Could a targeted drug make stem cell transplants more effective for tough blood cancers?
NCT ID NCT03613532
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy before and after a bone marrow transplant can help prevent cancer from coming back in people with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or related blood cancers. About 102 participants will receive venetoclax alongside standard conditioning drugs before transplant, and then as maintenance therapy afterward. The main goal is to find safe doses and schedules, while also tracking survival and relapse rates.
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 drug) combined with chemotherapy drugs (fludarabine, busulfan, azacitidine, decitabine/cedazuridine)
- What this could lead to
- If successful, this could lead to a safer and more effective way to prevent cancer relapse after a bone marrow transplant for people with high-risk blood cancers.
- What could go wrong
- This is an early-phase (Phase 1) trial focused on safety and dosing, so it is too soon to know if it works. Adding venetoclax may increase side effects like infections or organ damage.
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
-
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new pill boost the power of an existing leukemia treatment?
- Can a new drug combo outperform standard care for a tough blood cancer?
- Can donor immune cells fight leukemia relapse after transplant?
- Can a liposomal chemo combo outsmart tough leukemias?
- Can Real-World data unlock clues to a deadly Post-Transplant complication?
- Massive AML trial aims to find best chemo and transplant protection for younger patients