ABL001 for the Treatment of Chronic Myeloid Leukemia in Patients Who Are on Therapy With Tyrosine Kinase Inhibitor

Inclusion Criteria

• •Patients with a diagnosis of Philadelphia chromosome (Ph)- or BCR-ABL1-positive CML (as determined by cytogenetics, fluorescence in situ hybridization \[FISH\], or polymerase chain reaction \[PCR\])
• •Patients should be receiving therapy with nilotinib or dasatinib, whether as initial therapy or after prior TKI, at a dose equal or lower than the standard dose
• •Patients must have received TKI therapy for at least 24 months and not have required dose reductions, escalations, discontinuation or re-initiation after discontinuation of TKI in the last 6 months
• •Patients must be in CCyR (by conventional karyotype or FISH, or BCR-ABL/ABL =\< 1% IS if no cytogenetic analysis available within 3 months)
• •Patients must have detectable BCR-ABL1 transcript levels meeting at least one of the following criteria: a. Patient has never achieved a MMR after 18 months of therapy with their current TKI, or b. Patient has not achieved MR4.5 after 36 months of therapy with their current TKI, or c. Patient has lost MMR or MR4.5 confirmed in at least two consecutive analyses separated by at least 1 month, or d. BCR-ABL1 transcript levels have reached a plateau defined as a ratio that is stable in a molecular response category (i.e., MMR, MR4 or MR4.5) in the last at least 12 months, with at least 3 values obtained during this period
• •Patients must not have had a known continuous interruption of TKI therapy of greater than 14 days or for a total of 6 weeks in the 6 months prior to enrollment, unless the interruption was for an accident, unrelated hospitalization or surgical procedure, or for a treatment-free remission attempt that was unsuccessful and required re-initiation of therapy
• •Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
• •Creatinine =\<1.5 x institutional upper limit of normal
• •Amylase and lipase values =\< 3.0 x institutional upper limit of normal
• •Alkaline phosphatase =\< 2.5 x institutional upper limit of normal unless considered to be not of hepatic origin
• •Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3 x institutional upper limit of normal
• •Total bilirubin =\< 1.5 x institutional upper limit of normal (=\< 3 x upper limit of normal in patients with known Gilbert's syndrome)
• •The effects of ABL001 on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Women of child-bearing potential must agree to use highly effective methods of contraception during dosing and for 30 days after study treatment. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Allowable methods of birth control: Total abstinence (when this is in line with the preferred and usual lifestyle of the subject). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before the start of study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment. Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject. Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%), for example hormone vaginal ring or transdermal hormone contraception. Sexually active males must use a condom during intercourse while taking the drug and for 30 days after stopping treatment and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid