Eltrombopag as a New Treatment for Low-risk MDS and CMML with TET2 Mutations

Sponsor: Abhay Singh, MD MPH

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT06630221

Provider View

This study is testing if the drug eltrombopag can help improve blood cell counts in patients with low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) who have TET2 gene mutations. The study will also look at changes in the TET2 gene over time and how effective the treatment is.

Patient Parameters

Program Overview

The study aims to see if eltrombopag, a drug approved for other blood conditions, can help patients with low-risk MDS and CMML who have mutations in the TET2 gene. Eltrombopag is known to help the body make more platelets, which are important for blood clotting. In this study, it is being tested to see if it can also stop the growth of cells with TET2 mutations. The TET2 gene is often mutated in these conditions, and this study will explore if eltrombopag can be a new treatment option.

Description

This study focuses on understanding how eltrombopag can be used to treat low-risk Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) with TET2 mutations. These conditions involve changes in the blood and bone marrow, often due to mutations in the TET2 gene. Eltrombopag is a drug that helps increase platelet production and is being tested here for its potential to inhibit the growth of TET2 mutated cells. The study will look at how eltrombopag affects the TET2 gene and whether it can reduce the number of mutated cells. This could lead to a new treatment option for patients with these specific genetic changes. The study will also gather data on how patients respond to eltrombopag, especially those with TET2 mutations, to see if it can effectively lower the burden of these mutations.

Eligibility Criteria

Inclusion Criteria

  • Must be 18 years or older.
  • Willing to follow the study schedule and requirements.
  • Diagnosed with low-risk MDS or CMML with specific blood cell counts:
    • Hemoglobin less than 10 g/dL
    • Neutrophil count less than 1.5×10^9/L
    • Platelets less than 100×10^9/L
  • May have had previous treatments like erythropoietin-stimulating agents.
  • Must have a TET2 mutation at a frequency greater than 2%.
  • Must be in good general health with an ECOG performance status of 0-2.
  • Must have adequate organ function, including:
    • Bilirubin levels less than 2 times the upper limit of normal, unless due to specific conditions.
    • Creatinine clearance greater than 30 mL/min.
    • Normal levels of folate, B12, iron, and other related tests.
    • Negative for Hepatitis B, Hepatitis C, and HIV.
  • Women of childbearing potential must have a negative pregnancy test and agree to use effective contraception during and after treatment.
  • Men with partners who are women of childbearing potential must agree to use a barrier method of contraception.

Exclusion Criteria

  • High-risk MDS or CMML-2.
  • Previous treatment with certain medications (HMA).
  • High platelet count or high white blood cell count.
  • Bone marrow fibrosis.
  • Recent bone marrow biopsy showing high-risk MDS or CMML-2.
  • Elevated liver function tests.
  • Pre-existing heart disease or arrhythmia unless approved by a doctor.
  • History of blood clots and on blood thinners.
  • Severe liver disease.
  • Recent history of cancer with a high chance of recurrence.
  • Current or past blood cancer.
  • Conditions affecting drug absorption.
  • Active uncontrolled infections.
  • Positive Coombs test.
  • Enlarged spleen.
  • Pregnant or breastfeeding women.

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