Blood and Lymph Conditions

All Conditions

Cytopenia

Anemia

Leukopenia

Thrombocytopenia

Hematologic Diseases

Leukocyte Disorders

Blood Platelet Disorders

Participants will have CCUS with mutations in IDH2

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.

Enasidenib

Eytan Stein, MD

Aaron Goldberg, MD, PhD

Mrinal Patnaik, MD

Abhay Singh, MD, MPH

Uma Borate, MBBS

Brian Druker, MD

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.

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

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.

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.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance	NCT05102370	Clonal Cytopeni... CCUS Clonal Cyt...	Enasidenib	18 Years -	Memorial Sloan Kettering Cancer Center
A Study of Enasidenib in People With Clonal Cytopenia of Undetermined Significance	NCT05102370	Clonal Cytopeni... CCUS Clonal Cyt...	Enasidenib	18 Years -	Memorial Sloan Kettering Cancer Center

Spots Global Cancer Trial Database for ccus clonal cytopenia of undetermined significance