Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT06630221

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

Led by Abhay Singh, MD MPH · Updated on 2025-07-03

25

Participants Needed

1

Research Sites

237 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older at time of consent
  • Ability and willingness to follow study visit schedule and protocol requirements
  • Diagnosed with very low-, low-, or intermediate-risk MDS (IPSS-R < 3.5) with less than 5% myeloblasts or CMML-0 with less than 2% blasts in peripheral blood and less than 5% blasts in bone marrow
  • Presence of at least one cytopenia: hemoglobin < 10 g/dL, ANC < 1.5 x 10^9/L, or platelets < 100 x 10^9/L
  • Relapsed, refractory, intolerant, or inadequate response to known MDS therapies such as erythropoietins, luspatercept, hypomethylating agents, or lenalidomide for del(5q) patients
  • TET2 mutation frequency greater than 5%
  • ECOG performance status between 0 and 2
  • Adequate organ function including bilirubin less than twice the upper limit of normal (unless Gilbert's syndrome), creatinine clearance over 30 mL/min, normal folate, B12, iron studies, haptoglobin and peripheral smear for anemia cases
  • Negative hepatitis B and C tests
  • Negative HIV test
  • Women of childbearing potential must have negative pregnancy tests at screening and before treatment
  • Agreement to use effective contraception or abstain from sexual intercourse during and for 7 days after eltrombopag treatment
Not Eligible

You will not qualify if you...

  • High- or very high-risk MDS
  • CMML stages 1 or 2
  • Prior exposure to hypomethylating agents
  • Platelet count above 200 x 10^9/L or white blood cell count at least 25 x 10^9/L
  • Any grade of bone marrow fibrosis
  • Bone marrow biopsy within 1 month showing high-risk MDS or CMML-2
  • Liver function tests more than twice the upper limit of normal
  • Pre-existing cardiovascular disease such as recent coronary artery disease or stroke, or arrhythmias increasing thromboembolism risk unless approved by physician
  • History of blood clots and on anticoagulation therapy
  • Severe liver impairment (Child-Pugh Class C)
  • Recent cancer history with over 50% chance of recurrence within 5 years
  • Current or past blood cancers
  • Conditions affecting swallowing or drug absorption such as dysphagia or gastroparesis
  • Active uncontrolled infections
  • Positive direct Coombs test
  • Signs of hypersplenism
  • Pregnant or breastfeeding women

AI-Screening

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Trial Site Locations

Total: 1 location

1

Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute

Cleveland, Ohio, United States, 44195

Actively Recruiting

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Research Team

A

Abhay Singh, MD, MPH

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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