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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

Must not have

Prior allogeneic or autologous transplantation

Patients with prior coronary artery disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2.5 years

Awards & highlights

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a stem cell transplant before.

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I have had heart disease related to my arteries.

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I am HIV positive.

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I do not have any infections that are not responding to treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2.5 years

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Disease free survival (DFS)

Progression free survival

Secondary study objectives

Cumulative incidence of acute graft versus host disease (GVHD)

Cumulative incidence of chronic GVHD

Cumulative incidence of relapse

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

Busulfan

2008

Completed Phase 4

~1710

Allogeneic Hematopoietic Stem Cell Transplantation

2012

Completed Phase 2

~1240