NCT05483010

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

Recruiting now Phase 2 Last updated 8 April 2026

What this trial tests

Phase 2 trial testing Atorvastatin in Clonal Cytopenia of Undetermined Significance in 16 participants. Currently enrolling.

Timeline

19 February 2024

Primary endpoint
31 May 2027

31 May 2027

Quick facts

Lead sponsor	Washington University School of Medicine
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	19 February 2024
Primary completion	31 May 2027
Estimated completion	31 May 2027
Sites	1 location across United States

Drugs / interventions tested

• Atorvastatin (atorvastatin) — full drug profile →
• Rosuvastatin (rosuvastatin) — full drug profile →

Conditions studied

• Clonal Cytopenia of Undetermined Significance — all drugs for Clonal Cytopenia of Undetermined Significance →
• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →

Sponsor

Washington University School of Medicine

Who can join

18 and older, any sex, with Clonal Cytopenia of Undetermined Significance or Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. A blueprint for pursuing therapeutic interventions and early phase clinical trials in clonal haematopoiesis.
   Haque T, Shastri A, Desai P, Xie Z, et al · · 2025 · cited 9× · PMID 39653653 · DOI 10.1111/bjh.19925
2. Molecular and Immunomodulatory Mechanisms of Statins in Inflammation and Cancer Therapeutics with Emphasis on the NF-κB, NLRP3 Inflammasome, and Cytokine Regulatory Axes.
   Khan S, Huda B, Bhurka F, Patnaik R, et al · · 2025 · cited 7× · PMID 40943351 · DOI 10.3390/ijms26178429
3. Clonal Hematopoiesis of Indeterminate Potential and Cardiometabolic Disease: Challenges, Controversies and Future Perspectives.
   Anastasiou IA, Kounatidis D, Vallianou NG, Rebelos E, et al · · 2025 · cited 1× · PMID 41516113 · DOI 10.3390/ijms27010233
4. What value do zebrafish have to anticancer drug discovery?
   Xiao B, Landesman-Bollag E, Feng H. · · 2024 · cited 1× · PMID 38327017 · DOI 10.1080/17460441.2024.2313454
5. Clonal haematopoiesis and cardiovascular disease, defining risk, filling gaps, and framing the future.
   Morley AP, Carter P, Hargreaves R, Condurache DG, et al · · 2026 · PMID 42162181 · DOI 10.1038/s44325-026-00127-4
6. Clonal hematopoiesis and its progression to myeloid neoplasms: insights into risk, biology, and therapeutic strategies.
   Beeler JS, Walter MJ, Bolton KL. · · 2026 · PMID 41641638 · DOI 10.3324/haematol.2025.287488
7. Marrow Microenvironmental Pathobiology and Therapeutic Opportunities for <i>TP53</i>-Mutated Myelodysplastic Syndrome/Acute Myeloid Leukemia.
   Hunter CJ, Im AP, Shallis RM. · · 2026 · PMID 41595195 · DOI 10.3390/cancers18020275
8. Demystifying the diagnosis and management of ICUS, CHIP, and CCUS.
   Xie Z, Lee DH, Hansen DK, Loghavi S. · · 2025 · PMID 41348015 · DOI 10.1182/hematology.2025000766