Venetoclax + Chemotherapy Before Stem Cell Transplant for Leukemia

Recruiting in Palo Alto (17 mi)

Uday R Popat | MD Anderson Cancer Center

Overseen byUday R. Popat

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: M.D. Anderson Cancer Center

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

What is the purpose of this trial?

This randomized phase II trial studies how well venetoclax and sequential busulfan, cladribine, and fludarabine phosphate before donor stem cell transplant work in treating patients with acute myelogenous leukemia or myelodysplastic syndrome. Giving chemotherapy before a donor peripheral blood stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells.

Eligibility Criteria

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.

Inclusion Criteria

I, or someone legally allowed, can sign the consent form; if I'm 7-17, I can also agree to participate.

I am mostly active and can care for myself.

Forced expiratory volume in 1 second (FEV1) >= 50% of expected corrected for hemoglobin and/or volume

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Exclusion Criteria

I have had a stem cell transplant before.

I have had heart disease related to my arteries.

I am HIV positive.

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Treatment Details

Interventions

Allogeneic Hematopoietic Stem Cell Transplantation (Procedure)
Busulfan (Alkylating Agent)
Cladribine (Antimetabolite)
Fludarabine Phosphate (Antimetabolite)
Peripheral Blood Stem Cell Transplantation (Procedure)
Venetoclax (BCL-2 Inhibitor)

Trial OverviewThe 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.

Participant Groups

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.

Busulfan is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸 Approved in United States as Busulfex for: