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Ponatinib for Chronic Myeloid Leukemia Not Responding to Other Treatments

This study is testing ponatinib hydrochloride as a second treatment option for people with chronic myeloid leukemia (CML) in chronic phase. This is for patients whose CML has not responded to or who cannot tolerate previous treatments like imatinib mesylate, dasatinib, or nilotinib. Ponatinib hydrochloride works by blocking a protein that helps cancer cells grow. Researchers want to see how many participants achieve a major cytogenetic response (MCyR), which means a significant reduction in abnormal cells, within 6 months. They also want to understand the side effects of ponatinib hydrochloride and how long it takes for them to occur. You may be able to join if you are 18 or older, have Philadelphia chromosome (Ph)-positive or BCR-ABL-positive CML, and have not responded to a previous treatment.

Study design
This is an interventional study planning to enroll 50 participants. It is a Phase II trial, meaning it's focused on how well the treatment works and its safety.
What's involved
You would take ponatinib hydrochloride by mouth once a day, starting at 30 mg. You will also have blood draws for laboratory biomarker analysis and complete quality-of-life surveys.
Compensation
Not stated in the trial record.
Follow-up
Researchers will measure major cytogenetic response at 6 months and track side effects for up to 30 days after treatment ends. They will also assess responses at 3, 6, 12, 18, and 24 months.

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NCT01746836

Ponatinib Hydrochloride as Second Line Therapy in Treating Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib Mesylate, Dasatinib, or Nilotinib

Recruiting
PHASE2Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~50 participants
Updated 2026-03-09 on ClinicalTrials.gov
What's tested:Laboratory Biomarker AnalysisPonatinib HydrochlorideQuality-of-Life Assessment

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
MCyR at 6 months (MCyR6)
Measured over At 6 months
+1 more outcome measured

Conditions

Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
Philadelphia Chromosome Positive, BCR-ABL1 Positive Chronic Myelogenous Leukemia
Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive

Explore related trials

Chronic Myeloid Leukemia, BCR-ABL1 PositiveBCR/ABL1 Fusion GeneT315It(9;22)(q34.1;q11.2)

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Elias Jabbour, MD · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Elias Jabbour, MD
[email protected]
(713) 792-4764
Email the study team

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Eligibility criteria

Inclusion

Diagnosis of Philadelphia chromosome (Ph)-positive (by cytogenetics or fluorescent in situ hybridization \[FISH\]) or breakpoint cluster region (BCR)-ABL-positive (by polymerase chain reaction \[PCR\]) CML in chronic phase.
Participants should have demonstrated to have failure to therapy to one FDA-approved second-generation TKI (currently bosutinib, dasatinib, and nilotinib are approved as frontline therapy), defined as per European leukemiaNet (ELN)35 or National Comprehensive Cancer Network (NCCN) recommendations:
Less than complete hematologic response (CHR) at or beyond 3 months
No partial cytogenetic response at or beyond 3 months
BCR-ABL1 ≥ 10% at or beyond 3 months
BCR-ABL1 ≥ 1% at or beyond 6 months
Loss of CCyR or development of mutations or other clonal chromosomal abnormalities at any time during TKI treatment
Age \>18 years
ECOG performance of 0-2.
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 clearance (CrCL) ≥ 30 mL/min (Cockcroft-Gault formula).
Participants must sign an informed consent indicating they are aware of the investigational nature of this study, in keeping with the policies of the hospital.
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:
Prior to study enrollment, women of childbearing potential (WOCBP) must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy.
Postmenopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential OR women who are surgically sterile.
In addition, men enrolled on this study should understand the risks to any sexual partner of childbearing potential and should practice an effective method of birth control.
Women and men must continue birth control for the duration of the trial and at least 3 months after the last dose of study drug.
All WOCBP MUST have a negative pregnancy test prior to first receiving investigational product.
Participants should have discontinued therapy with bosutinib, dasatinib or nilotinib 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

Prior therapy with other BCR-ABL-targeted TKIs except bosutinib, dasatinib or nilotinib. Participants with T315I mutations are eligible and will be analyzed separately.
Active NYHA cardiac class 3-4 heart disease
Have clinically significant, uncontrolled, or active cardiovascular disease, specifically including, but not restricted to:
Any history of MI, unstable angina, cerebrovascular accident, or TIA
Any history of peripheral vascular infarction, including visceral infarction
Any revascularization procedure, including the placement of a stent
Congestive heart failure (CHF) (New York Heart Association \[NYHA\] class III or IV) within 6 months prior to enrollment, or left ventricular ejection fraction (LVEF) less than lower limit of normal, per local institutional standards, within 6 months prior to enrollment
History of clinically significant (as determined by the treating physician) atrial arrhythmia or any history of ventricular arrhythmia
Venous thromboembolism, including deep venous thrombosis or pulmonary embolism, within 6 months prior to enrollment
Participants with active, uncontrolled psychiatric disorders including: psychosis, major depression, and bipolar disorders.
Have uncontrolled hypertension (i.e., \>150 and \>90 for SBP and DBP, respectively). Participants with hypertension should be under treatment at study entry to ensure blood pressure control. Those requiring 3 or more antihypertensive medications should be discussed with the PI.
Have poorly controlled diabetes defined as HbA1c values of \> 7.5%. Participants with preexisting, well-controlled, diabetes are not excluded.
Pregnant or breast-feeding women are excluded.
Participants with history of pancreatitis within 1 year of study or history of chronic pancreatitis.
Participants in accelerated or blast phase, or patients who have ever been documented to be in blast phase CML, are excluded.
Peripheral or marrow blasts 15% or more
Peripheral or marrow basophils 20% or more
Thrombocytopenia \< 100 x 109/L unrelated to therapy
Documented extramedullary blastic disease outside liver or spleen 3. 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. However, participants with clonal evolution as the only criterion of accelerated phase have a significantly better prognosis. Thus, participants with clonal evolution and no other criteria for accelerated phase will be eligible for this study, but analyzed separately.
Participants who have received more than one FDA-approved TKI for CML, or any investigational, non-FDA approved TKI.

What this trial measures

  • MCyR at 6 months (MCyR6)At 6 months

    The method of Kaplan and Meier will be used to estimate the unadjusted distribution of duration of MCyR. An appropriate time-to-event regression model will be fit to the event time data to assess the effects of patient covariates on the event time variable, with the particular model determined by preliminary goodness-of-fit analyses. The distribution of MCyR6 will be tabulated and effects of baseline patient covariates on this variable will be assessed by logistic regression.

  • Time-to-toxicity defined as any grade 3 or 4 drug-related adverse event that is not responsive to standard therapeutic management and requires permanent treatment discontinuationUp to 30 days post-treatment

    Time-to-toxicity will be monitored using the Bayesian method of Thall, et al. The method of Kaplan and Meier will be used to estimate the unadjusted distribution of time to toxicity. An appropriate time-to-event regression model will be fit to the event time data to assess the effects of patient covariates on the event time variable, with the particular model determined by preliminary goodness-of-fit analyses.