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Study Summary
No. 2012-0669:.......Leukemia......Jorge Cortes......Leukemia
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Study Summary Title
Study Summary
Number:
2012-0669
Study Title:Ponatinib as Second Line Therapy for Patients with Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib
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Physician New Patient Referral
Name:Jorge CortesPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:LeukemiaReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-794-5783
Contact us about clinical trials
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General Information
Disease Group:LeukemiaSupported By:Ariad Pharmaceuticals
Phase of Study:Phase IIReturn
Visit:
Treatment
Agents:
PonatinibHome Care:
Treatment Loc:Only at MDACC
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of this clinical research study is to learn if ponatinib can help to
control CML in chronic phase. The safety of this drug will also be studied.

Ponatinib is designed to block the function of BCR-ABL, which is the abnormal
protein responsible for causing CML. This may cause the cancer cells to die.
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Study Objectives / Outcomes
Primary:
  • To estimate the proportion of patients with imatinib-resistant or intolerant, chronic phase CML (CP-CML) attaining major cytogenetic response (MCyR) at 6 months of treatment with second line ponatinib therapy.
· To estimate the time to toxicity related to ponatinib for patients with imatinib-intolerant or imatinib-resistant CP-CML.

Secondary:
  • To estimate the proportion of patients achieving a MCyR, complete cytogenetic response (CCyR), major molecular response (MMR) and complete molecular response (CMR) at 3, 6, 12, 18 and 24 months of treatment with ponatinib after imatinib failure (by resistance or intolerance to imatinib).
  • To estimate the time to CCyR, MMR, MCyR and CMR for patients treated with ponatinib as second line therapy for CP-CML
  • To evaluate the durations of hematologic, cytogenetic and molecular response to ponatinib after imatinib failure.
  • To define the time to progression and overall survival for patients with CML in chronic phase treated with ponatinib after imatinib failure.
  • To evaluate the toxicity profile of ponatinib in patients with CML in chronic phase after imatinib failure.
  • To evaluate the probability of developing ABL mutations for patients with CML in chronic phase treated with ponatinib after imatinib failure.
  • To analyze differences in response rates and in prognosis according to pre-treatment mutations and patient characteristics.
  • To investigate mechanisms of resistance in patients who develop resistance to ponatinib used as second line therapy for CP-CML.
  • To evaluate symptom burden in patients with CP-CML receiving ponatinib.

Exploratory:
  • To investigate the presence of miRNA that may be predictive of outcome.
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Study Status Information
Study Activation / Registration Date:
IRB Review and Approval Date:01/17/2013
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Open
Projected Accrual:N/A
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Diagnosis of Ph-positive (by cytogenetics or FISH) or BCR-ABL-positive (by PCR) CML in chronic phase.

2) Patients should have demonstrated to have failure to therapy to one FDA-approved TKI (currently imatinib, dastinib, and nilotinib are approved as frontline therapy), defined as per European leukemiaNet (ELN) recommendations: 1. Less than complete hematologic response (CHR) at or beyond 3 months; 2. No cytogenetic response at or beyond 6 months; 3. Less than PCyR (Ph+ >35%) at or beyond 12 months; 4. Less than CCyR at or beyond 18 months; 5. Loss of response or development of mutations or other clonal chromosomal abnormalities at any time during imatinib treatment; 6. Intolerance to imatinib defined as grade 3 or 4 toxicity, or persistent grade 2 toxicity despite optimal management including dose adjustment, or in a patient where dose reductions are considered to be not in the patients best interest to obtain or maintain an adequate response. Intolerant patients should not have achieved or have lost major cytogenetic response at the time of enrollment.

3) Age >/= 18 years.

4) ECOG performance of 0-2.

5) Adequate end organ function, defined as the following: total bilirubin </= 1.5x ULN (unless due to Gilbert syndrome, in which case it should be </= 3.0x ULN), SGPT </= 2.5x ULN, creatinine </=1.5x ULN.

6) Patients must sign an informed consent indicating they are aware of the investigational nature of this study, in keeping with the policies of the hospital.

7) Reliable telephone access to receive calls from an interactive voice response system (IVR) (only applicable to patients who will participate in optional symptom burden assessment).

8) Women of pregnancy potential must practice an effective method of birth control during the course of the study, in a manner such that risk of failure is minimized: 1. Prior to study enrollment, women of childbearing potential (WOCBP) must be advised of the importance of avoiding pregnancy during trial participation & the potential risk factors for an unintentional pregnancy; 2. Postmenopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential; 3. In addition, men enrolled on this study should understand the risks to any sexual partner of childbearing potential & should practice an effective method of birth control; 4. Women & men must continue birth control for the duration of the trial & at least 3 months after the last dose of study drug;

9) **continued from above: 5. All WOCBP MUST have a negative pregnancy test prior to first receiving investigational product. If the pregnancy test is positive, the patient must not receive investigational product & must not be enrolled in the study.

10) Patients should have discontinued therapy with imatinib or other anti-leukemia therapy (except hydroxyurea), at least 48 hours prior to start of study therapy and recovered from any toxicity due to these therapies to at least grade 1. The use of hydroxyurea is allowed immediately prior to study entry.

Exclusion Criteria:1) Prior therapy with other BCR-ABL-targeted TKIs except imatinib (e.g., dasatinib, nilotinib, bosutinib).

2) NYHA cardiac class 3-4 heart disease.

3) Cardiac Symptoms: Patients meeting the following criteria are not eligible: Unstable angina or myocardial infarction within 3 months; Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or Torsades de pointes); Prolonged QTc interval on pre-entry electrocardiogram (> 470 msec) on both the Fridericia and Bazett's correction; Congestive heart failure within 3 months prior to first dose of ponatinib.

4) Patients with active, uncontrolled psychiatric disorders including: psychosis, major depression, and bipolar disorders.

5) Pregnant or breast-feeding women are excluded.

6) Patients with history of pancreatitis.

7) Patients in accelerated or blast phase, as patients who have ever been documented to be in blast phase CML, are excluded. The definitions of CML phases are as follows: a. Early chronic phase: time from diagnosis to therapy </= 12 months; b. Late chronic phase: time from diagnosis to therapy > 12 months; c. Blastic phase: presence of 30% blasts or more in the peripheral blood or bone marrow; d. Accelerated phase CML: presence of any of the following features: Peripheral or marrow blasts 15% or more; Peripheral or marrow basophils 20% or more; Thrombocytopenia < 100 x 10(9)/L unrelated to therapy; Documented extramedullary blastic disease outside liver or spleen; e. Clonal evolution defined as the presence of additional chromosomal abnormalities other than the Ph chromosome has been historically been included as a criterion for accelerated phase.

8) **continued from above: However, patients with clonal evolution as the only criterion of accelerated phase have a significantly better prognosis. Thus, patients with clonal evolution and no other criteria for accelerated phase will be eligible for this study, but analyzed separately.

9) Patients who have received more than one FDA-approved TKI for CML, or any investigational, non-FDA approved TKI.

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Links
Registration Number: NCT01746836
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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