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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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20 people
The number who actually took part.
- Started
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Apr 2022
- Expected to finish
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Jan 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Unexplained cytopenia for at least 6 months. Cytopenia is defined as the presence of ≥1 blood count indexes below the following thresholds: * Hgb \<10 g/dL * ANC \<1.8 × 10\^9/L * Platelets \<100 × 10\^9/L * IDH1 gene mutation (R132) confirmed by droplet digital PCR (ddPCR) testing, at a frequency \> 2%. This will be performed locally and confirmed at Washington University. * At least 18 years of age. * ECOG performance status 0-2 * Adequate organ function as defined below: * AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN * Serum total bilirubin \< 1.5 x IULN (an upper limit of bilirubin 5mg/dL is acceptable if it can be attributed to Gilbert's syndrome or erythropoiesis) * Serum creatinine \< 2 x IULN or creatinine clearance \> 50 mL/min by Cockcroft-Gault glomerular filtration rate estimation * The effects of ivosidenib on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (defined in Section 5.5) prior to study entry, for the duration of study participation, and for 90 days after the last dose of ivosidenib. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of the study, and for 90 days after the last dose of ivosidenib. * Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable). Exclusion Criteria: * Indication of hematologic disease by bone marrow biopsy within 6 months of study entry. \*Evidence of disease progression from time of bone marrow biopsy to enrollment based on investigator review of symptoms and complete blood counts * Active malignancy (defined as \> 1 cm disease on most recent CT scan in the past 6 months). * Currently receiving therapy for solid tumor malignancy. * Currently receiving any other investigational agents. * Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. * A history of allergic reactions attributed to compounds of similar chemical or biologic composition to ivosidenib or other agents used in the study. * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia. * Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 72 hours of study entry. * Heartrate corrected QT interval (QTc) \> 450 msec or with other factors that increase the risk of QT prolongation or arrhythmic events (e.g. heart failure, hypokalemia, family history of long QT interval syndrome). * Known medical history of progressive multifocal leukoencephalopathy (PML). * Currently taking medications known to be CYP3A4 strong inducers and sensitive substrates.
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Genom att skicka in godkänner du våra Användarvillkor
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Cleveland Clinic - Case Comprehensive Cancer Center
Cleveland, Ohio, 44195, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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