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

NCT06240754 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 202405007
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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

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

Keywords

Clonal Cytopenia of Undetermined Significance; CCUS; IDH2; Enasidenib

Outcome Measures

Primary Outcomes (1)

• Best hematologic response

  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

  Up to 18 cycles (each cycle is 28 days) of treatment (up to approximately 17 months)

Secondary Outcomes (3)

• Toxicity as measured by the number of adverse events experienced by participant

  From start of treatment through 30 days after the last day of treatment (up to approximately 18 months)

• Change in mutant IDH2 variant allele fraction

  Baseline, day 1 of cycles 3/6/9/12/15 (each cycle is 28 days), and end of treatment (up to approximately 17 months)

• Duration of hematologic improvement

  From start of treatment through completion of treatment (estimated to be 17 months)

Study Arms (1)

Enasidenib

EXPERIMENTAL

Participants will receive enasidenib 100 mg daily for 18 cycles (each cycle is 28 days). 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.

Drug: Enasidenib

Interventions

Enasidenib DRUG

Provided by BMS.

Enasidenib

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Washington University School of Medicine: lead
• Bristol-Myers Squibb: collaborator
• Damon Runyon Cancer Research Foundation: collaborator

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

RECRUITING

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Interventions

enasidenib

Study Officials

• Giulia Petrone, M.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Giulia Petrone, M.D.

CONTACT

314-362-6826; gpetrone@wustl.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 16, 2024
• First Posted: February 5, 2024
• Study Start: October 10, 2024
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): February 28, 2029
• Last Updated: February 11, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: The data will be available immediately following publication with no end date.
• Access Criteria: The data will be available immediately following publication with no end date. Access will be provided to anyone and for any purpose.

Locations

US (1)