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NCT04550442
Venetoclax and Azacitidine for the Treatment of Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
Phase 1, PHASE2 trial testing Azacitidine in Recurrent Chronic Myelomonocytic Leukemia in 34 participants. Participants enrolled and being followed up; not accepting new ones.
31 March 2026
Quick facts
| Lead sponsor | M.D. Anderson Cancer Center |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Active, enrolled |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 34 |
| Start date | 4 September 2020 |
| Primary completion | 31 March 2026 |
| Estimated completion | 31 March 2026 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Azacitidine (azacitidine) — full drug profile →
- Venetoclax (venetoclax) — full drug profile →
Conditions studied
- Recurrent Chronic Myelomonocytic Leukemia — all drugs for Recurrent Chronic Myelomonocytic Leukemia →
- Recurrent Myelodysplastic Syndrome — all drugs for Recurrent Myelodysplastic Syndrome →
- Refractory Chronic Myelomonocytic Leukemia — all drugs for Refractory Chronic Myelomonocytic Leukemia →
- 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 Recurrent Chronic Myelomonocytic Leukemia or Recurrent Myelodysplastic Syndrome. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This phase I/II trial investigates the side effects and best dose of venetoclax when given together with azacitidine and to see how well it works in treating patients with high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back (relapsed) or has not responded to treatment (refractory). Venetoclax may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, 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 venetoclax and azacitidine together may help to control myelodysplastic syndrome or chronic myelomonocytic leukemia.
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 -
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 -
Definitions, Biology, and Current Therapeutic Landscape of Myelodysplastic/Myeloproliferative Neoplasms.
Gerke MB, Christodoulou I, Karantanos T. · · 2023 · cited 15× · PMID 37568631 · DOI 10.3390/cancers15153815 -
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 -
Myelodysplastic Syndromes and Metabolism.
Balaian E, Wobus M, Bornhäuser M, Chavakis T, et al · · 2021 · cited 10× · PMID 34681910 · DOI 10.3390/ijms222011250 -
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 -
Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax.
Bazinet A, Desikan SP, Li Z, Rodriguez-Sevilla JJ, et al · · 2024 · cited 6× · PMID 38300723 · DOI 10.1158/1078-0432.ccr-23-2860
Verify or expand the search:
- PubMed search for NCT04550442
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Other recruiting trials for Recurrent Chronic Myelomonocytic Leukemia
Currently open trials in the same condition.
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Other M.D. Anderson Cancer Center trials
Trials by the same sponsor.
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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)
- Trial protocol + status: ClinicalTrials.gov NCT04550442 (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 January 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/NCT04550442.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing