Evaluation Of Ruxolitinib And Azacytidine Combination As A Therapy For Patients With Myelofibrosis And Myelodysplastic Syndrome/ Myeloproliferative Neoplasm
Study Treatment:
If you are found to be eligible to take part in this study, you will take ruxolitinib 2
times every day by mouth. If you miss or vomit your dose of ruxolitinib, do not make up the
dose.
Beginning with Cycle 4, on Days 1-5, you will receive azacytidine either under the skin or
through a needle in your vein.
Beginning with Cycle 4, on Days 1-5, Your dose of ruxolitinib and/or azacytidine may be
raised, lowered, and/or delayed if the doctor thinks it is in your best interest.
You will be given a drug diary and asked to write down what time you take the study drugs.
Bring in any unused study drugs and bottles to each study visit.
Each cycle is 28 days.
Study Visits:
Each study visit may be performed at +/- 3 days than the day stated.
On Day 1 of Cycles 1-7 and then every 3 cycles:
- You will be asked if you have had any side effects.
- You will have a physical exam, including measurement of your vital signs and weight.
- Your spleen and liver will be measured.
- You will complete 2 questionnaires about how you are feeling.
On Day 1 of Cycles 1-6 and then every 3 cycles, your complete medical history will be
recorded.
Once a week during Cycles 1, 2, and 4, every 2 weeks during Cycles 3 and 6, and then 1 time
each month after that, blood (about 2-3 tablespoons) will be drawn for routine tests.
If the doctor thinks it is needed, after Cycles 6 and 12, you will have a bone marrow
aspiration to check the status of the disease.
Research Blood Tests:
Extra blood will be drawn for pharmacokinetic (PK) testing and pharmacodynamic (PD). PK
testing measures the amount of study drug in the body at different time points. PD testing
measures how the level of study drug in your body may affect the disease.
Blood (about 2-3 teaspoons each time) will be drawn for PK testing:
- On Day 1 of Cycle 1, before you receive the study drug, and then 1, 2, and 5 hours
later
- On Day 1 of Cycle 6, before you receive the study drug, and then 1, 2, and 5 hours
later
- On Day 1 of Cycle 12 (or at the end of study if you go off study early), before you
receive the study drug, and then 1, 2, and 5 hours later
Blood (about 2-3 teaspoons each time) will be drawn for PD testing:
- On Day 1 of Cycle 1, before you receive the study drug and then 2 hours later
- On Day 1 of Cycle 3, before you receive the study drug
- On Day 1 of Cycle 6, before you receive the study drug and then 2 hours later
- On Day 1 of Cycle 9, before you receive the study drug
- On Day 1 of Cycle 12 (or at the end of study if you go off study early), before you
receive the study drug and then 2 hours later
Length of Study:
You can take up to 15 cycles of ruxolitinib and 12 cycles of azacytidine. After 15 cycles of
ruxolitinib, if your doctor thinks it is in your best interest, you may continue taking it
off study. You will no longer be able to take the study drugs if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.
You will be called about 30 days after you go off study and asked if you have had any side
effects and/or any new treatment(s).
This is an investigational study. Ruxolitinib is FDA approved and commercially available to
treat myelofibrosis. Azacytidine is FDA approved and commercially available for
myelodysplastic syndrome. The combination of ruxolitinib and azacytidine to treat
myelofibrosis and myelodysplastic syndrome/myeloproliferative neoplasm is investigational.
Up to 50 participants will be enrolled in this study. All will take part at MD Anderson.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
Number of participants with Objective Response
Objective response, defined as CR (complete remission/response) + PR (partial remission/response)+ CI (clinical improvement) for MF patients (arm 1) and CR + PR + HI (hematologic improvement) for MDS/MPN patients (arm 2). Complete Response: No symptoms related to MDS/MPN and absolute neutrophil count >1x109/L and platelet count >100 x109/L, and normal marrow differential (< 5% blasts). Partial Response: CR with 6-25% abnormal cells in marrow or 50% decrease in bone marrow blasts. CR partial (CRp): As per CR but platelet count <100 x109/L. Hematologic Improvement (HI): Hematologic improvement described by the number of individual positively affected cell lines.
Following 6 treatment cycles of 28 days
No
Naval Daver, MD
Study Chair
UT MD Anderson Cancer Center
United States: Food and Drug Administration
2012-0737
NCT01787487
March 2013
Name | Location |
---|---|
UT MD Anderson Cancer Center | Houston, Texas 77030 |