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Expanded Access Program of Ponatinib

This treatment has been approved for sale to the public.
Information provided by (Responsible Party):
Ariad Pharmaceuticals Identifier:
First received: May 3, 2012
Last updated: March 12, 2013
Last verified: January 2013
This protocol will allow expanded access of ponatinib to patients ≥18 years with chronic myeloid leukemia (CML) any phase or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL) who have failed all available treatment options.

Condition Intervention
Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL) Drug: ponatinib

Study Type: Expanded Access     What is Expanded Access?
Official Title: Expanded Access Program of Ponatinib (AP24534) for Patients With Refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia

Resource links provided by NLM:

Further study details as provided by Ariad Pharmaceuticals:

Intervention Details:
    Drug: ponatinib
    Patients will receive ponatinib 45 mg orally as a single dose once daily with or without food. Each patient will receive daily ponatinib until disease progression, unacceptable toxicity, or withdrawal of consent
    Other Name: AP24534
Detailed Description:
This protocol will allow expanded access of ponatinib to patients ≥18 years with CML or Ph+ALL who have failed all available treatment options. Patients with chronic (CP) or accelerated phase (AP) CML must be previously treated with and resistant or intolerant to imatinib, dasatinib and nilotinib or develop the T315I mutation after any tyrosine kinase inhibitor (TKI) therapy. Patients with blast phase (BP) CML and Ph+ ALL must be previously treated with and resistant or intolerant to imatinib and dasatinib or develop the T315I mutation after any TKI therapy. No formal analysis will be performed on any data obtained. Safety information will be collected and adverse events will be tabulated for reporting purposes only.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All

Main Inclusion Criteria:

  1. CP-CML and AP-CML patients previously treated with and resistant or intolerant to imatinib, dasatinib and nilotinib or those who developed the T315I mutation after any TKI therapy. BP-CML and Ph+ ALL patients previously treated with and resistant or intolerant to imatinib and dasatinib or those who developed the T315I mutation after any TKI therapy.
  2. Patients must be ≥ 18 years old.
  3. Provide written informed consent.
  4. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
  5. Men and women of childbearing potential must agree to effective contraception from the time of signing informed consent through the Follow-up Visit, approximately 30 days after last dose of ponatinib.

Main Exclusion Criteria:

Patients are not eligible for participation in the study if they meet any of the following exclusion criteria:

  1. Are eligible for an ongoing and accessible clinical trial of ponatinib
  2. Have not adequately recovered from AEs due to agents previously administered
  3. Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
  4. Have previously been treated with ponatinib.
  5. Have significant or active cardiovascular disease, specifically including, but not restricted to:

    • Myocardial infarction within 3 months prior to first dose of ponatinib,
    • History of clinically significant atrial arrhythmia or any ventricular arrhythmia,
    • Unstable angina within 3 months prior to first dose of ponatinib,
    • Congestive heart failure within 3 months prior to first dose of ponatinib.
  6. Have abnormal QTcF (> 450 ms for males or > 470 ms for females)
  7. Have a significant bleeding disorder unrelated to CML or Ph+ ALL.
  8. Have a history of pancreatitis or alcohol abuse
  9. Have elevated amylase or lipase (> 1.5 x ULN for institution) at entry.
  10. Have inadequate hepatic function or any of the following:

    • Total bilirubin > 1.5 x ULN for institution at entry
    • Alanine aminotransferase and aspartate aminotransferase > 2.5 x ULN for institution at entry
    • Prothrombin time >1.5 x ULN for institution at entry
  11. Have inadequate renal function or serum creatinine > 2.5 x ULN for institution at entry
  12. Have uncontrolled hypertriglyceridemia or triglycerides > 450 mg/dL at entry.
  13. Have malabsorption syndrome or other gastrointestinal illness that could affect absorption of orally administered ponatinib.
  14. Women who are pregnant or lactating.
  15. Underwent major surgery within 14 days prior to the first dose of ponatinib.
  16. Have ongoing or active infection (including known history of human immunodeficiency virus [HIV], hepatitis B virus [HBV], or hepatitis C virus [HCV]).
  17. Suffer from any condition or illness that, in the opinion of the Investigator would compromise patient safety or interfere with the evaluation of the safety of the study drug.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01592136

  Hide Study Locations
United States, California
Moores UCSD Cancer Center, Site #165
La Jolla, California, United States, 92093
Southern California Permanente Medical Group, Site #161
San Marcos, California, United States, 92069
Kaiser Permanente Medical Center, Site #158
Vallejo, California, United States, 94589
United States, Connecticut
Smilow Cancer Hospital at Yale New Haven, Site #182
New Haven, Connecticut, United States, 06510
United States, Florida
Cancer Institute of Florida, Site #187
Altamonte Springs, Florida, United States, 32792
H. Lee Moffitt Cancer Center & Research Institute, Site #017
Tampa, Florida, United States, 33612
United States, Georgia
Emory University, Site # 058
Atlanta, Georgia, United States, 30322
United States, Illinois
University of Chicago Medical Center, Site #001
Chicago, Illinois, United States, 60637
United States, Indiana
Indiana Blood and Marrow Transplantation, Site #138
Indianapolis, Indiana, United States, 46237
United States, Kentucky
Norton Cancer Institute, Site #142
Louisville, Kentucky, United States, 40202
United States, Maryland
University of Maryland, Site #040
Baltimore, Maryland, United States, 21201
United States, Massachusetts
Tufts Medical Center, Site #141
Boston, Massachusetts, United States, 02111
Dana-Farber Cancer Institute, Site 008
Boston, Massachusetts, United States, 02215
University of Massachusetts Worcester, Site #152
Worcester, Massachusetts, United States, 01655
United States, Michigan
University of Michigan Health System, Site #011
Ann Arbor, Michigan, United States, 48109
Karmanos Cancer Institute, Site #034
Detroit, Michigan, United States, 48201
United States, Minnesota
Mayo Clinic, Site #044
Rochester, Minnesota, United States, 55905
United States, Missouri
Freeman Cancer Institute, Site #190
Joplin, Missouri, United States, 64804
Washington University School of Medicine, Site 007
St. Louis, Missouri, United States, 63110
United States, New Jersey
John Theurer Cancer Center at Hackensack University Medical Center, Site 128
Hackensack, New Jersey, United States, 07601
United States, New York
Roswell Park Cancer Institute, Site #029
Buffalo, New York, United States, 14263
Weill Cornell Medical College - New York Presbyterian Hospital, Site #006
New York, New York, United States, 10065
University of Rochester, Site 137
Rochester, New York, United States, 14627
United States, North Carolina
Duke University Medical Center, Site 003
Durham, North Carolina, United States, 27710
United States, Ohio
Jewish Hospital, Site #175
Cincinnati, Ohio, United States, 45236
United States, Oregon
Oregon Health & Science University (OHSU), Site 048
Portland, Oregon, United States, 97239
United States, Pennsylvania
Hospital of the University of Pennsylvania, Site #013
Philadelphia, Pennsylvania, United States, 19104
Jeanes Hospital of TUHS, Site #127
Philadelphia, Pennsylvania, United States, 19111
United States, South Carolina
Medical University of South Carolina, Site #148
Charleston, South Carolina, United States, 29425
United States, Tennessee
Tennesse Oncology, PLLC, Site # 076
Nashville, Tennessee, United States, 37203
United States, Texas
The University of Texas M.D. Anderson Cancer Center, Site #005
Houston, Texas, United States, 77030
United States, Utah
Huntsman Cancer Institute at the University of Utah, Site #043
Salt Lake City, Utah, United States, 84112
United States, Washington
Seattle Cancer Care Alliance, Site #100
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Ariad Pharmaceuticals
  More Information

Responsible Party: Ariad Pharmaceuticals Identifier: NCT01592136     History of Changes
Other Study ID Numbers: AP24534-12-901
Study First Received: May 3, 2012
Last Updated: March 12, 2013

Keywords provided by Ariad Pharmaceuticals:
Tyrosine kinase inhibitor

Additional relevant MeSH terms:
Leukemia, Myeloid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Philadelphia Chromosome
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Translocation, Genetic
Chromosome Aberrations
Pathologic Processes
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on August 23, 2017