Ipilimumab in Treating Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia


This phase I trial studies the side effects and best dose of ipilimumab in treating patients with relapsed or refractory high-risk myelodysplastic syndrome or acute myeloid leukemia. Monoclonal antibodies, such as ipilimumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them

Study Start Date

December 2012

Estimated Completion Date

December 2016


  • Biological: ipilimumab
  • Biological: Ipilimumab
  • Other: laboratory biomarker analysis
  • Other: Laboratory Biomarker Analysis


  • Oncology: Leukemia/Lymphoma,Pharmacology/Therapy
  • Pharmacy: Chemotherapy/Oncology,Drug Trials

MeSH Terms

  • Ipilimumab
  • Leukemia, Myeloid, Acute
  • Myelodysplastic Syndromes

Study ID

National Cancer Institute (NCI) -- NCI-2012-02990



Trial ID


Study Type


Trial Phase

Phase 1

Enrollment Quota



National Cancer Institute (NCI)

Inclusion Criteria

  • Patients must be able to understand and voluntarily sign an informed consent form
  • Able to adhere to the study visit schedule and other protocol requirements
  • Life expectancy of greater than 6 months
  • Must have one of the following diagnoses:
  • Pathologically confirmed MDS with high risk features as defined by intermediate (INT)-2 or high International Prognostic Scoring System (IPSS) score, or INT-1 MDS with excess blasts (>= 5% blasts in bone marrow [BM]) or red blood cell (RBC) transfusion-dependency who have failed to respond or relapsed after an initial response to hypomethylating agents 5-azacitidine or decitabine
  • failure to respond is defined as failing to achieve a CR, PR or HI after at least 4 cycles of hypomethylating therapy these patients could have received other therapies or not, but must have received hypomethylating therapy
  • Pathologically confirmed AML patients who have received one or two courses of induction chemotherapy or hypomethylating agent therapy AND no plans for further chemotherapy therapy, but remain with residual disease of < 5% blasts in BM, by morphology, cytogenetics, fluorescent in situ hybridization (FISH), polymerase chain reaction (PCR) or flow cytometry
  • Patients must not have received any other treatment for their disease, including hematopoietic growth factors, within three weeks of beginning the trial, and should have recovered from all toxicities of prior therapy (to grade 0 or 1)
  • Eastern Cooperative Oncology Group (ECOG) performance status of =< 2 at study entry ECOG, or Karnofsky >= 60%
  • Calculated creatinine clearance by Modification of Diet in Renal Disease (MDRD) (CrCL) > 50 ml/min/1.73 squared meter
  • Total bilirubin =< 2.0 mg/dL unless due to Gilbert's syndrome, hemolysis, or ineffective hematopoiesis
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x upper limit of normal (ULN)
  • Females of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to start of ipilimumab
  • Patients must have no clinical evidence of central nervous system (CNS) or pulmonary leukostasis, disseminated intravascular coagulation, or CNS leukemia
  • Patients must have no serious or uncontrolled medical conditions

Exclusion Criteria

  • Any serious medical condition, uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, laboratory abnormality, or psychiatric illness/social situations that would limit compliance with study requirements or prevent the subject from signing the informed consent form
  • Pregnant or breast feeding females (lactating females must agree not to breast feed while taking ipilimumab)
  • Any condition, including the presence of laboratory abnormalities, 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
  • Use of any other experimental drug or therapy within 21 days of baseline
  • Known hypersensitivity to ipilimumab or history of allergic reactions attributed to compounds of similar chemical or biologic composition to ipilimumab
  • Prior use of ipilimumab, other cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) blocking therapies, anti-programmed cell death 1 (PD 1) antibody, cluster of differentiation (CD) 137 agonist or other immune activating therapy such as anti-CD 40 antibody within the last 3 months of enrollment in the study
  • if any of these of these agents were used more than 3 months earlier to enrollment in study, the patient should have recovered from all toxicity and at least 3 months had passed since last use to allow for clearance and observation of any other side effects from the previous therapy
  • Concurrent use of other anti-cancer agents or treatments, including other investigational agents
  • Autoimmune disease: patients with a history of inflammatory bowel disease, including ulcerative colitis and Crohn's disease, are excluded from this study, as are patients with a history of symptomatic disease (e.g., rheumatoid arthritis, systemic progressive sclerosis [scleroderma], systemic lupus erythematosus, autoimmune vasculitis [e.g., Wegener's Granulomatosis])
  • central nervous system (CNS) or motor neuropathy considered of autoimmune origin (e.g. Guillain-Barre Syndrome and Myasthenia Gravis, multiple sclerosis)
  • Patients with known immune impairment who may be unable to respond to anti-CTLA-4 antibody
  • Patients with known other cancers, including brain metastases
  • Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
  • Patients with chronic human immunodeficiency virus (HIV), hepatitis B or hepatitis C infections should be excluded because of potential effects on immune function and/ or possible drug interactions
  • Pregnant women are excluded from this study
  • breastfeeding should be discontinued if the mother is treated with ipilimumab




18 Years and older

Accepts Healthy Volunteers


Study Locations and Contact Information (7)

Study Location Distance Name Phone Email
YaleNew Haven Hospital North Haven Medical Center - North Haven, Connecticut 23.3 miles Amer M Zeidan 203-785-5702 amer.zeidan@yale.edu
Columbia UniversityHerbert Irving Cancer Center - New York, New York 44.8 miles Mark G Frattini 212-851-4872 mgf2122@columbia.edu
Johns Hopkins UniversitySidney Kimmel Cancer Center - Baltimore, Maryland 219.6 miles B D Smith 410-614-5068 bdsmith@jhmi.edu
Duke University Medical Center - Durham, North Carolina 474.7 miles Herbert I Hurwitz 919-681-6006 Herbert.Hurwitz@dm.duke.edu
UNC Lineberger Comprehensive Cancer Center - Chapel Hill, North Carolina 482.1 miles Joshua F Zeidner 919-962-5164 Joshua_Zeidner@med.unc.edu
Washington University School of Medicine - Saint Louis, Missouri 919.8 miles Mark A Schroeder 314-454-8304 mschroed@dom.wustl.edu
Texas Oncology at Baylor Irving Cancer Center - Irving, Texas 1,428.1 miles Moshe Y Levy 214-370-1033 mylevy@gmail.com

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