Compassionate Use Ponatinib
![]() |
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: NCT01549548 |
Expanded Access Status :
No longer available
First Posted : March 9, 2012
Last Update Posted : March 15, 2013
|
- Study Details
- Tabular View
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment |
---|---|
Philadelphia Chromosome Positive (Ph+) Leukemias Chronic Myeloid Leukemia | Drug: Ponatinib |
Study Type : | Expanded Access |
Official Title: | Treatment Plan for the Compassionate Use of Ponatinib (AP24534) in Patients With Imatinib-, Dasatinib-, and Nilotinib- Resistant/Intolerant Philadelphia Chromosome Positive Leukemias. |

- Drug: Ponatinib
Patients will receive Ponatinib 45 mg by mouth as a single daily dose on an empty stomach (no food 2 hours prior to and after dosing) on day 1 and continuous once-daily dosing everyday thereafter. Each patient will receive daily Ponatinib until disease progression, unacceptable toxicity, or withdrawal of consent.Other Name: AP24534

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, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Inclusion Criteria:
- Signed and dated informed consent
- Cytogenetic or PCR-based diagnosis of any phase of Ph+ acute lymphoblastic leukemia (ALL) and documented resistance or intolerance to imatinib and a second TKI (nilotinib, dasatinib, bosutinib)
- Eastern Cooperative Oncology Group(ECOG) performance status of 0-2
- Washout from prior anti-proliferative or anti-leukemia treatment: 3 days for hydroxyurea/anagrelide and tyrosine kinase inhibitors
- < Grade 2 or baseline recovery from prior therapy related toxicities (except alopecia)
- At least 3 months post allogeneic stem cell transplantation
- Able to take oral capsules reliably
- AST/ALT less than or equal to 2.5 times ULN, or less than 5 times ULN if attributable to involvement of leukemia
- No active clinical or radiographic pancreatitis
- At least 18 years of age
- Willingness of male and female subjects to use reliable methods of birth control (when applicable)
Exclusion Criteria:
- Subjects with Philadelphia Chromosome and BCR-ABL-negative chronic myeloid leukemia (CML)
- Major surgery or radiotherapy within 7 days before the first dose of Ponatinib (recovery from any previous surgery should be complete before day 1)
- Clinically significant active/uncompensated or uncontrolled cardiac disease (active congestive heart failure; uncontrolled angina or hypertension; myocardial infarction in the past 3 months; clinically significant untreated ventricular arrhythmia; diagnosed or suspected congenital or acquired prolonged QT syndrome; unexplained syncope; history of prolonged QTc)
- Prolonged QTc (> 0.48 sec)
- Pregnant or breastfeeding women
- Evidence of serious active infection, or significant medical or psychiatric illness
- Known seropositivity to HIV, or current acute or chronic Hepatitis B or Hepatitis C (antigen positive), cirrhosis, or clinically significant abnormal lab finding that would, in the investigator's judgment, make the subject inappropriate for this study
- Prior resistance to Ponatinib

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): NCT01549548
United States, Oregon | |
OHSU Knight Cancer Institute | |
Portland, Oregon, United States, 97239 |
Responsible Party: | OHSU Knight Cancer Institute |
ClinicalTrials.gov Identifier: | NCT01549548 |
Other Study ID Numbers: |
IRB00008238 |
First Posted: | March 9, 2012 Key Record Dates |
Last Update Posted: | March 15, 2013 |
Last Verified: | March 2013 |
CML |
Leukemia Leukemia, Myelogenous, Chronic, BCR-ABL Positive Philadelphia Chromosome Neoplasms by Histologic Type Neoplasms Leukemia, Myeloid 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 |