Compassionate Use Ponatinib

Expanded access is no longer available for this treatment.
Ariad Pharmaceuticals
Information provided by (Responsible Party):
OHSU Knight Cancer Institute Identifier:
First received: March 6, 2012
Last updated: March 13, 2013
Last verified: March 2013
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.

Condition Intervention
Philadelphia Chromosome Positive (Ph+) Leukemias
Chronic Myeloid Leukemia
Drug: Ponatinib

Study Type: Expanded Access     What is 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.

Resource links provided by NLM:

Further study details as provided by OHSU Knight Cancer Institute:

Intervention Details:
    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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01549548

United States, Oregon
OHSU Knight Cancer Institute
Portland, Oregon, United States, 97239
Sponsors and Collaborators
OHSU Knight Cancer Institute
Ariad Pharmaceuticals
  More Information

No publications provided

Responsible Party: OHSU Knight Cancer Institute Identifier: NCT01549548     History of Changes
Other Study ID Numbers: IRB00008238
Study First Received: March 6, 2012
Last Updated: March 13, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by OHSU Knight Cancer Institute:

Additional relevant MeSH terms:
Abnormal Karyotype
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Philadelphia Chromosome
Bone Marrow Diseases
Chromosome Aberrations
Hematologic Diseases
Leukemia, Myeloid
Myeloproliferative Disorders
Neoplasms by Histologic Type
Pathologic Processes
Translocation, Genetic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protein Kinase Inhibitors
Therapeutic Uses processed this record on December 01, 2015