Experimental combo for rare blood cancer shows promise but trial halted early

NCT ID NCT03878199

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tested a combination of two drugs—ruxolitinib and CPX-351—in 12 people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The goal was to find the best dose and see if the combo could shrink cancer. However, the study was stopped early, so we have limited information on how well it works or what side effects it may cause.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

ruxolitinib and CPX-351 (a liposomal combination of two chemotherapy drugs, daunorubicin and cytarabine)

What this could lead to

If successful, this combination could offer a new treatment option for patients with advanced myeloproliferative neoplasms that have progressed to leukemia-like stages.

What could go wrong

The trial was terminated early with only 12 participants, so results are very limited. The combination may be too toxic or not effective enough, and it is unclear if it will work for most patients.

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.

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Conditions

The condition(s) this trial relates to.

acquired polycythemia vera blast phase chronic myelogenous leukemia, BCR-ABL1 positive essential thrombocythemia myelofibrosis Myeloproliferative Disorders myeloproliferative neoplasm primary myelofibrosis therapy related acute myeloid leukemia and myelodysplastic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • OHSU

    Portland, Oregon, 97239, United States

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210, United States

  • Simmons Cancer Center

    Dallas, Texas, 75390, United States