Actively Recruiting
A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2: A Decentralized Trial
Led by Washington University School of Medicine · Updated on 2026-05-27
15
Participants Needed
1
Research Sites
56 weeks
Total Duration
On this page
Sponsors
W
Washington University School of Medicine
Lead Sponsor
B
Bristol-Myers Squibb
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying enasidenib as a potential treatment for people with clonal cytopenia of undetermined significance (CCUS) who have mutations in the IDH2 gene. The study aims to find out whether enasidenib is a safe and effective option for improving blood cell counts by blocking the mutated IDH2 protein. This is a Phase 2 clinical trial conducted by Washington University School of Medicine. Participants in this trial will take enasidenib 100 mg daily for up to 18 cycles, with each cycle lasting 28 days. Treatment may continue until the disease progresses to acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), unacceptable side effects occur, or disease progression is suspected but the patient is still benefiting clinically. The decision to continue treatment will be made by the principal investigator. During the study, researchers will monitor participants regularly for blood cell response and safety by tracking adverse events. They will also assess changes in the mutant IDH2 gene variant over time and measure how long blood improvements last. Participants will be followed for up to about 17 months to evaluate these outcomes and to observe any toxicities up to 30 days after treatment ends.
CONDITIONS
Brief Title
Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2A Decentralized Trial
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Unexplained cytopenia for at least 6 months with at least one blood count below thresholds: hemoglobin <10 g/dL, ANC <1.8 x 10^9/L, or platelets <100 x 10^9/L
- IDH2 gene mutation (R140 or R172) with frequency ≥ 2%
- Age 18 years or older
- ECOG performance status 0-2
- Adequate organ function: AST/ALT ≤ 3 times upper limit, total bilirubin <1.5 times upper limit (or up to 5 mg/dL if due to Gilbert's syndrome), creatinine clearance >50 mL/min or serum creatinine ≤ 2 times upper limit
- Agreement to use contraception during study and for specified periods after treatment
- Ability to understand and sign informed consent
You will not qualify if you...
- Evidence of hematologic disease progression by bone marrow biopsy within 6 months before study entry
- Active malignancy with measurable disease >1 cm on recent CT scan within 6 months
- Receiving or recently received therapy for solid tumor malignancy within 6 months
- Receiving any other investigational agents
- Conditions limiting oral drug absorption such as dysphagia, short-gut syndrome, or gastroparesis
- History of allergic reactions to enasidenib or similar compounds
- Uncontrolled illnesses including active infection, heart failure, unstable angina, or arrhythmia
- Pregnant or breastfeeding women; negative pregnancy test required for women of childbearing potential
- Positive direct Coombs test
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 18 cycles (each cycle is 28 days)
Participants receive enasidenib 100 mg daily for up to 18 cycles (each cycle is 28 days) until disease progression, unacceptable toxicity, or clinical benefit ends.
Visits on day 1 of cycles 1, 3, 6, 9, 12, 15, and end of treatment
Duration - 30 days after last day of treatment
Participants are monitored for toxicity and adverse events for 30 days after the last day of treatment.
1 follow-up visit
Trial Site Locations
Total: 1 location
1
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
G
Giulia Petrone, M.D.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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