Clinical trial NCT01549548: Compassionate Use Ponatinib
Official title: Treatment Plan for the Compassionate Use of Ponatinib (AP24534) in Patients With Imatinib-, Dasatinib-, and Nilotinib- Resistant/Intolerant Philadelphia Chromosome Positive Leukemias.
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Philadelphia Chromosome Positive (Ph+) Leukemias
Chronic Myeloid Leukemia
Keywords: CML
Lead sponsor: OHSU Knight Cancer Institute
Sponsor class: OTHER
Collaborators: name: Ariad Pharmaceuticals
First posted: 2012-03-09
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

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.


Eligibility criteria: 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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Portland
country: United States
facility: OHSU Knight Cancer Institute
state: Oregon
Study officials:
References: