Compassionate Use Ponatinib

Study Description

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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.

Condition or disease	Intervention/treatment
Philadelphia Chromosome Positive (Ph+) Leukemias; Chronic Myeloid Leukemia	Drug: Ponatinib

Study Design

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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.

Interventions

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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

Eligibility Criteria

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Ages Eligible for Study:	18 Years and older (Adult, Senior)
Sexes Eligible for Study:	All

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

Contacts and Locations

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Locations

United States, Oregon
OHSU Knight Cancer Institute
Portland, Oregon, United States, 97239

Sponsors and Collaborators

OHSU Knight Cancer Institute

Ariad Pharmaceuticals

More Information

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Responsible Party:	OHSU Knight Cancer Institute
ClinicalTrials.gov Identifier:	NCT01549548 History of Changes
Other Study ID Numbers:	IRB00008238
First Posted:	March 9, 2012
Last Update Posted:	March 15, 2013
Last Verified:	March 2013

Keywords provided by OHSU Knight Cancer Institute:

CML

Additional relevant MeSH terms:

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