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

Part of paid clinical trials in St Louis, Missouri.

Sponsor
Washington University School of Medicine
Study ID
NCT05483010
Phase
PHASE2
Status
Recruiting
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Conditions

  • Clonal Cytopenia of Undetermined Significance
  • Myelodysplastic Syndromes

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Atorvastatin — DRUG
    Atorvastatin is commercially available.
  • Rosuvastatin — DRUG
    Rosuvastatin is commercially available.

Study Details

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.

Key Dates

Start date
Feb 19, 2024
Status verified
Apr 2026
Primary completion
May 31, 2027
Completion
May 31, 2027

Study Design

Enrollment
16 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Atorvastatin
    * 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).
  • Experimental: Rosuvastatin
    * 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).

Primary Outcome Measure

Change in hs-CTRP levels in peripheral blood during statin therapy [ Time Frame: Pre-treatment, every 3 months while on treatment, end of treatment, 3 months after end of treatment and time of progression (estimated to be 15 months) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Washington University School of MedicineSt LouisMissouri63110
Amber Afzal, M.D., MSCI
[email protected]
314-273-0564
Amber Afzal, M.D., MSCI (PRINCIPAL_INVESTIGATOR)
Meagan Jacoby, M.D., Ph.D. (SUB_INVESTIGATOR)
Matthew Walter, M.D. (SUB_INVESTIGATOR)
Feng Gao, Ph.D. (SUB_INVESTIGATOR)

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