Could a targeted drug boost transplant success for tough blood cancers?
NCT ID NCT04708054
First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 2 times
Summary
This clinical trial is testing whether adding the drug venetoclax to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study will enroll 324 participants and aims to see if the combination helps more patients stay cancer-free one year after transplant. The approach uses venetoclax to target cancer cells while the other drugs prepare the body for the transplant.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Venetoclax (a targeted cancer drug) combined with busulfan, cladribine, and fludarabine (chemotherapy drugs)
- What this could lead to
- If it works, this could lead to a more effective pre-transplant regimen that helps more people with high-risk AML or MDS stay cancer-free for longer.
- What could go wrong
- This is a mid-stage trial, so results are not yet proven. Adding venetoclax may increase side effects like low blood counts or infections, and it may not improve outcomes for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a gentler chemo prep make stem cell transplants safer for blood cancer patients?