Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for MDS patients: drug may end need for platelet transfusions

NCT ID NCT04797000

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

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
Paused for now. It may or may not start again.
Ongoing This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tests a drug called eltrombopag in adults with a low-risk form of myelodysplastic syndromes (MDS) who regularly need platelet transfusions. The goal is to see if the drug can help them go without transfusions for at least 8 weeks. About 36 people in Japan will take part, and half will get the drug while the other half gets a placebo.

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

36 people

The number who actually took part.

Started

May 2021

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

20 to 100 years

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: * Patients diagnosed with MDS according to the WHO classification revised 4th edition by investigator assessment with one of the following prognostic risk categories, based on the International Prognostic Scoring System (IPSS-R): * very low (0-1.5) * low (2-3) * intermediate risks (3.5-4.5) All following criteria for prognostic variables per IPSS-R should be met. * Bone marrow blast \< 5% (per both investigator's assessment and central review) * Cytogenetic very good, good or intermediate risk corresponding to IPSS-R * Platelet transfusion dependence * Refractory, intolerant to, or ineligible for MDS treatments * Eastern Cooperative Oncology Group (ECOG) Performance status (PS) 0 or 1 Exclusion Criteria: * Patients with a history of prior administration of eltrombopag, romiplostim, or other TPO-RA * Therapy-related MDS per WHO classification revised 4th edition * MDS/myeloproliferative neoplasms including chronic myelomonocytic leukaemia per the WHO classification revised 4th edition * MDS with excess blasts (EB) per WHO classification revised 4th edition * Known history of IPSS-R high or very high risk MDS * Currently receiving treatments for MDS (e.g., HMA, cyclosporine A (CsA) or lenalidomide). Supportive treatment with erythropoiesis-stimulating agents (ESAs) or erythroid mutation agent in anemic patients or granulocyte-colony stimulating factor (G-CSF) in patients with severe neutropenia and recurrent infections is allowed if at stable dosage for 3 months prior to screening and continued at the same dosing/schedule until the optimal dose of eltrombopag has been established. * Patients scheduled for hematopoietic stem cell transplantation * Bone marrow fibrosis that leads to an inability to aspirate adequate bone marrow sample * Known thrombophilic risk factors (except in cases where potential benefits of participating in the study outweighed potential risks of thromboembolic events (TEE), as determined by the investigator) Other protocol-defined inclusion/exclusion criteria may apply.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Myelodysplastic syndromes are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myelodysplastic syndromes

Contacts and locations

Locations

  • Novartis Investigative Site

    Narita, Chiba, 286-8523, Japan

  • Novartis Investigative Site

    Kurume, Fukuoka, 830-8543, Japan

  • Novartis Investigative Site

    Ohtake, Hiroshima, 739-0696, Japan

  • Novartis Investigative Site

    Nishinomiya, Hyōgo, 663 8501, Japan

  • Novartis Investigative Site

    Kanazawa, Ishikawa-ken, 920-8530, Japan

  • Novartis Investigative Site

    Isehara, Kanagawa, 259-1193, Japan

  • Novartis Investigative Site

    Yokohama, Kanagawa, 221-0855, Japan

  • Novartis Investigative Site

    Sendai, Miyagi, 9808574, Japan

  • Novartis Investigative Site

    Nagasaki, Nagasaki, 852-8501, Japan

  • Novartis Investigative Site

    Sakai, Osaka, 590-0197, Japan

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 113-8603, Japan

  • Novartis Investigative Site

    Itabashi Ku, Tokyo, 173 8606, Japan

  • Novartis Investigative Site

    Shimonoseki, Yamaguchi, 750-0061, Japan

  • Novartis Investigative Site

    Aomori, 030 8553, Japan

  • Novartis Investigative Site

    Chiba, 260-0852, Japan

  • Novartis Investigative Site

    Fukushima, 9601295, Japan

  • Novartis Investigative Site

    Gifu, 5008513, Japan

  • Novartis Investigative Site

    Kumamoto, 860-0008, Japan

  • Novartis Investigative Site

    Osaka, 5340021, Japan

  • Novartis Investigative Site

    Yamagata, 990 9585, Japan

More trials for these conditions

Other studies related to the condition(s) this trial covers.