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Alkylating Agent

Venetoclax + Chemotherapy Before Stem Cell Transplant for Leukemia

Phase 2
Waitlist Available
Led By Uday R Popat
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Performance score of >= 70 by Karnofsky/Lansky or performance status (PS) 0 or 1 (Eastern Cooperative Oncology Group [ECOG] =< 1)
Human leukocyte antigen (HLA)-identical sibling or 8/8 matched unrelated donor transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2.5 years
Awards & highlights

Study Summary

This trial is testing a new chemotherapy regimen before a stem cell transplant to see if it can better kill cancer cells and help the stem cell transplant work better.

Who is the study for?
This trial is for adults aged 18-70 with acute myelogenous leukemia or myelodysplastic syndrome, either untreated or in remission. They must have a matched donor for stem cell transplant and good organ function (heart, liver, kidneys). Children may join after safety checks on adults. Exclusions include previous transplants, HIV positivity, severe comorbidities, uncontrolled infections, and heart disease.Check my eligibility
What is being tested?
The study tests venetoclax combined with chemotherapy (busulfan, cladribine, fludarabine phosphate) before a donor stem cell transplant to treat blood cancers. The goal is to see if this approach better eliminates cancer cells and supports healthy bone marrow growth compared to current methods.See study design
What are the potential side effects?
Potential side effects include reactions from the chemotherapy like nausea and fatigue; risks associated with stem cell transplantation such as infection risk due to immune suppression; possible damage to organs like the heart or liver; and complications from venetoclax including digestive issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am mostly active and can care for myself.
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I have a perfect match for a bone marrow transplant.
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I have acute myeloid leukemia or myelodysplastic syndrome, either ongoing or in remission.
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My child cannot do lung tests due to age but has a pulse oximetry of 92% or higher.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2.5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2.5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Disease free survival (DFS)
Progression free survival
Secondary outcome measures
Cumulative incidence of acute graft versus host disease (GVHD)
Cumulative incidence of chronic GVHD
Cumulative incidence of relapse
+5 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Arm II (busulfan days -20 and -13 before PBSCT)Experimental Treatment8 Interventions
PREPARATIVE REGIMEN: Patients receive venetoclax PO QD on days -22 to -3 and busulfan IV over 3 hours on days -20 and -13. Patients then receive fludarabine phosphate IV over 1 hour, cladribine IV over 2 hours, and busulfan IV over 3 hours on days -6 to -3. TRANSPLANT: Patients undergo allogeneic PBSCT on day 0.
Group II: Arm I (busulfan days -13 and -12 before PBSCT)Experimental Treatment8 Interventions
PREPARATIVE REGIMEN: Patients receive venetoclax PO QD on days -22 to -3 and busulfan IV over 3 hours on days -13 and -12. Patients then receive fludarabine phosphate IV over 1 hour, cladribine IV over 2 hours, and busulfan IV over 3 hours on days -6 to -3. TRANSPLANT: Patients undergo allogeneic PBSCT on day 0.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Allogeneic Hematopoietic Stem Cell Transplantation
2012
Completed Phase 2
~1200
Cladribine
2014
Completed Phase 4
~4390
Busulfan
2008
Completed Phase 3
~1120
Venetoclax
2019
Completed Phase 3
~1950
Fludarabine Phosphate
1997
Completed Phase 3
~2390
Peripheral Blood Stem Cell Transplantation
1997
Completed Phase 3
~1330

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)NIH
13,717 Previous Clinical Trials
40,953,247 Total Patients Enrolled
M.D. Anderson Cancer CenterLead Sponsor
2,987 Previous Clinical Trials
1,797,898 Total Patients Enrolled
Uday R PopatPrincipal InvestigatorM.D. Anderson Cancer Center
5 Previous Clinical Trials
412 Total Patients Enrolled

Media Library

Busulfan (Alkylating Agent) Clinical Trial Eligibility Overview. Trial Name: NCT02250937 — Phase 2
Myelodysplastic Syndrome Research Study Groups: Arm I (busulfan days -13 and -12 before PBSCT), Arm II (busulfan days -20 and -13 before PBSCT)
Myelodysplastic Syndrome Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT02250937 — Phase 2
Busulfan (Alkylating Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02250937 — Phase 2
~14 spots leftby Oct 2025
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