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New drug aims to boost blood counts in rare pre-leukemia condition

NCT ID NCT06240754

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2024

Expected to finish

Jun 2029

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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

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How to take part

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  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

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