigator: Melissa Larson

University of Chicago Medical Center
Chicago, Illinois, 60637

Contact: 773-702-4400

Investigator: Hongtao Liu

Indiana Blood and Marrow Institute Indiana Blood and Marrow Trans
Beech Grove, Indiana, 46107

Contact: +1 317 528 7298

Investigator: Michael John Dugan

Ft. Wayne Medical Oncology & Hematology, Inc.
Fort Wayne, Indiana, 46815

Contact: 219-484-8830

Investigator: Sunil Babu

Norton Cancer Institute Norton Cancer Institute
Louisville, Kentucky, 40202

Contact: +1 502-899-3366 X152


Investigator: Don A. Stevens

University of Louisville / James Graham Brown Cancer Center Louisville 529-539
Louisville, Kentucky, 40202

Contact: 502-562-3429

Investigator: Maxim Krim

Tulane University Medical Center
New Orleans, Louisiana, 70112

Contact: 504-588-5135

Investigator: Hana Safah

Oschner Clinic Foundation
New Orleans, Louisiana, 70121

Contact: 312-942-5495

Investigator: Andrew Dalovisio

Tufts Medical Center
Boston, Massachusetts, 02111

Contact: 617-636-7438

Investigator: Kellie Sprague

Karmanos Cancer Institute Harper Hosp. Invest. Pharmacy
Detroit, Michigan, 48201

Investigator: Jay Yang

Henry Ford Hospital SC
Detroit, Michigan, 48202-2689

Contact: 313-916-1784

Investigator: Ahmad Mattour

Mayo Clinic Mayo Clinic
Rochester, Minnesota, 55905

Contact: 507-284-2358

Investigator: Mark R. Litzow

Nebraska Cancer Specialist/Missouri Valley Cancer Consortium
Omaha, Nebraska, 68106

Contact: 402-991-8070

Investigator: Gamini S Soori

Healthcare Partners Medical Group
Las Vegas, Nevada, 89109

Contact: 702-735-7154

Investigator: Vikas Gupta

Weill Cornell Medical College NY Presbyterian Hospital
New York, New York, 10021

Contact: 212-746-6736

Investigator: Gail J Roboz

University of Rochester Medical Center Univ of Rochester (7)
Rochester, New York, 14642

Contact: 724-900-0558

Investigator: Jane L. Liesveld

Kaiser Permanente NW Region Kaiser Permanente Northwest
Clackamas, Oregon, 97015

Contact: +1 503 249 3316

Investigator: Abdul Hai Mansoor

Penn State University / Milton S. Hershey Medical Center Penn State Cancer Institute
Hershey, Pennsylvania, 17033-085

Contact: 717-531-8309

Investigator: David Claxton

University of Pennsylvania Cancer Center University of Pen/Abr Canc ctr
Philadelphia, Pennsylvania, 19104 4283

Contact: 215-615-5858

Investigator: Alexander Perl

Medical University of South Carolina Hematology-Oncology Division
Charleston, South Carolina, 29425

Contact: 843-792-9300

Investigator: Robert K. Stuart

Erlanger Medical Center Erlanger Health System
Chattanooga, Tennessee, 37403

Contact: +1 423 752 5004

Investigator: Robert Matthew Graham

Vanderbilt University Medical Center, Clinical Trials Center Division Hematology/Oncology
Nashville, Tennessee, 37212

Contact: 615-322-6515

Investigator: Stephen Strickland

University of Texas Medical Branch University of Texas MB
Galveston, Texas, 77555-1188

Contact: 409-772-1164

Investigator: Avi B. Markowitz

McGovern Medical School at the University of Texas Health
Houston, Texas, 77004

Contact: 713-486-5660

Investigator: Modupe Idowu

Oncology Consultants Oncology Consultants
Houston, Texas, 77024

Contact: 713-600-0992

Investigator: Julio Peguero

Methodist Healthcare System
San Antonio, Texas, 78229

Contact: +1 210 575 4000

Investigator: Jose Carlos Cruz

Intermountain Healthcare - Huntsman Cancer Clinics Intermountain Healthcare (2)
Murray, Utah, 84107

Contact: +1 801408 3729

Investigator: Julie Asch

Huntsman Cancer Institute Univ of Utah
Salt Lake City, Utah, 84112 0550

Contact: 801-585-2626

Investigator: Tibor Kovacsovics

Virginia Oncology Associates Virginia Oncology Assoc. (6)
Norfolk, Virginia, 23502

Contact: 757-213-5624

Investigator: Scott J. Cross

Swedish Cancer Institute Cancer Institute
Seattle, Washington, 98104

Contact: 206-215-2658

Investigator: Raya Mawad

Veterans Affairs Puget Sound Health Care System VAMC Seattle, WA Divison
Seattle, Washington, 98108

Investigator: Thomas Chauncey

Wenatchee Valley Hospital and Clinics
Wenatchee, Washington, 98801

Contact: 509-663-8711

Investigator: Lindsay Overton

Cheyenne Regional Medical Center
Cheyenne, Wyoming, 82001

Contact: 917-331-0613

Investigator: Natalie Alana Workman


Novartis Pharmaceuticals

Novartis Pharmaceuticals

Novartis Pharmaceuticals
EA Number: 
AML, FLT3, ITD, TKD, acute myeloid leukemia, induction, consolidation, continuation, EAP, ETP, midostaurin, PKC412
MeSH Terms: 
Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Midostaurin, Staurosporine
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