Ariad Pharmaceuticals

Expanded Access Program of Ponatinib

Official Title: 
Expanded Access Program of Ponatinib (AP24534) for Patients With Refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia
Brief Summary: 
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.
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.

Approved for marketing
Chronic Myeloid Leukemia (CML), Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)

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

Eligibility Criteria: 

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.

Eligibility Gender: 
All
Eligibility Age: 
Minimum: 18 Years
Countries: 
United States
Locations: 

Moores UCSD Cancer Center, Site #165
La Jolla, California, 92093

Southern California Permanente Medical Group, Site #161
San Marcos, California, 92069

Kaiser Permanente Medical Center, Site #158
Vallejo, California, 94589

Smilow Cancer Hospital at Yale New Haven, Site #182
New Haven, Connecticut, 06510

Cancer Institute of Florida, Site #187
Altamonte Springs, Florida, 32792

H. Lee Moffitt Cancer Center & Research Institute, Site #017
Tampa, Florida, 33612

Emory University, Site # 058
Atlanta, Georgia, 30322

University of Chicago Medical Center, Site #001
Chicago, Illinois, 60637

Indiana Blood and Marrow Transplantation, Site #138
Indianapolis, Indiana, 46237

Norton Cancer Institute, Site #142
Louisville, Kentucky, 40202

University of Maryland, Site #040
Baltimore, Maryland, 21201

Tufts Medical Center, Site #141
Boston, Massachusetts, 02111

Dana-Farber Cancer Institute, Site 008
Boston, Massachusetts, 02215

University of Massachusetts Worcester, Site #152
Worcester, Massachusetts, 01655

University of Michigan Health System, Site #011
Ann Arbor, Michigan, 48109

Karmanos Cancer Institute, Site #034
Detroit, Michigan, 48201

Mayo Clinic, Site #044
Rochester, Minnesota, 55905

Freeman Cancer Institute, Site #190
Joplin, Missouri, 64804

Washington University School of Medicine, Site 007
Saint Louis, Missouri, 63110

John Theurer Cancer Center at Hackensack University Medical Center, Site 128
Hackensack, New Jersey, 07601

Roswell Park Cancer Institute, Site #029
Buffalo, New York, 14263

Weill Cornell Medical College - New York Presbyterian Hospital, Site #006
New York, New York, 10065

University of Rochester, Site 137
Rochester, New York, 14627

Duke University Medical Center, Site 003
Durham, North Carolina, 27710

Jewish Hospital, Site #175
Cincinnati, Ohio, 45236

Oregon Health & Science University (OHSU), Site 048
Portland, Oregon, 97239

Hospital of the University of Pennsylvania, Site #013
Philadelphia, Pennsylvania, 19104

Jeanes Hospital of TUHS, Site #127
Philadelphia, Pennsylvania, 19111

Medical University of South Carolina, Site #148
Charleston, South Carolina, 29425

Tennesse Oncology, PLLC, Site # 076
Nashville, Tennessee, 37203

The University of Texas M.D. Anderson Cancer Center, Site #005
Houston, Texas, 77030

Huntsman Cancer Institute at the University of Utah, Site #043
Salt Lake City, Utah, 84112

Seattle Cancer Care Alliance, Site #100
Seattle, Washington, 98109

Ariad Pharmaceuticals
EA Number: 
Keywords: 
Ponatinib, Tyrosine kinase inhibitor
MeSH Terms: 
Leukemia, Leukemia, Myeloid, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Lymphoid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Philadelphia Chromosome, Ponatinib
Disclaimer:
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.