18 and older, any sex, with Leukemia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Overall Response Rate (ORR)Primary· 116 days
Overall response rate (ORR) defined as complete response plus partial response (CR + PR) and hematological improvement (HI). MDS International Working Group criteria used to assess response.
Group
Value
95% CI
Low or Intermediate-1 MDS Group - Lirilumab
1
High Risk MDS Group - Azacitidine + Lirilumab
6
Adverse events — posted to ClinicalTrials.gov
Time frame: 2 years, 3 months.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Low or Intermediate-1 MDS Group - Lirilumab
Serious: 2/2 (100%)
Deaths: 0/2
Low or Intermediate-1 MDS Group - Nivolumab + Lirilumab
Serious: 0
Deaths: 0
High Risk MDS Group - Azacitidine + Lirilumab
Serious: 7/8 (88%)
Deaths: 0/8
High Risk MDS Group - Azacitidine + Lirilumab + Nivolumab
The goal of this clinical research study is to learn if lirilumab and Opdivo (nivolumab), alone or in combination with Vidaza (azacitidine), can help to control MDS. The safety of these drug combinations will also be studied.
This is an investigational study. Lirilumab is not FDA approved or commercially available. It is currently being used for research purposes. Nivolumab is FDA approved and commercially available for the treatment of melanoma and non small cell lung cancer (NSCLC). Azacitidine is FDA approved and commercially available for the treatment of MDS. The study doctor can explain how the study drugs are designed to work.
Up to 80 participants will be enrolled in this study. All will take part at MD Anderson.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03347123 — A Study of Epacadostat and Nivolumab in Combination With Immune Therapies in Participants With Advanced or Metastatic Ma
· Phase 1, PHASE2
· terminated
NCT03341936 — Neodjuvant Nivolumab and Lirilumab, Followed by Surgery, Followed by Adjuvant Nivolumab and Lirilumab, in SCCHN
· Phase 2
· active not recruiting
NCT03203876 — A Safety Study of Lirilumab in Combination With Nivolumab or in Combination With Nivolumab and Ipilimumab in Advanced an
· Phase 1
· completed
NCT02481297 — Lirilumab With Rituximab for Relapsed, Refractory or High-risk Untreated Chronic Lymphocytic Leukemia (CLL) Patients
· Phase 2
· completed
NCT02399917 — Lirilumab and Azacitidine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
· Phase 2
· terminated
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Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by M.D. Anderson Cancer Center
Last refreshed: 14 January 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02599649.