Expanded Access Program of Ponatinib

Chronic Myeloid Leukemia (CML) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT01592136
• Other study ID #: AP24534-12-901
• Status: Approved for marketing
• Phase: N/A
• First received: May 3, 2012
• Last updated: February 5, 2018

Study information

• Verified date: February 2018
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Chronic Myeloid Leukemia (CML)
• Leukemia
• Leukemia, Lymphoid
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid
• Philadelphia Chromosome
• Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
• Precursor Cell Lymphoblastic Leukemia-Lymphoma

Intervention

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

Locations

Country	Name	City	State
United States	Cancer Institute of Florida, Site #187	Altamonte Springs	Florida
United States	University of Michigan Health System, Site #011	Ann Arbor	Michigan
United States	Emory University, Site # 058	Atlanta	Georgia
United States	University of Maryland, Site #040	Baltimore	Maryland
United States	Dana-Farber Cancer Institute, Site 008	Boston	Massachusetts
United States	Tufts Medical Center, Site #141	Boston	Massachusetts
United States	Roswell Park Cancer Institute, Site #029	Buffalo	New York
United States	Medical University of South Carolina, Site #148	Charleston	South Carolina
United States	University of Chicago Medical Center, Site #001	Chicago	Illinois
United States	Jewish Hospital, Site #175	Cincinnati	Ohio
United States	Karmanos Cancer Institute, Site #034	Detroit	Michigan
United States	Duke University Medical Center, Site 003	Durham	North Carolina
United States	John Theurer Cancer Center at Hackensack University Medical Center, Site 128	Hackensack	New Jersey
United States	The University of Texas M.D. Anderson Cancer Center, Site #005	Houston	Texas
United States	Indiana Blood and Marrow Transplantation, Site #138	Indianapolis	Indiana
United States	Freeman Cancer Institute, Site #190	Joplin	Missouri
United States	Moores UCSD Cancer Center, Site #165	La Jolla	California
United States	Norton Cancer Institute, Site #142	Louisville	Kentucky
United States	Tennesse Oncology, PLLC, Site # 076	Nashville	Tennessee
United States	Smilow Cancer Hospital at Yale New Haven, Site #182	New Haven	Connecticut
United States	Weill Cornell Medical College - New York Presbyterian Hospital, Site #006	New York	New York
United States	Hospital of the University of Pennsylvania, Site #013	Philadelphia	Pennsylvania
United States	Jeanes Hospital of TUHS, Site #127	Philadelphia	Pennsylvania
United States	Oregon Health & Science University (OHSU), Site 048	Portland	Oregon
United States	Mayo Clinic, Site #044	Rochester	Minnesota
United States	University of Rochester, Site 137	Rochester	New York
United States	Washington University School of Medicine, Site 007	Saint Louis	Missouri
United States	Huntsman Cancer Institute at the University of Utah, Site #043	Salt Lake City	Utah
United States	Southern California Permanente Medical Group, Site #161	San Marcos	California
United States	Seattle Cancer Care Alliance, Site #100	Seattle	Washington
United States	H. Lee Moffitt Cancer Center & Research Institute, Site #017	Tampa	Florida
United States	Kaiser Permanente Medical Center, Site #158	Vallejo	California
United States	University of Massachusetts Worcester, Site #152	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Ariad Pharmaceuticals

Country where clinical trial is conducted

United States,