Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Leukemia
About this trial
This is an interventional treatment trial for Leukemia focused on measuring Leukemia, Myelodysplastic syndrome, MDS, Low or intermediate-1 risk, Maximum tolerated dose, MTD, Ruxolitinib, Jakafi, INCB018424, INC424
Eligibility Criteria
Inclusion Criteria:
- Patients with previously treated low or intermediate-1 risk MDS by the IPSS classification (this is defined in table 1)
- Patients must have one of the following: elevated b2-microglobulin levels (defined as 2 times compared to normal), carry a JAK2 mutation, or presence of phosphorylated p65 NF-kB component in at least 5% of bone marrow cells.
- Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC.
- Age >/= 18 years old
- Prior therapy with growth factor support, lenalidomide, 5-azacytidine, decitabine or other investigational agents are allowed. A four week wash out period will be required before receiving study medication.
- Patients must have the following non-hematologic values Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) </= 2.5 x Upper Limit of Normal (ULN) or </= 5.0 x ULN if hepatic involvement is present; Serum bilirubin </= 2 x ULN; Serum creatinine </= 2 x ULN or 24-hour creatinine clearance >/= 60 ml/min
- Patients with Childbearing potential must agree to use appropriate forms of birth control
Exclusion Criteria:
- Previously untreated low or intermediate-1 risk MDS patients because there are approved therapies for these patients.
- Uncontrolled undercurrent illness that in the opinion of the treating physician would contraindicate the use of the drug.
- Patients with active infections including uncontrolled HIV infection, active hepatitis B, C, or any other symptomatic systemic infection requiring active therapy will be excluded from study
- Patients receiving potent CYP3A4 (such as but not limited to boceprevir, clarithromycin, conivaptan, grapefruit juice, indinavir, itraconazole, ketoconazole, lopinavir/ritonavir, mibefradil, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, voriconazole) inhibitors will be excluded from the study.
- Women who are pregnant or lactating.
- Patients with a white blood cell count of more than 30x10^3 K/uL will not be eligible for this study.
- Patients that have received prior allogeneic stem cell transplantation are excluded from this study.
Sites / Locations
- University of Texas MD Anderson Cancer Center
Arms of the Study
Arm 1
Experimental
Ruxolitinib
Part 1: Dose of ruxolitinib received will depend when patient joined study. The first group of patients receive the lowest dose of ruxolitinib. Starting dose level for Part 1, 5 mg by mouth twice a day for a 28 day cycle. The second group of patients receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of patients receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of patients take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. If patients enrolled in Part 2, they will receive ruxolitinib at the highest dose that was tolerated in Part 1.