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Could a platelet-boosting drug improve blood counts in certain blood cancers?

NCT ID NCT06630221

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused This study
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 25 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Jan 2030

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. 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.

    Open the record ↗

  3. 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

  • Case Comprehensive Cancer Center, Cleveland Clinic Foundation Taussig Cancer Institute

    Cleveland, Ohio, 44195, United States

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