A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance

January 26, 2026 updated by: Memorial Sloan Kettering Cancer Center

A Pilot Study of Enasidenib for Patients With Clonal Cytopenia of Undetermined Significance and Mutations in IDH2

Study researchers think that a drug called enasidenib may help people with clonal cytopenia of undetermined significance (CCUS) because the drug blocks the mutated IDH2 protein, which may improve blood cell counts. The purpose of this study is to find out whether enasidenib is a safe and effective treatment for CCUS.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic (Data Collection Only)
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University (Data Collection and Specimen Analysis)
    • New Jersey
      • Basking Ridge, New Jersey, United States, 07920
        • Memorial Sloan Kettering at Basking Ridge (All protocol activities)
      • Middletown, New Jersey, United States, 07748
        • Memorial Sloan Kettering Monmouth (All Protocol Activities)
      • Montvale, New Jersey, United States, 07645
        • Memorial Sloan Kettering Bergen (Limited Protocol Activities)
    • New York
      • Commack, New York, United States, 11725
        • Memorial Sloan Kettering Suffolk-Commack (Limited Protocol Activities)
      • Harrison, New York, United States, 10604
        • Memorial Sloan Kettering Westchester (All Protocol Activities)
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center (All Protocol Activities)
      • Rockville Centre, New York, United States, 11553
        • Memorial Sloan Kettering Nassau (Limited Protocol Activities)
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic (Data Collection Only)
      • Columbus, Ohio, United States, 43210
        • Ohio State University (Data Collection Only)
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University (Data Collection Only)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age ≥18 years at the time of signing the informed consent form.
  • Willing and able to adhere to the study visit schedule and other protocol requirements.
  • 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 × 10^9/L
  • Platelets <100 × 10^9/L
  • Results of bone marrow biopsy within 1 month of study entry (screening bone marrow biopsy) must not indicate hematologic disease.
  • IDH2 gene mutation (R140 or R172), performed locally, at a frequency >2%.
  • ECOG performance status of 0-2.

  • Adequate organ function, defined as:

    • Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤3x upper limit of normal (ULN), unless the subject has Gilbert's syndrome.
    • Serum total bilirubin <1.5x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis. In these cases, approval from the study Principal Investigator is required.
    • Creatinine clearance greater than 50 mL/min based on the Cockroft-Gault glomerular filtration rate estimation.
  • Patients being enrolled on study on the basis of anemia, will only be eligible if folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin and peripheral smear within normal limits
  • Women of childbearing potential may participate provided they have a negative serum pregnancy test at screening and a negative serum or urine pregnancy test within 72 h of starting treatment.
  • Women of childbearing potential (WOCBP) and males with partners who are WOCBP must agree to abstain from sexual intercourse or to use 1 highly effective form of contraception during the study and for at least 4 months following the last dose of enasidenib. Males with partners who are WOCBP must agree to use a barrier method.

Exclusion Criteria:

  • Active malignancy defined as >1-cm disease on most recent CT scan in the past 6 months or recent history of cancer (i.e. within the past 5 years) with >50% chance of cancer recurrence in the next 5 years.
  • Current or prior history of hematologic malignancy.
  • Therapy (including maintenance therapy) for solid-tumor malignancy within the last 6 months.
  • Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
  • Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
  • Positive direct Coombs test
  • Evidence of hypersplenism on physical exam
  • Pregnant or lactating (women).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Participants with CCUS with mutations in IDH2
Participants will have CCUS with mutations in IDH2
Drug: Enasidenib
Study participants will receive enasidenib 100 mg daily for 18 months. Participants will continue treatment with enasidenib until confirmed progression to AML or MDS, development of unacceptable toxicity, or suspicion of disease progression, provided the patient is deriving clinical benefit, which will be determined at the discretion of the principal investigator.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Best Response
Time Frame: Up to 18 months
The primary objective is to estimate the rate of hematologic improvement. The endpoint is evaluated as the best response at any point in up to 18 months of treatment with enasidenib.
Up to 18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Memorial Sloan Kettering Cancer Center

Investigators

  • Principal Investigator: Eytan Stein, MD, Memorial Sloan Kettering Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 6, 2021

Primary Completion (Actual)

January 26, 2026

Study Completion (Actual)

January 26, 2026

Study Registration Dates

First Submitted

October 20, 2021

First Submitted That Met QC Criteria

October 20, 2021

First Posted (Actual)

November 1, 2021

Study Record Updates

Last Update Posted (Actual)

January 27, 2026

Last Update Submitted That Met QC Criteria

January 26, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 21-268

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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