Chronic Myeloid Leukemia, Philadelphia Chromosome Positive (Ph+) Leukemias
Conditions
Keywords
CML
Brief summary
The main purpose of this protocol is to provide expanded access to the study drug (Ponatinib/AP24534) for people with imatinib-, dasatinib-, and nilotinib- resistant/intolerant Philadelphia Chromosome Positive (Ph+) Leukemias. The other purpose of this protocol is to monitor the safety of the study drug in people with Ph+ Leukemias that have not responded to prior treatment.
Interventions
DRUGPonatinib
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.
Sponsors
Ariad Pharmaceuticals
OHSU Knight Cancer Institute
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Countries
United States
Outcome results
None listed