Compassionate Use Ponatinib

Chronic Myeloid Leukemia Clinical Trial

Official title:

Treatment Plan for the Compassionate Use of Ponatinib (AP24534) in Patients With Imatinib-, Dasatinib-, and Nilotinib- Resistant/Intolerant Philadelphia Chromosome Positive Leukemias.

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT01549548
• Other study ID #: IRB00008238
• Status: No longer available
• Phase: N/A
• First received: March 6, 2012
• Last updated: March 13, 2013

Study information

• Verified date: March 2013
• Source: OHSU Knight Cancer Institute
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

N/A

Related Conditions & MeSH terms

• Abnormal Karyotype
• Chronic Myeloid Leukemia
• Leukemia
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Philadelphia Chromosome
• Philadelphia Chromosome Positive (Ph+) Leukemias

Intervention

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.

Locations

Country	Name	City	State
United States	OHSU Knight Cancer Institute	Portland	Oregon

Sponsors (2)

Lead Sponsor	Collaborator
OHSU Knight Cancer Institute	Ariad Pharmaceuticals

Country where clinical trial is conducted

United States,