NCT02890329

Ipilimumab and Decitabine in Treating Patients With Relapsed or Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

Active, enrolled Phase 1 Results posted Last updated 9 September 2025

What this trial tests

Phase 1 trial testing Decitabine in Previously Treated Myelodysplastic Syndrome in 54 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

5 September 2017

Primary endpoint
24 October 2022

19 August 2026

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	54
Start date	5 September 2017
Primary completion	24 October 2022
Estimated completion	19 August 2026
Sites	11 locations across United States

Drugs / interventions tested

• Decitabine (decitabine) — full drug profile →
• Ipilimumab — full drug profile →

Conditions studied

• Previously Treated Myelodysplastic Syndrome — all drugs for Previously Treated Myelodysplastic Syndrome →
• Recurrent Acute Myeloid Leukemia — all drugs for Recurrent Acute Myeloid Leukemia →
• Recurrent Acute Myeloid Leukemia, Myelodysplasia-Related — all drugs for Recurrent Acute Myeloid Leukemia, Myelodysplasia-Related →
• Recurrent Myelodysplastic Syndrome — all drugs for Recurrent Myelodysplastic Syndrome →

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Previously Treated Myelodysplastic Syndrome or Recurrent Acute Myeloid 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.

Maximum Tolerated Dose (Recommended Phase 2 Dose) of Ipilimumab in Combination With Decitabine Primary · Monitoring for DLTs occurs continuously on treatment during the first 8 weeks of combination therapy (56 days from the start of combination) during the induction phase only.

Defined as the highest dose at which 1 or fewer of 6 patients experience a dose limiting toxicity graded by Common Terminology Criteria for Adverse Events version 5.0 criteria.

Group	Value	95% CI
Arm A (Decitabine, Ipilimumab), Post Allo-HCT	10
Arm B (Decitabine, Ipilimumab), Transplant Naive	10

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events (AE) were continuously monitored and recorded prior to each course of therapy, from the date of registration through the date of off study, for a total of 1 lead-in and 12 combination cycles (corresponding to 52 weeks).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm A (Decitabine, Ipilimumab), Post Allo-HCT, Dose Level 0

Serious: 4/7 (57%)

Deaths: 7/8

Arm B (Decitabine, Ipilimumab), Transplant Naive, Dose Level 0

Serious: 3/4 (75%)

Deaths: 6/6

Arm A (Decitabine, Ipilimumab), Post Allo-HCT, Dose Level 1

Serious: 3/3 (100%)

Deaths: 4/4

Arm B (Decitabine, Ipilimumab), Transplant Naive, Dose Level 1

Serious: 3/3 (100%)

Deaths: 5/5

Arm A (Decitabine, Ipilimumab), Post Allo-HCT, Dose Level 2

Serious: 12/15 (80%)

Deaths: 9/15

Arm B (Decitabine, Ipilimumab), Transplant Naive, Dose Level 2

Serious: 13/16 (81%)

Deaths: 11/16

Serious adverse events (85 terms)

Reaction	System	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Colitis	Gastrointestinal disorders	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Lung infection	Infections and infestations	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—
Acute kidney injury	Renal and urinary disorders	—	—	—	—	—	—
Heart failure	Cardiac disorders	—	—	—	—	—	—
Pericardial effusion	Cardiac disorders	—	—	—	—	—	—
Disease progression	General disorders	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Pneumonitis	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Rash maculo-papular	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Atrial fibrillation	Cardiac disorders	—	—	—	—	—	—
Vertigo	Ear and labyrinth disorders	—	—	—	—	—	—
Hypoadrenalism	Endocrine disorders	—	—	—	—	—	—
Hypophysitis	Endocrine disorders	—	—	—	—	—	—
Hypopituitarism	Endocrine disorders	—	—	—	—	—	—
Pre-septal cellulitis/Dacroadentitis	Eye disorders	—	—	—	—	—	—
Septal cellultiis	Eye disorders	—	—	—	—	—	—
C. diff infection	Gastrointestinal disorders	—	—	—	—	—	—
Diverticulitis	Gastrointestinal disorders	—	—	—	—	—	—

Other adverse events (247 terms — click to expand)

Reaction	System	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…	Arm A (Decitabine, Ipilimu…	Arm B (Decitabine, Ipilimu…
Rash maculo-papular	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Edema limbs	General disorders	—	—	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—
Colitis	Gastrointestinal disorders	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Platelet count decreased	Investigations	—	—	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—
Sinus tachycardia	Cardiac disorders	—	—	—	—	—	—
Fever	General disorders	—	—	—	—	—	—
Creatinine increased	Investigations	—	—	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—	—	—
Hyponatremia	Metabolism and nutrition disorders	—	—	—	—	—	—
Generalized muscle weakness	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Alkaline phosphatase increased	Investigations	—	—	—	—	—	—
Weight loss	Investigations	—	—	—	—	—	—
Pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Hyperglycemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypomagnesemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypophosphatemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Productive cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Sore throat	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—	—	—
Heart failure	Cardiac disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Dry mouth	Gastrointestinal disorders	—	—	—	—	—	—
Oral pain	Gastrointestinal disorders	—	—	—	—	—	—

Most-reported serious reactions: Febrile neutropenia, Colitis, Hypoxia, Lung infection, Sepsis, Acute kidney injury, Heart failure, Pericardial effusion.

Data from ClinicalTrials.gov NCT02890329 adverse events section.

Sponsor's own description

This phase I trial studies the side effects and best dose of ipilimumab when given together with decitabine in treating patients with myelodysplastic syndrome or acute myeloid leukemia that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Immunotherapy with monoclonal antibodies, such as ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Drugs used in chemotherapy, such as decitabine, 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 ipilimumab and decitabine may work better in treating patients with relapsed or refractory myelodysplastic syndrome or acute myeloid leukemia.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Epigenetic regulation in the tumor microenvironment: molecular mechanisms and therapeutic targets.
   Yang J, Xu J, Wang W, Zhang B, et al · · 2023 · cited 251× · PMID 37217462 · DOI 10.1038/s41392-023-01480-x
2. T-cell-based immunotherapy of acute myeloid leukemia: current concepts and future developments.
   Daver N, Alotaibi AS, Bücklein V, Subklewe M. · · 2021 · cited 178× · PMID 33953290 · DOI 10.1038/s41375-021-01253-x
3. Recent advances in targeted therapies in acute myeloid leukemia.
   Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
4. Combining epigenetic and immune therapy to overcome cancer resistance.
   Gomez S, Tabernacki T, Kobyra J, Roberts P, et al · · 2020 · cited 130× · PMID 31877341 · DOI 10.1016/j.semcancer.2019.12.019
5. Recent developments in immunotherapy of acute myeloid leukemia.
   Lichtenegger FS, Krupka C, Haubner S, Köhnke T, et al · · 2017 · cited 103× · PMID 28743264 · DOI 10.1186/s13045-017-0505-0
6. Targeting the Immune Microenvironment in Acute Myeloid Leukemia: A Focus on T Cell Immunity.
   Lamble AJ, Lind EF. · · 2018 · cited 95× · PMID 29951373 · DOI 10.3389/fonc.2018.00213
7. Checkpoint inhibitors in hematological malignancies.
   Ok CY, Young KH. · · 2017 · cited 93× · PMID 28482851 · DOI 10.1186/s13045-017-0474-3
8. Novel therapy in Acute myeloid leukemia (AML): moving toward targeted approaches.
   Winer ES, Stone RM. · · 2019 · cited 92× · PMID 31321011 · DOI 10.1177/2040620719860645