Clinical trial · Interventional
Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Phase I Study of Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
NCT01895842CI-TRIAL-00086607completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with low or intermediate-1 risk MDS. The safety of this drug will also be studied, and whether it can help to control the disease.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Ruxolitinib | Drug | Ruxolitinib | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Ruxolitinib
- description
- 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.
- interventionNames
- Drug: Ruxolitinib
Primary outcomes (1)
- measure
- Maximum Tolerated Dose (MTD) of Ruxolitinib
- timeFrame
- 28 days
- description
- Maximum tolerated dose (MTD) is the maximum dose at which \<33% of patients experience a dose limiting toxicity (DLT). Dose limiting toxicity defined as any grade 3 or higher non-hematologic toxicity.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: 1. Patients with previously treated low or intermediate-1 risk MDS by the IPSS classification (this is defined in table 1) 2. 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. 3. Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC. 4. Age \>/= 18 years old 5. 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. 6. 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 7. Patients with Childbearing potential must agree to use appropriate forms of birth control Exclusion Criteria: 1. Previously untreated low or intermediate-1 risk MDS patients because there are approved therapies for these patients. 2. Uncontrolled undercurrent illness that in the opinion of the treating physician would contraindicate the use of the drug. 3. 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 4. 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. 5. Women who are pregnant or lactating. 6. Patients with a white blood cell count of more than 30x10\^3 K/uL will not be eligible for this study. 7. Patients that have received prior allogeneic stem cell transplantation are excluded from this study.
References
Publications (1)
- DERIVEDAbaza Y, Hidalgo-Lopez JE, Verstovsek S, Jabbour E, Ravandi F, Borthakur G, Estrov Z, Alvarado Y, Burger J, Schneider H, Soltysiak KA, Wei Y, Kantarjian HM, Bueso-Ramos CE, Garcia-Manero G. Phase I study of ruxolitinib in previously treated patients with low or intermediate-1 risk myelodysplastic syndrome with evidence of NF-kB activation. Leuk Res. 2018 Oct;73:78-85. doi: 10.1016/j.leukres.2018.09.004. Epub 2018 Sep 14. PMID 30245189