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NCT04160052
Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome
Terminated
Phase 1, PHASE2
Last updated 12 February 2026
What this trial tests
Phase 1, PHASE2 trial testing Azacitidine in Chronic Myelomonocytic Leukemia in 51 participants. Terminated before completion.
Timeline
1 October 2019
Primary endpoint
3 February 2026
3 February 2026
3 February 2026
Quick facts
| Lead sponsor | M.D. Anderson Cancer Center |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 51 |
| Start date | 1 October 2019 |
| Primary completion | 3 February 2026 |
| Estimated completion | 3 February 2026 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Azacitidine (azacitidine) — full drug profile →
- Venetoclax (venetoclax) — full drug profile →
Conditions studied
- Chronic Myelomonocytic Leukemia — all drugs for Chronic Myelomonocytic Leukemia →
- Myelodysplastic Syndrome — all drugs for Myelodysplastic Syndrome →
- Recurrent Myelodysplastic Syndrome — all drugs for Recurrent Myelodysplastic Syndrome →
- Refractory Myelodysplastic Syndrome — all drugs for Refractory Myelodysplastic Syndrome →
Sponsor
M.D. Anderson Cancer Center — full company profile →
Who can join
18 and older, any sex, with Chronic Myelomonocytic Leukemia or Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This phase I/II trial studies the side effects and best dose of venetoclax when given together with azacitidine in treating patients with high-risk myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory). Drugs used in chemotherapy, such as venetoclax and 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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Crosstalk between metabolic reprogramming and epigenetics in cancer: updates on mechanisms and therapeutic opportunities.
Ge T, Gu X, Jia R, Ge S, et al · · 2022 · cited 129× · PMID 36266736 · DOI 10.1002/cac2.12374 -
Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1-2 study.
Bazinet A, Darbaniyan F, Jabbour E, Montalban-Bravo G, et al · · 2022 · cited 64× · PMID 36063832 · DOI 10.1016/s2352-3026(22)00216-2 -
Emerging treatment options for patients with high-risk myelodysplastic syndrome.
Bewersdorf JP, Carraway H, Prebet T. · · 2020 · cited 24× · PMID 33240476 · DOI 10.1177/2040620720955006 -
Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML).
Bewersdorf JP, Zeidan AM. · · 2021 · cited 21× · PMID 33807279 · DOI 10.3390/cancers13071610 -
Emerging treatments for myelodysplastic syndromes: Biological rationales and clinical translation.
Rodriguez-Sevilla JJ, Adema V, Garcia-Manero G, Colla S. · · 2023 · cited 14× · PMID 36787738 · DOI 10.1016/j.xcrm.2023.100940 -
Targeting epigenetic mechanisms to overcome venetoclax resistance.
Prado G, Kaestner CL, Licht JD, Bennett RL. · · 2021 · cited 12× · PMID 33945824 · DOI 10.1016/j.bbamcr.2021.119047 -
Approaching First-Line Treatment in Patients With Advanced CMML: Hypomethylating Agents or Cytotoxic Treatment?
Liapis K, Kotsianidis I. · · 2021 · cited 9× · PMID 34966690 · DOI 10.3389/fonc.2021.801524 -
Hematopoietic stem cells with granulo-monocytic differentiation state overcome venetoclax sensitivity in patients with myelodysplastic syndromes.
Rodriguez-Sevilla JJ, Ganan-Gomez I, Ma F, Chien K, et al · · 2024 · cited 8× · PMID 38499526 · DOI 10.1038/s41467-024-46424-3
Verify or expand the search:
- PubMed search for NCT04160052
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Currently open trials in the same condition.
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Other M.D. Anderson Cancer Center trials
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT04160052 (US National Library of Medicine, public domain)
- 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: 12 February 2026
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/NCT04160052.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing