Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years and Over|
- - Patients with a diagnosis of primary myelofibrosis (PM), post polycythemia vera myelofibrosis (PPV MF), or post essential thrombocythemia myelofibrosis (PET MF) requiring therapy, including those previously treated and relapsed or refractory, or if newly diagnosed, with intermediate or high risk according to International Working Group (IWG-MRT) criteria.
- - Patients with a diagnosis of myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U) that require therapy.
- - Understanding and voluntarily signing an Institutional Review Board (IRB)-approved informed consent form.
- - Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
- - Direct bilirubin of =< 2 mg/dL.
- - Serum glutamate pyruvate transaminase (SGPT) =< 2.5 x upper limit of normal (ULN) or 5 x ULN if related to MF or MDS/MPN associated liver infiltration.
- - If total bilirubin is =< 2, fractionation is not required for eligibility determination.
- - Creatinine =< 2.5 mg/dL.
- - Platelets >= 50 x 10^9/L.
- - Absolute neutrophil count (ANC) >= 1.0 x 10^9/L.
- - For the MF and MDS/MPN-U arms (arms 1 & 2), use of any other standard drug (except hydroxyurea, anagrelide, growth factors, Revlimid, clofarabine, etc) or experimental drug or therapy within 14 days of starting study therapy.
- - Patients previously treated with RUX or AZA (only applicable for the MF and MDS/MPN arms) - Any serious psychological condition or psychiatric illness that would prevent the subject from signing the informed consent document, in the investigator opinion.
- - Pregnant or lactating females.
- - Subjects of childbearing potential who are unwilling to take appropriate precautions (from screening through follow-up) to avoid becoming pregnant or fathering a child; females of non-childbearing potential are defined as women who a) are 55 years of age with history of amenorrhea for 1 year OR b) are surgically sterile for at least 3 months; for females of childbearing potential, or for males, pregnancy must be avoided by taking appropriate precautions; these precautions and the methods of contraception should be communicated to the subjects and their understanding confirmed.
- - Any condition which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- - Known positive for human immunodeficiency virus (HIV) or with known active infectious hepatitis, type A, B or C.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|M.D. Anderson Cancer Center|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Principal Investigator Affiliation||M.D. Anderson Cancer Center|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Myelofibrosis Transformation in Essential Thrombocythemia, Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase, Primary Myelofibrosis|
|Study Website:||View Trial Website|
- I. To determine the efficacy of the combination of RUX (ruxolitinib phosphate) with AZA (azacytidine) in patients with myelofibrosis (MF) (primary myelofibrosis, post polycythemia vera myelofibrosis, or post essential thrombocythemia myelofibrosis [PMF, post- PV MF, or post - ET MF]) in achieving objective improvements in disease status.
- II. To determine the efficacy of the combination of RUX + AZA in patients with myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (breakpoint cluster region [BCR]-c-abl oncogene 1, non-receptor tyrosine kinase [ABL1] negative: aCML), and myelodysplastic syndromes/myeloproliferative neoplasms, unclassifiable (MDS/MPN-U), in achieving objective improvements in disease status.
- I. To determine the safety of the RUX + AZA combination in patients with MF and MDS/MPN.
- I. To explore time to response (TTR) and duration of response (DOR).
- II. To explore the effect of the combination on anemia and transfusion dependence in patients with MF and MDS/MPN.
- III. To explore the impact of baseline mutational profile on International Working Group (IWG)-Myeloproliferative Neoplasms Research and Treatment (MRT) response and overall survival in patients with MF and MDS/MPN.
- IV. To explore the impact of baseline anemia on overall survival in patients with MF and MDS/MPN.
- I. After completion of study treatment, patients are followed up for 30 days and up to 5 years.
Experimental: Arm I (MF patients)
Patients with MF receive ruxolitinib phosphate PO BID on days 1-28. Beginning course 4, patients also receive azacytidine SC or IV for 5 days. Treatment repeats every 28 days for 15 courses in the absence of disease progression or unacceptable toxicity.
Experimental: Arm II (MDS/MPN patients)
Patients with MDS/MPN receive ruxolitinib phosphate and azacytidine as in Arm I.
Drug: - Azacitidine
Given SC or IV
Other: - Laboratory Biomarker Analysis
Drug: - Ruxolitinib Phosphate
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.