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Compassionate Use Ponatinib

Primary Purpose

Philadelphia Chromosome Positive (Ph+) Leukemias, Chronic Myeloid Leukemia

Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Ponatinib
Sponsored by
OHSU Knight Cancer Institute
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Philadelphia Chromosome Positive (Ph+) Leukemias focused on measuring CML

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

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

Sites / Locations

  • OHSU Knight Cancer Institute

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
March 6, 2012
Last Updated
March 13, 2013
Sponsor
OHSU Knight Cancer Institute
Collaborators
Ariad Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT01549548
Brief Title
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

2. Study Status

Record Verification Date
March 2013
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
OHSU Knight Cancer Institute
Collaborators
Ariad Pharmaceuticals

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Philadelphia Chromosome Positive (Ph+) Leukemias, Chronic Myeloid Leukemia
Keywords
CML

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Ponatinib
Other Intervention Name(s)
AP24534
Intervention Description
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.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
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
Facility Information:
Facility Name
OHSU Knight Cancer Institute
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States

12. IPD Sharing Statement

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Compassionate Use Ponatinib

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