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
Ponatinib as Second Line Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib, Dasatinib or Nilotinib
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests how well ponatinib hydrochloride works as a second treatment for adults with chronic myeloid leukemia (CML), a blood cancer, in its chronic phase. It is for people whose first treatment did not work well enough or caused side effects they could not tolerate. Ponatinib may slow cancer growth by blocking a protein cancer cells need to grow.
This phase II trial studies how well ponatinib hydrochloride works as second line therapy in treating patients with chronic myeloid leukemia in chronic phase that has not responded to initial treatment (first line) with imatinib mesylate, dasatinib, or nilotinib or cannot tolerate imatinib mesylate, dasatinib, or nilotinib. Ponatinib hydrochloride may stop or control the growth of cancer cells by blocking a protein needed for cell growth.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be older than 18 and have chronic-phase CML with a gene change called Philadelphia chromosome or BCR-ABL.
- Have CML that did not respond well enough to one newer targeted drug: bosutinib, dasatinib, or nilotinib. Blood and gene tests must meet the study’s rules for treatment failure.
- Treatment failure may also include loss of an earlier response, new gene changes, or new chromosome changes during treatment.
- Be able to care for yourself and be up and moving for at least half of your waking hours.
- Have liver and kidney test results within the study’s limits.
- Sign a consent form showing you understand this is a research study.
- If you or your partner could become pregnant, use effective birth control during the study and for at least 3 months after the last dose. Women who could become pregnant need a negative pregnancy test before treatment.
- Stop other leukemia drugs at least 48 hours before study treatment, except hydroxyurea. Side effects from earlier treatment must improve to mild or better.
Who can't join
- Have taken more than one approved targeted CML drug, any unapproved targeted drug, or a BCR-ABL-targeted drug other than bosutinib, dasatinib, or nilotinib. The T315I gene change does not rule you out.
- Have serious heart or blood vessel disease, including past heart attack, stroke, mini-stroke, unstable chest pain, a blocked artery that damaged tissue, or a procedure to restore blood flow. Certain heart rhythm problems, recent severe heart failure, weak heart pumping, or a vein or lung blood clot within 6 months also rule you out.
- Have an active mental health condition that is not controlled, such as psychosis, major depression, or bipolar disorder.
- Have uncontrolled blood pressure above the study’s limits of 150 for the top number and 90 for the bottom number. People needing 3 or more blood pressure drugs need review by the lead study doctor.
- Have poorly controlled diabetes, with an HbA1c above 7.5%. This blood test measures average blood sugar.
- Be pregnant or breastfeeding.
- Have had pancreatitis, or swelling of the pancreas, within the past year, or have long-term pancreatitis.
- Have accelerated-phase or blast-phase CML, which are more advanced forms, or have ever had blast-phase CML. Extra chromosome changes alone do not rule you out.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * 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 Criteria: * 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. The definitions of excluded CML phases are as follows: 1. Blastic phase: presence of 30% blasts or more in the peripheral blood or bone marrow. 2. 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 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.
Study sites (1)
TX
- M D Anderson Cancer Center · Houston, TX 77030Elias Jabbour, MD · Study contact · 713-792-4764 · jcortes@mdanderson.orgElias Jabbour, MD · Principal investigatorMore trials in Houston