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Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations

NCT06630221 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2025-07-03

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Eltrombopag (EPAG)

50 mg, 28-day cycles, 3 initial cycles + 12 cycles on extension arm (maximum of 15 cycles)

Sponsors & Collaborators

  • Abhay Singh, MD MPH

    lead OTHER

Principal Investigators

  • Abhay Singh, MD, MPH · Case Comprehensive Cancer Center, Cleveland Clinic Taussig Cancer Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-17
Primary Completion
2028-01-01
Completion
2030-01-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06630221 on ClinicalTrials.gov