Expanded Access Program of Ponatinib

Sponsor

Ariad Pharmaceuticals (Industry)

Overall Status

Approved for marketing

CT.gov ID

NCT01592136

Collaborator

(none)

Locations

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)	Drug: ponatinib

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.

Study Design

Study Type:

Expanded Access

Official Title:

Expanded Access Program of Ponatinib (AP24534) for Patients With Refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Main Inclusion Criteria:

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.
Patients must be ≥ 18 years old.
Provide written informed consent.
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
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:

Are eligible for an ongoing and accessible clinical trial of ponatinib
Have not adequately recovered from AEs due to agents previously administered
Require concurrent treatment with immunosuppressive agents, other than corticosteroids prescribed for a short course of therapy.
Have previously been treated with ponatinib.
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.

Have abnormal QTcF (> 450 ms for males or > 470 ms for females)
Have a significant bleeding disorder unrelated to CML or Ph+ ALL.
Have a history of pancreatitis or alcohol abuse
Have elevated amylase or lipase (> 1.5 x ULN for institution) at entry.
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

Have inadequate renal function or serum creatinine > 2.5 x ULN for institution at entry
Have uncontrolled hypertriglyceridemia or triglycerides > 450 mg/dL at entry.
Have malabsorption syndrome or other gastrointestinal illness that could affect absorption of orally administered ponatinib.
Women who are pregnant or lactating.
Underwent major surgery within 14 days prior to the first dose of ponatinib.
Have ongoing or active infection (including known history of human immunodeficiency virus [HIV], hepatitis B virus [HBV], or hepatitis C virus [HCV]).
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

Locations

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