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

NCT05102370 | Completed Phase 1 FDA Drug

• 1 other identifier: 21-268
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 12

Brief Summary

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.

Conditions

CCUS Clonal Cytopenia of Undetermined Significance; Clonal Cytopenia of Undetermined Significance

Keywords

CCUS; Clonal Cytopenia of Undetermined Significance; Enasidenib; 21-268; Memorial Sloan Kettering Cancer Center

Outcome Measures

Primary Outcomes (1)

• Best Response

  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

Study Arms (1)

Participants with CCUS with mutations in IDH2

EXPERIMENTAL

Participants will have CCUS with mutations in IDH2

Drug: Enasidenib

Interventions

Enasidenib DRUG

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.

Participants with CCUS with mutations in IDH2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead

Study Sites (12)

Mayo Clinic (Data Collection Only)

Rochester, Minnesota, 55905, United States

Location

Washington University (Data Collection and Specimen Analysis)

St Louis, Missouri, 63110, United States

Location

Memorial Sloan Kettering at Basking Ridge (All Protocol Activities)

Basking Ridge, New Jersey, 07920, United States

Location

Memorial Sloan Kettering Monmouth (All protocol activities)

Middletown, New Jersey, 07748, United States

Location

Memorial Sloan Kettering Bergen (Limited protocol activities)

Montvale, New Jersey, 07645, United States

Location

Memorial Sloan Kettering Suffolk-Commack (Limited protocol activities)

Commack, New York, 11725, United States

Location

Memorial Sloan Kettering Westchester (All Protocol Activities)

Harrison, New York, 10604, United States

Location

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York, 10065, United States

Location

Memorial Sloan Kettering Nassau (Limited protocol activities)

Rockville Centre, New York, 11553, United States

Location

Cleveland Clinic (Data Collection Only)

Cleveland, Ohio, 44195, United States

Location

Ohio State University (Data Collection Only)

Columbus, Ohio, 43210, United States

Location

Oregon Health & Science University (Data Collection Only)

Portland, Oregon, 97239, United States

Location

Related Links

• Memorial Sloan Kettering Cancer Center

MeSH Terms

Interventions

enasidenib

Study Officials

• Eytan Stein, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2021
• First Posted: November 1, 2021
• Study Start: October 6, 2021
• Primary Completion: January 26, 2026
• Study Completion: January 26, 2026
• Last Updated: January 27, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (12)