A Phase 1/2 interventional study of Ruxolitinib in Leukemia, sponsored by M.D. Anderson Cancer Center. Terminated at 1 site in United States. Open to participants aged 14 Years and older. Per ClinicalTrials.gov, last updated 2025-06-08.
Sponsored by M.D. Anderson Cancer Center · Phase 1/2, Interventional, and Treatment
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with acute leukemia and to learn if the study drug can help control the disease. The safety of the drug will also be studied.
The Study Drug:
Ruxolitinib is designed to block a gene mutation that may be important in cancer cell growth and survival. By blocking the gene mutation, this may cause the cancer cells to die.
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 30 participants will be enrolled in the Phase I portion of the study, and up to 136 participants will be enrolled in Phase II.
If you are enrolled in the Phase I portion, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose level of ruxolitinib. Each new group will receive a higher dose of ruxolitinib than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of ruxolitinib is found.
If you are enrolled in the Phase II portion, you will receive ruxolitinib at the highest dose that was tolerated in the Phase I portion or at a lower dose.
Study Drug Administration:
You will take ruxolitinib tablet(s) by mouth 2 times a day on Days 1-28 of each 28-day study cycle.
You will be asked to keep a diary to record the doses taken. You will be asked to bring your diary and any unused drug to your next visit.
Study Visits:
On Days 1, 7, 14, and 21 of Cycle 1:
On Day 1 of Cycle 2:
During Cycles 2 and beyond, blood (about 2 teaspoons) will be drawn for routine tests at least every 1-2 weeks. This blood may be drawn at a clinic close to your home.
On Day 1 of Cycles 3, 6, 9, and beyond:
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse or intolerable side effects occur.
Your participation on the study will be over once you have completed the end-of-study visit and the follow-up call.
End-of-Study Visit:
After your last dose of study drug, you will have an end-of-study visit. At this visit, the following tests and procedures will be performed:
Follow-Up:
About one month after your end-of-study visit, the study staff will call and ask about any side effects you may be having. This call should last about 5 minutes.
This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis. Its use to treat acute leukemia is investigational.
Up to 166 patients will take part in this study. All will be enrolled at MD Anderson.
5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.
This study's enrollment of 27 is below the median of 38 across 4,247 interventional studies indexed under Leukemia.
Browse Leukemia studies →M.D. Anderson Cancer Center is the lead sponsor of 2,999 studies on the registry; 581 are open to participants now.
Of its 599 completed or terminated interventional studies of FDA-regulated products, 402 (67%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Phase I - Starting dose of Ruxolitinib 50 mg by mouth twice a day for 28 day cycle.
Drug: Ruxolitinib
Phase I dose of Ruxolitinib 100 mg by mouth twice a day for 28 day cycle.
Drug: Ruxolitinib
Phase I dose of Ruxolitinib 200 mg by mouth twice a day for 28 day cycle.
Drug: Ruxolitinib
Phase I - Starting dose of 50 mg by mouth twice a day for 28 day cycle. Phase II - MTD reached in Phase I.
Also known as: Jakafi, INCBO18424, INC424
Number of Participants With Dose Limiting Toxicities (DLTs)
MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study. Participants who received at least 80% of the originally assigned doses in the first cycle were evaluable for DLT assessment of each cohort.
Time frame: End of first 28 day cycle for toxicity
Maximum Tolerated Dose (MTD) of Ruxolitinib
The MTD is defined as the highest dose level at which no more than one out of six subject experiences DLT during the first cycle (28 days) of therapy.
Time frame: End of first 28 day cycle
Participants With a Response
Response is defined as complete remission (CR) + complete remission with incomplete blood count (CRi) + Hematologic improvement (HI). Response was to be assessed for participants who were evaluated for the Phase II portion of this study. CR is absolute neutrophil count (ANC) \>/= 1x109/L and platelet count \>/= 100x109/L, absence of leukemia blast cells, normal marrow differential, and complete resolution of extramedullary disease. CRi is CR but platelets are \< 100x109/L or ANC is \<1x109/L. HI is described by the number of individual, positively affected cell lines without the use of growth factors and/or transfusions (lasting at least 4 weeks).
Time frame: Up to 1 year
Recruitment Period: December 9, 2010 to September 26, 2012. All recruitment done at The University of Texas (UT) MD Anderson Cancer Center.
| Milestone | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| Started | 4 | 5 | 18 |
| Completed | 3 | 3 | 7 |
| Not completed | 1 | 2 | 11 |
| Withdrew: Participant/physician decision | 1 | 2 | 0 |
| Withdrew: Death | 0 | 0 | 11 |
MTD defined as highest dose level at which no more than one out of six subject experiences dose limiting toxicity (DLT) during first cycle (28 days) of therapy. A non-hematologic DLT defined as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value (according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria) assessed as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during first 28 days on study. Participants who received at least 80% of the originally assigned doses in the first cycle were evaluable for DLT assessment of each cohort.
| Participants | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| Number of Participants With Dose Limiting Toxicities (DLTs) | 0 | 0 | 0 |
The MTD is defined as the highest dose level at which no more than one out of six subject experiences DLT during the first cycle (28 days) of therapy.
| mg | Ruxolitinib |
|---|---|
| Maximum Tolerated Dose (MTD) of Ruxolitinib | NA |
Response is defined as complete remission (CR) + complete remission with incomplete blood count (CRi) + Hematologic improvement (HI). Response was to be assessed for participants who were evaluated for the Phase II portion of this study. CR is absolute neutrophil count (ANC) \>/= 1x109/L and platelet count \>/= 100x109/L, absence of leukemia blast cells, normal marrow differential, and complete resolution of extramedullary disease. CRi is CR but platelets are \< 100x109/L or ANC is \<1x109/L. HI is described by the number of individual, positively affected cell lines without the use of growth factors and/or transfusions (lasting at least 4 weeks).
No measurements were reported for this outcome.
Collected over Adverse events collected during 28 day cycle, up to 9 cycles.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ruxolitinib 50 mg BID | — | 4/4 (100%) | 4/4 (100%) |
| Ruxolitinib 100 mg BID | — | 5/5 (100%) | 5/5 (100%) |
| Ruxolitinib 200 mg BID | — | 18/18 (100%) | 18/18 (100%) |
| Event | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| HemoptysisRespiratory, thoracic and mediastinal disorders | 3/4 | 1/5 | 0/18 |
| DeathGeneral disorders | 0/4 | 0/5 | 11/18 |
| PneumoniaRespiratory, thoracic and mediastinal disorders | 2/4 | 3/5 | 1/18 |
| Alveolar hemorrhageRespiratory, thoracic and mediastinal disorders | 1/4 | 0/5 | 0/18 |
| Cerebral edema and massNervous system disorders | 1/4 | 0/5 | 0/18 |
| FeverGeneral disorders | 1/4 | 1/5 | 1/18 |
| NeutropeniaInvestigations | 1/4 | 0/5 | 0/18 |
| PainGeneral disorders | 1/4 | 0/5 | 0/18 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/4 | 0/5 | 2/18 |
| Skin ulcerationSkin and subcutaneous tissue disorders | 1/4 | 0/5 | 0/18 |
| Event | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| InfectionsInfections and infestations | 4/4 | 2/5 | 18/18 |
| Platelets decreasedInvestigations | 2/4 | 3/5 | 6/18 |
| Neutrophils count decreasedInvestigations | 2/4 | 2/5 | 6/18 |
| Alanine aminotransferase increased (ALT)Investigations | 1/4 | 2/5 | 4/18 |
| Blood bilirubin increasedInvestigations | 0/4 | 2/5 | 1/18 |
| Creatinine IncreasedInvestigations | 1/4 | 2/5 | 4/18 |
| Muscle weaknessMusculoskeletal and connective tissue disorders | 0/4 | 2/5 | 1/18 |
| Hemoglobin IncreasedInvestigations | 1/4 | 1/5 | 0/18 |
| FatigueGeneral disorders | 1/4 | 1/5 | 1/18 |
| NauseaGastrointestinal disorders | 1/4 | 0/5 | 0/18 |
| Age, Continuous(years) | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID | Total |
|---|---|---|---|---|
| Median | 66 (51 to 69) | 70 (41 to 85) | 71 (55 to 83) | 69 (41 to 85) |
| Sex: Female, Male(Participants) | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID | Total |
|---|---|---|---|---|
| Female | 3 | 1 | 9 | 13 |
| Male | 1 | 4 | 9 | 14 |
| Region of Enrollment(Participants) | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID | Total |
|---|---|---|---|---|
| United States | 4 | 5 | 18 | 27 |
This study is terminated, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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M.D. Anderson Cancer Center