Using statins to treat patients with clonal cytopenia and myelodysplastic syndromes

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This pilot study investigates the effects of statins, specifically atorvastatin and rosuvastatin, on patients diagnosed with clonal cytopenia of undetermined significance (CCUS) and lower-risk myelodysplastic syndromes (MDS). The study aims to assess changes in inflammatory biomarkers and variant allele frequency (VAF) of somatic mutations as indicators of response to statin therapy. Given the lack of FDA-approved treatments for these conditions, the research seeks to explore the potential of statins to improve patient outcomes and prevent disease progression. Participants must be transfusion independent and meet specific diagnostic criteria for CCUS or lower-risk MDS.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Diagnosis of CCUS or lower-risk MDS as defined below:

  * CCUS is defined as the presence of somatic mutation(s) in recurrently mutated genes identified through the clinical MyeloSeq assay with a VAF ≥ 2% in the absence of bone marrow morphology/cytogenetic changes diagnostic of MDS PLUS 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 109/L
    * Platelets \< 150 x 109/L
  * MDS is defined using the WHO 2016 definition and classified into lower-risk if IPSS-R score is ≤ 3.5 . Lower-risk MDS will be required to have at least one mutation in a recurrent mutated gene with a VAF ≥ 2%.
* Patient must be transfusion independent.
* At least 18 years of age.
* Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).

Exclusion Criteria:

* CCUS patients with cytogenetic change alone.
* 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
* Prior use of a statin within 1 year prior to start of treatment.
* A history of other malignancy with the exception of malignancies for which all treatment was completed at least 2 years before registration and the patient has no evidence active of disease.
* 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.
* A history of allergic reactions or intolerance attributed to compounds of similar chemical or biologic composition to atorvastatin, rosuvastatin, any other statin, or other agents used in the study.
* Uncontrolled intercurrent illness including, but not limited to, symptomatic infection, sepsis, or active liver disease (acute liver failure, decompensated cirrhosis, or persistent elevation in ALT or AST \> 3 x ULN), or any other comorbidity that would preclude statin use based on FDA recommendation.
* Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.
* Patients with HIV and HCV are not eligible for the trial if they are concomitantly receiving active treatment for HIV/HCV given the concern for potential drug interactions. The minimum interval between the last dose of antiviral and enrollment into the study should be 28 days or 5 half-lives of the antiviral drug, whichever is longer. The liver function profile of eligible HIV/HCV patients must be within the acceptable limits.

Where it's running

• Washington University School of Medicine
  St Louis, Missouri, United States Recruiting

Study contacts

• Principal investigator: Amber Afzal, M.D., MSCI — Washington University School of Medicine
• Study coordinator: Amber Afzal, M.D., MSCI
• Email: afzalamber@wustl.edu
• Phone: 314-273-0564

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.