Using enasidenib to treat clonal cytopenia of undetermined significance

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the safety and effectiveness of enasidenib, a drug that targets the mutated IDH2 protein, in patients with clonal cytopenia of undetermined significance (CCUS). Participants must have unexplained cytopenia for at least six months and an IDH2 gene mutation. The study aims to improve blood cell counts and overall health outcomes for these patients. It is a Phase 2 interventional trial conducted at Washington University School of Medicine.

Why it matters

Who can join: the full rules

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.

Where it's running

• Washington University School of Medicine
  St Louis, Missouri, United States Recruiting

Study contacts

• Principal investigator: Giulia Petrone, M.D. — Washington University School of Medicine
• Study coordinator: Giulia Petrone, M.D.
• Email: gpetrone@wustl.edu
• Phone: 314-362-6826

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.