New drug aims to boost blood counts in rare pre-leukemia condition
NCT ID NCT06240754
First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 2 times
Summary
This study tests whether enasidenib, a drug that blocks a faulty IDH2 protein, can safely improve blood cell counts in people with clonal cytopenia of undetermined significance (CCUS). CCUS causes low blood counts and raises the risk of blood cancers. The trial enrolls 15 adults with IDH2 mutations and will monitor blood counts and side effects over time.
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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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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2024
- Expected to finish
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Jun 2029
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 × 109/L * Platelets \<100 × 109/L * IDH2 gene mutation (R140 or R172), performed locally, at a frequency ≥ 2%. * 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 (un upper limit of bilirubin 5 mg/dL is acceptable if it can be attributed to Gilbert's syndrome or erythropoiesis) * Creatinine clearance \> 50 mL/min by Cockcroft-Gault glomerular filtration rate estimation or serum creatinine ≤ 2 x IULN * The effects of enasidenib on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 24 months after the last dose of enasidenib. 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 4 months after the last dose of enasidenib. * Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants. 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 or received within the last 6 months. * 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 enasidenib 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. * Positive direct Coombs test.
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As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
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