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Anti-metabolite
Ruxolitinib + Azacytidine for Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm
Phase 2
Recruiting
Led By Naval Daver
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
Patients with a diagnosis of myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U) that require therapy
Must not have
Known positive for human immunodeficiency virus (HIV) or with known active infectious hepatitis, type A, B or C
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 30 days after completion of study treatment
Awards & highlights
No Placebo-Only Group
Summary
This trial is testing two drugs, ruxolitinib phosphate and azacytidine, in patients with specific types of blood cancers that are hard to treat. Ruxolitinib blocks enzymes needed for cancer cell growth, while azacytidine kills cancer cells or stops them from dividing. Azacytidine is a well-known anticancer drug used in the treatment of various cancers, including breast cancer, melanoma, and colon cancer. The goal is to find a more effective treatment for these patients.
Who is the study for?
This trial is for patients with myelofibrosis or myelodysplastic/myeloproliferative neoplasm who need treatment. Eligible participants include those previously treated and relapsed, or newly diagnosed with intermediate/high risk. They must have an ECOG performance status of 0-2, acceptable organ function tests, and not be pregnant or at risk of pregnancy without taking precautions.
What is being tested?
The study is testing the combination of ruxolitinib phosphate (which blocks enzymes needed for cancer cell growth) and azacytidine (a chemotherapy drug that kills or stops cancer cells from growing). The goal is to see how well these drugs work together in treating specific blood disorders.
What are the potential side effects?
Potential side effects may include reactions related to immune system suppression such as infection risks, liver issues indicated by changes in bilirubin levels, kidney problems reflected by creatinine levels, and blood-related side effects like low platelet counts or neutrophil levels.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can take care of myself and am up and about more than half of my waking hours.
Select...
I have been diagnosed with MDS/MPN-U and need treatment.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have HIV or active hepatitis A, B, or C.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 30 days after completion of study treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 30 days after completion of study treatment
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Myeloproliferative disease
Objective response rate (complete remission, partial remission, clinical improvement) in patients with myelofibrosis
Secondary study objectives
Incidence of adverse events defined as grade 3-4 clinically relevant non-hematologic toxicity or a serious adverse event that is felt to be drug related as assessed by the Common Terminology Criteria for Adverse Events version 4.0
Side effects data
From 2021 Phase 2 trial • 71 Patients • NCT0143120923%
anemia
13%
Fever
11%
platelet count decreased
9%
neutrophil count decreased
9%
sepsis
9%
lymphocyte count decreased
9%
dyspnea
6%
thromboembolic event
4%
fever
4%
hypoxia
4%
hypercalcemia
4%
hypophosphatemia
4%
Acute kidney injury
4%
sinus tachycardia
4%
respiratory failure
4%
fatigue
4%
lung infection
4%
ileus
4%
hypotension
2%
anorexia
2%
cognitive disturbance
2%
Investigations
2%
hypertension
2%
pneumonitis
2%
Hepatobiliary - Other
2%
nausea
2%
dizziness
2%
abdominal pain
2%
diarrhea
2%
pain in extremity
2%
confusion
2%
creatinine increased
2%
delirium
2%
pleural effusion
2%
upper respiratory infection
2%
vomiting
2%
wound infection
2%
Fall
2%
cardiac disorder, Other
2%
seizure
2%
skin infection
2%
syncope
2%
tumor lysis syndome
2%
Infections and infestiations - Other
2%
Anemia
2%
Musculoskeletal, Other
2%
fracture
2%
gait disturbance
2%
gastric hemorrhage
2%
sore throat
2%
leukemia secondary to oncology chemotherapy
2%
lymphedema
100%
80%
60%
40%
20%
0%
Study treatment Arm
Diffuse Large B-cell Lymphoma (DLBCL)
Peripheral T-cell Non-Hodgkin Lymphoma (PTCL)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Arm II (MDS/MPN patients)Experimental Treatment3 Interventions
Patients with MDS/MPN receive ruxolitinib phosphate and azacytidine as in Arm I.
Group II: Arm I (MF patients)Experimental Treatment3 Interventions
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ruxolitinib Phosphate
2011
Completed Phase 2
~390
Azacitidine
2012
Completed Phase 3
~1440
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Ruxolitinib Phosphate, a JAK1/2 inhibitor, blocks the overactive Janus kinase pathway in Polycythemia Vera (PV), reducing abnormal blood cell proliferation. Azacytidine, a hypomethylating agent, incorporates into DNA and RNA to inhibit DNA methyltransferase, restoring normal gene function that controls cell growth and death.
These treatments are vital for PV patients as they address the disease's root cause, helping to manage blood cell production and lower the risk of complications like thrombosis and disease progression.
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,943 Previous Clinical Trials
41,024,576 Total Patients Enrolled
M.D. Anderson Cancer CenterLead Sponsor
3,071 Previous Clinical Trials
1,803,106 Total Patients Enrolled
Naval DaverPrincipal InvestigatorM.D. Anderson Cancer Center
3 Previous Clinical Trials
205 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I haven't taken any standard or experimental drugs other than hydroxyurea, anagrelide, growth factors, Revlimid, or clofarabine in the last 14 days.I have been treated with RUX or AZA for myelofibrosis or MDS/MPN.I only have the cancer type needed for this study or a non-aggressive cancer that's treated.I have been diagnosed with a type of myelofibrosis and need treatment.I can take care of myself and am up and about more than half of my waking hours.I am willing to use birth control to prevent pregnancy during the study.I do not have HIV or active hepatitis A, B, or C.I have been diagnosed with MDS/MPN-U and need treatment.
Research Study Groups:
This trial has the following groups:- Group 1: Arm I (MF patients)
- Group 2: Arm II (MDS/MPN patients)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.