NCT05102370

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2021-10-06
• Primary completion: 2026-01-26
• Completion: 2026-01-26
• First posted: 2021-11-01
• Last updated: 2026-01-27

Locations

12 sites across 1 country: United States

Regulatory

• FDA-regulated drug study