Could a targeted drug boost transplant success for tough blood cancers?
NCT ID NCT04708054
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 37 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.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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M D Anderson Cancer Center
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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.
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.