Clinical trial · Interventional
Adding Venetoclax to the High-dose Chemotherapy Regimen Prior to Mismatche Allogeneic Stem Cell Transplant
A Phase II Trial of Venetoclax-Enhanced Reduced Intensity HLA-Mismatched Allogeneic Transplant for Ultra-High-Risk Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
NCT06337331CI-TRIAL-00077973withdrawnPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Unable to accommodate FDA recommendations for protocol revisions
Summary
Brief summary (as posted)
Patients eligible for a mismatch allogeneic stem cell transplant will receive Venetoclax daily for 7 days prior to transplant in addition to the following chemotherapy regimen: Decitabine daily for 5 days, Fludarabine daily for 5 days, and Busulfan daily for 2 days followed by 1 day of total body irradiation. Stem cell transplant will occur thereafter.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Myelodysplastic Syndromes | Myelodysplastic Syndrome | ALIAS | 0.90 |
Interventions
Interventions (5)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Busulfan | Drug | Busulfan | ALIAS |
| Decitabine | Drug | Decitabine | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Total Body Irradiation | Radiation | — | UNRESOLVED |
| Venetoclax | Drug | Venetoclax | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- High-dose Chemotherapy + Mismatched Allogeneic Stem Cell Transplant
- interventionNames
- Drug: Venetoclax
- Drug: Decitabine
- Drug: Fludarabine
- Drug: Busulfan
- Radiation: Total Body Irradiation
Primary outcomes (1)
- measure
- Incidence of relapse/progression by conducting blood and bone marrow biopsy evaluations at one-year post-transplant
- timeFrame
- 12 months
Secondary outcomes (8)
- measure
- Number of patients alive at one-year post transplant
- timeFrame
- 12 months
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Availability of a 4/8 - 6/8 HLA-matched related donor or a 7/8 HLA-matched unrelated donor * Receiving first allogeneic transplant * KPS \>/= 70% * MDS associated with TP53 mutation AND R-IPSS high or very high risk at diagnosis OR * AML with adverse risk cytogenetics or molecular abnormalities according to the 2017 ELN risk stratification OR pre-transplant MRD by either flow cytometry, cytogenetics or FISH * Less than 5% myeloblasts in the marrow pre-transplant Exclusion Criteria: * Poor cardiac function defined as LVEF \<45% * Poor pulmonary function defined as FEV1, FVC, or DLCO \<50% predicted * Poor liver function defined as bilirubin \>/=2.5mg/dL, AST/ALT \>3xULN * Poor renal function defined as creatinine \>/=2.0mg/dL or CrCl \<40mL/min * Ongoing or active systemic infection, active Hepatitis B or C virus infection, or known HIV positivity * Patient requiring treatment with a moderate or strong inhibitor or inducer of CYP3A4 or a P-gp inhibitor within 7 days prior to starting preparative chemotherapy through Day +4 post-transplant
References
Publications (26)
- RESULTEstey E, de Lima M, Tibes R, Pierce S, Kantarjian H, Champlin R, Giralt S. Prospective feasibility analysis of reduced-intensity conditioning (RIC) regimens for hematopoietic stem cell transplantation (HSCT) in elderly patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). Blood. 2007 Feb 15;109(4):1395-400. doi: 10.1182/blood-2006-05-021907. Epub 2006 Oct 12. PMID 17038533
- RESULTStelljes M, Beelen DW, Braess J, Sauerland MC, Heinecke A, Berning B, Kolb HJ, Holler E, Schwerdtfeger R, Arnold R, Spiekermann K, Muller-Tidow C, Serve HL, Silling G, Hiddemann W, Berdel WE, Buchner T, Kienast J; German AML Cooperative Group (AMLCG). Allogeneic transplantation as post-remission therapy for cytogenetically high-risk acute myeloid leukemia: landmark analysis from a single prospective multicenter trial. Haematologica. 2011 Jul;96(7):972-9. doi: 10.3324/haematol.2011.041004. Epub 2011 Apr 1. PMID 21459795
- RESULTMawad R, Gooley TA, Sandhu V, Lionberger J, Scott B, Sandmaier BM, O'Donnell P, Becker PS, Petersdorf S, Dorcy KS, Hendrie P, Sorror ML, Walter RB, Deeg HJ, Appelbaum FR, Estey EH, Pagel JM. Frequency of allogeneic hematopoietic cell transplantation among patients with high- or intermediate-risk acute myeloid leukemia in first complete remission. J Clin Oncol. 2013 Nov 1;31(31):3883-8. doi: 10.1200/JCO.2013.50.2567. Epub 2013 Sep 23. PMID 24062388
- RESULTMedeiros BC, Satram-Hoang S, Hurst D, Hoang KQ, Momin F, Reyes C. Big data analysis of treatment patterns and outcomes among elderly acute myeloid leukemia patients in the United States. Ann Hematol. 2015 Jul;94(7):1127-38. doi: 10.1007/s00277-015-2351-x. Epub 2015 Mar 20. PMID 25791241
- RESULTOstgard LSG, Lund JL, Norgaard JM, Norgaard M, Medeiros BC, Nielsen B, Nielsen OJ, Overgaard UM, Kallenbach M, Marcher CW, Riis AH, Sengelov H. Impact of Allogeneic Stem Cell Transplantation in First Complete Remission in Acute Myeloid Leukemia: A National Population-Based Cohort Study. Biol Blood Marrow Transplant. 2018 Feb;24(2):314-323. doi: 10.1016/j.bbmt.2017.10.019. Epub 2017 Oct 16.