Expanded Access Program of Ponatinib
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01592136 |
Expanded Access Status :
Approved for marketing
First Posted : May 7, 2012
Last Update Posted : February 6, 2018
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Condition or disease | Intervention/treatment |
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Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL) | Drug: ponatinib |
Study Type : | Expanded Access |
See clinical trials of the intervention/treatment in this expanded access record. | |
Official Title: | Expanded Access Program of Ponatinib (AP24534) for Patients With Refractory Chronic Myeloid Leukemia or Ph+ Acute Lymphoblastic Leukemia |

- 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 consentOther Name: AP24534

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
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.
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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.
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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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01592136

Responsible Party: | Ariad Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01592136 History of Changes |
Other Study ID Numbers: |
AP24534-12-901 |
First Posted: | May 7, 2012 Key Record Dates |
Last Update Posted: | February 6, 2018 |
Last Verified: | February 2018 |
Ponatinib Tyrosine kinase inhibitor |
Leukemia Leukemia, Myeloid Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Lymphoid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Philadelphia Chromosome Neoplasms by Histologic Type Neoplasms Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases |
Myeloproliferative Disorders Bone Marrow Diseases Hematologic Diseases Translocation, Genetic Chromosome Aberrations Pathologic Processes Ponatinib Antineoplastic Agents Protein Kinase Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action |