Actively Recruiting
Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)
Led by Washington University School of Medicine · Updated on 2026-04-08
16
Participants Needed
1
Research Sites
171 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of CCUS or lower-risk MDS
- CCUS defined by somatic mutations with variant allele frequency (VAF) ≥ 2% and unexplained persistent cytopenia in at least one blood lineage for 6 months or more
- Hemoglobin less than 11.3 g/dL in females or less than 13 g/dL in males
- Absolute neutrophil count (ANC) less than 1.8 x 10^9/L
- Platelet count less than 150 x 10^9/L
- Lower-risk MDS defined by WHO 2016 and IPSS-R score ≤ 3.5 with at least one mutation with VAF ≥ 2%
- Patient must be independent of blood transfusions
- At least 18 years old
- Ability and willingness to sign informed consent
You will not qualify if you...
- CCUS patients with only cytogenetic changes without mutations
- Use of disease-modifying therapies for CCUS/MDS within last 3 months except erythropoetin stimulating agents
- Use of statins within 1 year before treatment start
- History of other cancers unless fully treated over 2 years ago with no active disease
- Currently receiving any investigational agent for CCUS/MDS without sufficient washout period
- Allergic or intolerant to atorvastatin, rosuvastatin, or similar compounds
- Uncontrolled illnesses such as active infection, sepsis, or liver disease that contraindicate statin use
- Pregnant or breastfeeding women; females must have negative pregnancy test within 14 days prior to enrollment
- Patients with HIV or HCV currently on active treatment without required washout and liver function within acceptable limits
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
Research Team
A
Amber Afzal, M.D., MSCI
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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