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Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
Sponsor: Abhay Singh, MD MPH
Summary
The purpose of this study is to evaluate if a study drug called eltrombopag can improve the blood cell counts in patients with low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) with mutations in TET2 gene, observe changes in the TET2 gene over time, and evaluate the effectiveness of the treatment. TET2 gene is one of the most frequently mutated genes (altered parts of the DNA) in MDS and CMML. Eltrombopag is a Food and Drug Administration (FDA) approved drug for the treatment of severe aplastic anemia and low levels of platelets in patients with persistent or chronic immune thrombocytopenia (ITP) and chronic hepatitis C. Eltrombopag is considered investigational (experimental) in this study because the FDA has not approved its use in the treatment of low-risk MDS or CMML. Eltrombopag is a drug that helps stimulate the body's process of making more platelets (small components of blood that help with clotting) by interacting with specific parts of cells. This interaction starts a series of signals that encourage the growth and development of the cells that produce platelets. It was found that this drug could stop the growth of TET2 mutated cells.
Official title: A Phase II Study of Eltrombopag as a Novel Therapeutic Approach for Patients With Low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) With TET2 Mutations
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
25
Start Date
2025-06-17
Completion Date
2030-01-01
Last Updated
2025-07-03
Healthy Volunteers
No
Interventions
Eltrombopag (EPAG)
50 mg, 28-day cycles, 3 initial cycles + 12 cycles on extension arm (maximum of 15 cycles)
Locations (1)
Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute
Cleveland, Ohio, United States