OncoMatch/Clinical Trials/NCT04708054
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
Is NCT04708054 recruiting? Yes, currently enrolling (Jun 2026). This Phase 2/3 trial studies multiple treatments for acute myeloid leukemia.
Treatment: Busulfan · Cladribine · Fludarabine Phosphate · Thiotepa · Venetoclax — This phase II trial studies the effect of venetoclax together with busulfan, cladribine, and fludarabine in treating patients with high-risk acute myeloid leukemia or myelodysplastic syndrome who are undergoing stem cell transplant. Chemotherapy drugs, such as venetoclax, busulfan, cladribine, and fludarabine, 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. Adding venetoclax to the current standard of care stem cell transplant regimen of busulfan, fludarabine, and cladribine may help to control high-risk acute myeloid leukemia or myelodysplastic syndrome.
Check if I qualifyExtracted eligibility criteria
Treatments studied
Targeted therapy
Chemotherapy
Cancer type
Acute Myeloid Leukemia
Myelodysplastic Syndrome
Biomarker criteria
Required: TP53 mutation
Mutated P53
Required: CBL mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1/PTPN11)
Required: NRAS mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1/PTPN11)
Required: KRAS mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1/PTPN11)
Required: NF1 mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1/PTPN11)
Required: PTPN1 mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN1)
Required: PTPN11 mutation
Mutated ... Ras pathway genes (CBL, NRAS, KRAS, NF1, PTPN11)
Required: DNMT3A mutation
Mutated DNMT 3a
Required: ASXL1 mutation
Mutated ASXL1
Required: RUNX1 mutation
Mutated RUNX1
Performance status
WHO 0–1
Demographics
Prior therapy
Must have received: induction therapy
Patients with acute myeloid leukemia who have previously received induction therapy
Cannot have received: allogeneic stem cell transplantation
Prior allogeneic stem cell transplantation
Lab requirements
Kidney function
creatinine clearance ≥ 50 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hours urine collection
Liver function
AST and ALT < 3.0X ULN; Bilirubin <1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
Cardiac function
Cardiac history of CHF requiring treatment or Ejection Fraction < 50% or unstable angina [excluded]
Adequate hepatic and renal function per local laboratory reference range as follows: AST and ALT < 3.0X ULN; Bilirubin <1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin); creatinine clearance ≥ 50 mL/min; Cardiac history of CHF requiring treatment or Ejection Fraction < 50% or unstable angina [excluded]
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- M D Anderson Cancer Center · Houston, Texas
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT04708054 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior allogeneic stem cell transplantation disqualifies patients from enrollment.
Does this trial require TP53?
Yes, TP53 mutation is a required biomarker for enrollment.
Does this trial require CBL?
Yes, CBL mutation is a required biomarker for enrollment.
Does this trial require NRAS?
Yes, NRAS mutation is a required biomarker for enrollment.
Is there an age limit?
Yes. Patients must be 70 years or younger.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
Check if I qualify