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.

Memorial Sloan Kettering Cancer Center

Symptoms and General Pathology

Neoplasms

Rare Diseases

Syndrome

Inflammation

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Precancerous Conditions

Lipid Regulating Agents

All Drugs and Chemicals

Bone Density Conservation Agents

Rosuvastatin Calcium

Atorvastatin

Hydroxymethylglutaryl-CoA Reductase Inhibitors

Calcium

Calcium, Dietary

Anticholesteremic Agents

Hypolipidemic Agents

Antimetabolites

Enzyme Inhibitors

* Choice of statin is at the discretion of the treating physician and may depend on insurance approval.
* Atorvastatin dosing starts at 80 mg once daily.
* In the absence of disease progression or intolerable side effects, patients may receive up to 12 months of treatment.
* If a patient switches statins due to toxicity, treatment time is still limited to 12 months total (ie, if a patient receives 6 months of atorvastatin and switches to rosuvastatin, the duration of rosuvastatin will be no more than 6 months).

* Choice of statin is at the discretion of the treating physician and may depend on insurance approval.
* Rosuvastatin dosing starts at 40 mg once daily.
* In the absence of disease progression or intolerable side effects, patients may receive up to 12 months of treatment.
* If a patient switches statins due to toxicity, treatment time is still limited to 12 months total (ie, if a patient receives 6 months of atorvastatin and switches to rosuvastatin, the duration of rosuvastatin will be no more than 6 months).

Rosuvastatin

Amber Afzal, M.D., MSCI

Meagan Jacoby, M.D., Ph.D.

Matthew Walter, M.D.

Feng Gao, Ph.D.

Patients with clonal cytopenia of undetermined significance (CCUS) and lower-risk myelodysplastic syndromes (MDS) have a life expectancy of 5 to 10 years. Mortality in these patients results from progression of disease to higher-risk MDS or acute myeloid leukemia (AML) and cardiovascular events. Currently there are no FDA-approved treatments with the potential to improve survival of patients with CCUS and lower-risk MDS. Statins are an appealing class of drugs to consider in this situation as preclinical data support their potential to suppress progression of myeloid malignancy, and they have a well-established role in prevention of major cardiovascular events. This is a pilot study to explore the role of statins in treatment of patients with CCUS and lower-risk MDS. In this study, change in inflammatory biomarkers and variant allele frequency (VAF) of somatic mutations will be used as a surrogate marker of response to statin therapy. The hypothesis is that the use of statins at diagnosis of CCUS or lower-risk MDS will reduce inflammation and delay or prevent the expected increase in the VAF of somatic mutations over time.

Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)

Pilot Study of Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)

Washington University in St Louis 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 research awards and/or as otherwise required).

-Assessed by next generation sequencing (NGS) performed on peripheral blood/bone marrow.

Washington University School of Medicine

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
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
Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)	NCT05483010	Clonal Cytopeni... Myelodysplastic...	Atorvastatin Rosuvastatin	18 Years -	Washington University School of Medicine
Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)	NCT05483010	Clonal Cytopeni... Myelodysplastic...	Atorvastatin Rosuvastatin	18 Years -	Washington University School of Medicine
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

Every month we try and update this database with for ccus cancer trials from around the world to help patients and their families find trials that might be right for them.
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Spots Global Cancer Trial Database for ccus

Every month we try and update this database with for ccus cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for ccus cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.