18 and older, any sex, with High Risk Myelodysplastic Syndrome or IPSS Risk Category Intermediate-1. 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) Defined as Complete Response + Partial Response + Hematological ImprovementPrimary· Up to 2 years 4 months
Will estimate the ORR for the experimental treatments, along with the 95% credible intervals. The association between ORR and patient's clinical characteristics will be examined by Wilcoxon's rank sum test or Fisher's exact test, as appropriate.
Group
Value
95% CI
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
13
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
5
Event Free SurvivalPrimary· Up to 4 years
Will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests.
Group
Value
95% CI
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
9.24
3.98 – 31.23
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) failureHMA Failure
5.39
1.48 – 9.07
Overall SurvivalPrimary· Up to 4 years
Will be estimated using the method of Kaplan and Meier. Comparisons of time-to-event endpoints by important subgroups will be made using the log-rank tests. Overall Survival will be presented by median survival, which is the time point at which the cumulative survival drops below 50%. If there is no median survival (not reached), it means the cumulative survival was more than 50%.
Group
Value
95% CI
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
NA
1 – 32
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) failureHMA Failure
5.79
4.47 – 18.61
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 4 years.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Treatment (Azacitidine, Pembrolizumab) in Untreated Patients
Serious: 9/17 (53%)
Deaths: 1/17
Treatment (Azacitidine, Pembrolizumab) Patients With Hypomethylating Agent (HMA) Failure
This phase II trial studies the side effects of azacitidine and pembrolizumab and to see how well they work in treating patients with myelodysplastic syndrome. Azacitidine may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving azacitidine and pembrolizumab may work better at treating myelodysplastic syndrome.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07508982 — Phase1b/2 Trial Of AZA + APG1252 In Patients With High-Risk AML
· Phase 1, PHASE2
· not yet recruiting
NCT05554419 — Testing the Use of Combination Therapy in Patients With Persistent Low Level Acute Myeloid Leukemia Following Initial Tr
· Phase 2
· not yet recruiting
NCT07490288 — Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML
· NA
· not yet recruiting
NCT07469046 — VAH vs VA in Newly Diagnosed Elderly AML
· Phase 3
· not yet recruiting
NCT06782542 — Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for I
· Phase 2
· recruiting
Other recruiting trials for High Risk Myelodysplastic Syndrome
Currently open trials in the same condition.
NCT04128748 — Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Ris
· Phase 1, PHASE2
· recruiting
NCT04262843 — Total Marrow and Lymphoid Irradiation as Conditioning Regimen Before Hematopoietic Cell Transplantation in Patients With
· Phase 2
· active not recruiting
NCT04047641 — Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory A
· Phase 1, PHASE2
· recruiting
NCT02530034 — Hu8F4 in Treating Patients With Advanced Hematologic Malignancies
· Phase 1
· active not recruiting
NCT03672539 — Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory
· Phase 2
· active not recruiting
Other M.D. Anderson Cancer Center trials
Trials by the same sponsor.
NCT07053020 — A Phase 1b/2 Open-label, Dose-ranging Safety and Efficacy Study of Oral Cladribine in Patients With Acute Myeloid Leukem
· Phase 1, PHASE2
· not yet recruiting
NCT07052994 — A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and
· Phase 1
· not yet recruiting
NCT07137481 — Phase II Study of CD5 CAR Engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Che
· Phase 2
· not yet recruiting
NCT07162480 — Phase II Trial of Puxitatug Samrotecan (AZD8205) in Advanced, Recurrent or Metastatic (R/M) Aggressive Adenoid Cystic Ca
· Phase 2
· not yet recruiting
NCT07076498 — Engineered HSV-1 M032 for the Treatment of Children and Adults With Newly Diagnosed Diffuse Midline Glioma After Standar
· Phase 1
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 9 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: 22 March 2023
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/NCT03094637.