Nivolumab and/or Ipilimumab With or Without Azacitidine in Treating Patients With Myelodysplastic Syndrome
Combination of Nivolumab and Ipilimumab With 5-Azacitidine in Patients With Myelodysplastic Syndromes (MDS)
2 other identifiers
interventional
99
1 country
1
Brief Summary
This phase II trial studies the side effects of nivolumab and/or ipilimumab with or without azacitidine and to see how well they work in treating patients with myelodysplastic syndrome. Monoclonal antibodies, such as nivolumab and ipilimumab, may block cancer growth in different ways by targeting certain cells. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nivolumab and/or ipilimumab with or without azacitidine may work better in treating myelodysplastic syndrome.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2 leukemia
Started Sep 2015
Longer than P75 for phase_2 leukemia
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 19, 2015
CompletedFirst Posted
Study publicly available on registry
August 21, 2015
CompletedStudy Start
First participant enrolled
September 8, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 30, 2027
March 5, 2026
March 1, 2026
12.1 years
August 19, 2015
March 3, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Overall Response Rate (ORR) in MDS Participants with Hypomethylating Agent Failure
Overall response rate (ORR) defined as complete response plus partial response (CR + PR) + hematological improvement (HI).
24 weeks
Overall Response Rate (ORR) in MDS Participants Who Have Not Received Hypomethylating Agents
Overall response rate (ORR) defined as complete response plus partial response (CR + PR) + hematological improvement (HI).
30 weeks
Study Arms (6)
Cohort I (nivolumab)
EXPERIMENTALPatients receive nivolumab IV over 30 minutes on days 1 and 15. Treatment repeats every 4 weeks for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Patients with disease progression may receive nivolumab and azacitidine at the discretion of the treating physician.
Cohort II (ipilimumab)
EXPERIMENTALPatients receive ipilimumab IV over 30 minutes on day 1. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients with disease progression may receive ipilimumab and azacitidine at the discretion of the treating physician.
Cohort III (nivolumab, ipilimumab)
EXPERIMENTALPatients receive nivolumab IV over 30 minutes on days 1 and 15 and ipilimumab IV over 30 minutes on day 1. Treatment repeats every 4 weeks for up to 4 cycles in the absence of disease progression or unacceptable toxicity. Patients then receive nivolumab IV over 30 minutes every 2 weeks (or every 4 weeks if patients receive azacitidine) in the absence of disease progression or unacceptable toxicity. Patients with disease progression may receive ipilimumab, nivolumab, and azacitidine at the discretion of the treating physician.
Cohort IV (azacitidine, nivolumab)
EXPERIMENTALPatients receive azacitidine IV over 10-40 minutes on days 1-5 and nivolumab IV over 30 minutes on days 6 and 20. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cohort V (azacitidine, ipilimumab)
EXPERIMENTALPatients receive azacitidine IV over 10-40 minutes on days 1-5 and ipilimumab IV over 30 minutes on day 6. Cycles repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cohort VI (azacitidine, nivolumab, ipilimumab)
EXPERIMENTALPatients receive azacitidine IV over 10-40 minutes on days 1-5 and nivolumab IV over 30 minutes and ipilimumab IV over 30 minutes on day 6. Treatment with ipilimumab repeats every 4 weeks for 4 cycles in the absence of disease progression or unacceptable toxicity. Cycles with nivolumab and azacitidine repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Interventions
Given IV
Correlative studies
Given IV
Given IV
Eligibility Criteria
You may qualify if:
- Patients with MDS (up to 20% blasts) of any risk as defined as:
- Previously untreated
- Previously treated with hypomethylating agent (HMA) agent; patients need to have relapsed or progressed after any number of cycles of HMA therapy; patients that do not respond to HMA therapy will also be allowed in the study; relapse or progression will be measured by International Working Group (IWG) 2006 criteria; no response will be lack of clinical benefit after at least 6 cycles of HMA therapy
- Creatinine =\< 2.0 x upper limit of normal (ULN)
- Serum bilirubin =\< 2.0 x ULN
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 2.0 x ULN
- Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
- Females of childbearing potential must have a negative serum or urine beta human chorionic gonadotropin (beta-hCG) pregnancy test result within 24 hours prior to the first dose of treatment and must agree to use an effective contraception method to avoid pregnancy for 23 weeks (30 days plus the time required for nivolumab to undergo five half-lives) after the last dose of investigational drugs; females of non-childbearing potential are those who are postmenopausal greater than 1 year or who have had a bilateral tubal ligation or hysterectomy
- Males who have partners of childbearing potential must agree to use an effective contraceptive method during the study and a period of 31 weeks after the last dose of investigational drug
- Patients or their legally authorized representative must provide written informed consent
You may not qualify if:
- Second malignancy currently requiring active therapy, except breast or prostate cancer stable on or responding to endocrine therapy
- Any major surgery, radiotherapy, chemotherapy, biologic therapy, immunotherapy, experimental therapy within 2 weeks prior to the first dose of the study drugs
- Patients with any other known concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes; cardiovascular disease including congestive heart failure New York Heart Association \[NYHA\] class III or IV, myocardial infarction within 6 months, and poorly controlled hypertension; chronic renal failure; or active uncontrolled infection) which, in the opinion of the investigator could compromise participation in the study
- Patients unwilling or unable to comply with the protocol
- History of pneumonitis
- Patients who are on high dose steroid (equivalent of prednisone more than 10 mg a day) or immune suppression medications
- Patients with autoimmune diseases (e.g., rheumatoid arthritis, systemic progressive sclerosis \[scleroderma\], systemic lupus erythematosus, autoimmune vasculitis \[e.g., Wegener's granulomatosis\])
- Patients with a history of inflammatory bowel disease such as Crohn's disease and ulcerative colitis
- Patients known to be positive for hepatitis B surface antigen expression or with active hepatitis C infection (positive by polymerase chain reaction or on antiviral therapy for hepatitis C within the last 6 months); patients with history of human immunodeficiency virus (HIV) disease are also excluded from the study
- Current therapy with other systemic anti-neoplastic or anti-neoplastic investigational agents
- Females who are pregnant or lactating
- Prior treatment with allogeneic stem cell transplantation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- M.D. Anderson Cancer Centerlead
- National Cancer Institute (NCI)collaborator
Study Sites (1)
M D Anderson Cancer Center
Houston, Texas, 77030, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Guillermo Garcia-Manero
M.D. Anderson Cancer Center
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 19, 2015
First Posted
August 21, 2015
Study Start
September 8, 2015
Primary Completion (Estimated)
September 30, 2027
Study Completion (Estimated)
September 30, 2027
Last Updated
March 5, 2026
Record last verified: 2026-03