Targeted drug shows promise for rare blood condition in early trial

NCT ID NCT05102370

First seen Nov 06, 2025 · Last updated May 07, 2026 · Updated 24 times

Summary

This early study tested a drug called enasidenib in 4 adults with clonal cytopenia of undetermined significance (CCUS) who have a specific IDH2 gene mutation. The goal was to see if the drug could safely improve low blood cell counts. Researchers believe enasidenib may block the faulty protein made by the mutated gene, helping the body produce healthier blood cells.

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 CLONAL CYTOPENIA OF UNDETERMINED SIGNIFICANCE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • Cleveland Clinic (Data Collection Only)

    Cleveland, Ohio, 44195, United States

  • Mayo Clinic (Data Collection Only)

    Rochester, Minnesota, 55905, United States

  • Memorial Sloan Kettering Bergen (Limited protocol activities)

    Montvale, New Jersey, 07645, United States

  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)

    New York, New York, 10065, United States

  • Memorial Sloan Kettering Monmouth (All protocol activities)

    Middletown, New Jersey, 07748, United States

  • Memorial Sloan Kettering Nassau (Limited protocol activities)

    Rockville Centre, New York, 11553, United States

  • Memorial Sloan Kettering Suffolk-Commack (Limited protocol activities)

    Commack, New York, 11725, United States

  • Memorial Sloan Kettering Westchester (All Protocol Activities)

    Harrison, New York, 10604, United States

  • Memorial Sloan Kettering at Basking Ridge (All Protocol Activities)

    Basking Ridge, New Jersey, 07920, United States

  • Ohio State University (Data Collection Only)

    Columbus, Ohio, 43210, United States

  • Oregon Health & Science University (Data Collection Only)

    Portland, Oregon, 97239, United States

  • Washington University (Data Collection and Specimen Analysis)

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.