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OncoMatch/Clinical Trials/NCT05483010

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

Is NCT05483010 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments including Atorvastatin and Rosuvastatin for clonal cytopenia of undetermined significance.

Phase 2RecruitingWashington University School of MedicineNCT05483010Data as of May 2026

Treatment: Atorvastatin · RosuvastatinPatients 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.

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Extracted eligibility criteria

Cancer type

Myelodysplastic Syndrome

Disease stage

Required: Stage LOWER-RISK (IPSS-R)

MDS is defined using the WHO 2016 definition and classified into lower-risk if IPSS-R score is ≤ 3.5

Prior therapy

Cannot have received: disease-modifying therapy (lenalidomide, luspatercept, imtelstat, hypomethylating agents, venetoclax)

Exception: concurrent use of erythropoetin stimulating agents

Current or prior use of disease-modifying therapy (e.g., lenalidomide, Luspatercept, Imitelstat, HMAs, venetoclax) with any dose within the last 3 months, with the exception of concurrent use of erythropoetin stimulating agents

Cannot have received: statin (statin)

Prior use of a statin within 1 year prior to start of treatment

Cannot have received: investigational agent for CCUS/MDS

Currently receiving any investigational agent for CCUS/MDS. The minimum interval between the last dose of investigational agent used for CCUS/MDS and Day 1 of this trial should be 5 half-lives of the investigational agent.

Lab requirements

Blood counts

unexplained persistent cytopenia in at least one lineage for at least 6 months: Hemoglobin < 11.3 g/dL in females or < 13 g/dL in males; ANC < 1.8 x 10^9/L; Platelets < 150 x 10^9/L

Liver function

active liver disease (acute liver failure, decompensated cirrhosis, or persistent elevation in ALT or AST > 3 x ULN) excluded; liver function profile of eligible HIV/HCV patients must be within acceptable limits

unexplained persistent cytopenia in at least one lineage for at least 6 months; active liver disease (acute liver failure, decompensated cirrhosis, or persistent elevation in ALT or AST > 3 x ULN) excluded; liver function profile of eligible HIV/HCV patients must be within acceptable limits

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • Washington University School of Medicine · St Louis, Missouri

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