Can a targeted drug revive blood cell production in a Pre-Leukemic condition?

NCT ID NCT05030441

First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time

Summary

This trial tests whether ivosidenib, a drug that targets IDH1 mutations, can improve blood counts in people with clonal cytopenia of undetermined significance (CCUS) who have these mutations. CCUS is a condition with low blood counts and an increased risk of developing blood cancers. The study will measure improvements in red blood cells, platelets, and neutrophils after treatment. It also explores whether the drug reduces the proportion of cells carrying the IDH1 mutation.

What this could mean

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

Active substance
ivosidenib (Tibsovo), an oral drug that targets IDH1 mutations
What this could lead to
If successful, this could offer a targeted treatment to improve blood counts in people with CCUS and IDH1 mutations, potentially slowing or preventing progression to more serious blood disorders.
What could go wrong
This is a small pilot study, so results may not be conclusive. The drug may not improve blood counts for everyone, and side effects are possible.

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As listed by the trial registrant

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

Contacts and locations

Locations

  • Cleveland Clinic - Case Comprehensive Cancer Center

    Cleveland, Ohio, 44195, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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