Could a platelet-boosting drug improve blood counts in certain blood cancers?
NCT ID NCT06630221
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests whether eltrombopag, a drug already approved for other blood disorders, can improve blood cell counts in people with low-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) who have a mutation in the TET2 gene. Participants take eltrombopag daily for up to 15 cycles. The study measures how well the drug restores healthy blood counts and tracks changes in the TET2 mutation over time.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- eltrombopag (Promacta)
- What this could lead to
- If it works, this could offer a new treatment option for people with low-risk MDS or CMML who have TET2 mutations, potentially improving blood counts and delaying disease progression.
- What could go wrong
- This is a small, early-phase trial with only 25 participants, so results may not apply to everyone. Eltrombopag is not yet approved for this use, and side effects or lack of response are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 25 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2025
- Expected to finish
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Jan 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age ≥ 18 years at the time of signing the informed consent form. * Willing and able to adhere to the study visit schedule and other protocol requirements. * Established diagnosis of 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)z,\[14\] with any one of the notable cytopenias as defined below: 1. Hgb \< 10 g/dL prior to enrollment 2. ANC \< 1.5×10\^9/L 3. Platelets \< 100×10\^9/L * 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. * TET2 mutation performed at a frequency of at least \> 5%. * ECOG performance status of 0-2. * Adequate organ function, defined as: 1. 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. 2. Creatinine clearance greater than 30 mL/min based on the Cockroft-Gault glomerular filtration rate estimation. 3. 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 4. Hepatitis panel negative for Hep B and Hep C infection 5. Negative for HIV infection * Women of childbearing potential (WOCBP) may participate provided they have a negative serum pregnancy test at screening and a negative serum or urine pregnancy test within 72 h of starting treatment. * WOCBP and males with partners who are WOCBP must agree to abstain from sexual intercourse or use effective contraception (methods that result in \< 1% pregnancy rates) during eltrombopag therapy and for at least 7 days after the last eltrombopag dose. Males with partners who are WOCBP must agree to use a barrier method. Exclusion Criteria: * High- and Very High-risk MDS (per IPSS-R) * CMML 1-2 * Prior HMA exposure * Platelet count \> 200×10\^9/uL or leukocytosis of at least 25×10⁹/L * Marrow fibrosis (any grade) * Results of bone marrow biopsy within 1 month of study entry (screening bone marrow biopsy) indicating high-risk MDS or CMML-2. * Elevated LFTs (aminotransferases and bilirubin) \> 2x ULN * Pre-existing cardiovascular disease (e.g., known coronary artery disease with percutaneous intervention or stroke within the last year) or arrhythmia (e.g., atrial fibrillation) associated with an increased risk of thromboembolic events, unless deemed acceptable by the enrolling treating physician. * History of arterial or venous thromboembolism, and on anticoagulation. * Severe hepatic impairment (Child-Pugh Class C) * Recent history of cancer (i.e., within the past 5 years) with \> 50% chance of cancer recurrence in the next 5 years * Current or prior history of hematologic malignancy * Known dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally. * Active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment.) * Positive direct Coombs test * Evidence of hypersplenism on physical exam * Pregnant or lactating (women)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute
Cleveland, Ohio, 44195, United States
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