NCT02530463

Nivolumab and/or Ipilimumab With or Without Azacitidine in Treating Patients With Myelodysplastic Syndrome

Active, enrolled Phase 2 Last updated 5 March 2026

What this trial tests

Phase 2 trial testing Azacitidine in Leukemia in 99 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

8 September 2015

Primary endpoint
30 September 2027

30 September 2027

Quick facts

Lead sponsor	M.D. Anderson Cancer Center
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	99
Start date	8 September 2015
Primary completion	30 September 2027
Estimated completion	30 September 2027
Sites	1 location across United States

Drugs / interventions tested

• Azacitidine (azacitidine) — full drug profile →
• Ipilimumab — full drug profile →
• Laboratory Biomarker Analysis
• Nivolumab (nivolumab) — full drug profile →

Conditions studied

• Leukemia — all drugs for Leukemia →
• Myelodysplastic Syndrome — all drugs for Myelodysplastic Syndrome →
• Recurrent Myelodysplastic Syndrome — all drugs for Recurrent Myelodysplastic Syndrome →

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

18 and older, any sex, with Leukemia or Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

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

1. Targeting epigenetic regulators for cancer therapy: mechanisms and advances in clinical trials.
   Cheng Y, He C, Wang M, Ma X, et al · · 2019 · cited 760× · PMID 31871779 · DOI 10.1038/s41392-019-0095-0
2. Immune checkpoint therapy in liver cancer.
   Xu F, Jin T, Zhu Y, Dai C. · · 2018 · cited 299× · PMID 29843754 · DOI 10.1186/s13046-018-0777-4
3. 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
4. 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
5. 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
6. Recent developments in epigenetic cancer therapeutics: clinical advancement and emerging trends.
   Nepali K, Liou JP. · · 2021 · cited 122× · PMID 33840388 · DOI 10.1186/s12929-021-00721-x
7. Epi-drugs in combination with immunotherapy: a new avenue to improve anticancer efficacy.
   Mazzone R, Zwergel C, Mai A, Valente S. · · 2017 · cited 113× · PMID 28572863 · DOI 10.1186/s13148-017-0358-y
8. 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