OncoMatch/Clinical Trials/NCT06630221
Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
Is NCT06630221 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies Eltrombopag (EPAG) for myelodysplastic syndromes.
Treatment: Eltrombopag (EPAG) — 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.
Check if I qualifyExtracted eligibility criteria
Cancer type
Myelodysplastic Syndrome
Acute Myeloid Leukemia
Biomarker criteria
Required: TET2 mutation (frequency > 5%)
TET2 mutation performed at a frequency of at least > 5%
Disease stage
Required: Stage VERY LOW-RISK, LOW-RISK, INTERMEDIATE-RISK, CMML-0 (IPSS-R)
Excluded: Stage HIGH-RISK, VERY HIGH-RISK, CMML-1, CMML-2
very low-, low-, or intermediate-risk MDS (IPSS-R < 3.5) and < 5% myeloblasts or CMML 0 (CMML-0, for cases with < 2% blasts in PB and < 5% blasts in bone marrow (BM)); High- and Very High-risk MDS (per IPSS-R) and CMML 1-2 excluded
Performance status
ECOG 0–2(Ambulatory, capable of self-care)
Prior therapy
Must have received: therapies with known clinical benefits for MDS (erythropoiesis-stimulating agents (EPOs), luspatercept, hypomethylating agents (azacytidine, decitabine))
Must be relapsed, refractory/resistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as EPOs, luspatercept, and HMAs (i.e., azacytidine or decitabine). Patients with del (5q) must have failed prior lenalidomide therapy.
Cannot have received: hypomethylating agent
Prior HMA exposure
Lab requirements
Blood counts
Hgb < 10 g/dL, ANC < 1.5×10^9/L, Platelets < 100×10^9/L prior to enrollment; Platelet count > 200×10^9/uL or leukocytosis ≥ 25×10⁹/L excluded
Kidney function
Creatinine clearance > 30 mL/min (Cockroft-Gault)
Liver function
Serum total bilirubin < 2x ULN, unless Gilbert's syndrome or attributed to ineffective erythropoiesis with PI approval; aminotransferases and bilirubin ≤ 2x ULN; severe hepatic impairment (Child-Pugh Class C) excluded
Adequate organ function, defined as: Serum total bilirubin < 2x ULN, unless the subject has Gilbert's syndrome. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis. In these cases, approval from the study PI is required. Creatinine clearance greater than 30 mL/min based on the Cockroft-Gault glomerular filtration rate estimation. Participants being enrolled on study on the basis of anemia, will only be eligible if folate, B12, serum iron, serum ferritin, total iron binding capacity, haptoglobin and peripheral smear within normal limits. Hepatitis panel negative for Hep B and Hep C infection. Negative for HIV infection. Elevated LFTs (aminotransferases and bilirubin) > 2x ULN [excluded]. Severe hepatic impairment (Child-Pugh Class C) [excluded].
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute · Cleveland, Ohio
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