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.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ACUTE MYELOID LEUKEMIA (AML) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

acute myeloid leukemia chronic myelomonocytic leukemia myelodysplastic syndrome Myelodysplastic Syndromes myelodysplastic/myeloproliferative neoplasm myeloproliferative neoplasm, unclassifiable

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