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Enasidenib for Clonal Cytopenia of Undetermined Significance with IDH2 Mutation

This study is looking into whether enasidenib is a safe and effective treatment for people with clonal cytopenia of undetermined significance (CCUS). CCUS is a condition where you have low blood cell counts (cytopenia) for at least six months without a clear cause. Researchers believe enasidenib may help because it blocks a specific mutated protein called IDH2, which could improve blood cell counts. To join, you must be at least 18 years old, have unexplained low blood counts, and have a specific IDH2 gene mutation. The main goal is to see if enasidenib improves your blood counts over approximately 17 months. This study is currently recruiting about 15 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is designed to enroll about 15 participants.
What's involved
Participants will receive enasidenib for up to 18 cycles, with each cycle lasting 28 days, for a total of approximately 17 months.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures best hematologic response for up to approximately 17 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06240754

Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial

Recruiting
PHASE2Ages 18+InterventionalTreatment
Washington University School of Medicine
~15 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:Enasidenib

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Best hematologic response
Measured over Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)

Conditions

Clonal Cytopenia of Undetermined Significance
CCUS Clonal Cytopenia of Undetermined Significance

Explore related trials

Clonal Cytopenia of Undetermined SignificanceIDH2 Gene

Where it's being run

1 sites across 1 states
Missouri1

Study leadership

  • Giulia Petrone, M.D. · PRINCIPAL_INVESTIGATOR · Washington University School of Medicine

Who to contact

Giulia Petrone, M.D.
[email protected]
314-362-6826
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Best hematologic responseUp to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)

    Hematologic response to enasidenib will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials