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Can a Platelet-Boosting drug help control a rare bone marrow disorder?

NCT ID NCT07825207

Disease control Sponsor: Fondazione Italiana Sindromi Mielodisplastiche-ETS 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 17, 2026 · Last updated Sep 18, 2026 · Updated 1 time

Summary

Researchers are observing how well eltrombopag works in adults with hypocellular myelodysplastic neoplasms, a bone marrow condition that lowers blood cell counts. The study follows patients in Italy who receive eltrombopag alone or with immunosuppressive therapy as part of routine care. By reviewing medical records and bone marrow samples, the team aims to measure response rates, survival, and safety over 12 months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
eltrombopag, a drug that stimulates platelet production
What this could lead to
If eltrombopag helps, doctors could have clearer real-world evidence for treating low-risk hypocellular MDS, either alone or with immunosuppressive therapy.
What could go wrong
This observational study cannot prove cause and effect, and results may not apply to all MDS patients. Eltrombopag carries known risks, and responses may vary.

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.

Participants

About 100 people

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

Started

May 2026

Expected to finish

Jul 2027

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.

Who is studied

Adult patients diagnosed with h-MDS according to WHO 2022 and treated with eltrombopag included in FISiM registry

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: * Adult patients (i.e. aged ≥18 years) diagnosed with MDS-h according to WHO 2022 * IPSS-R risk very-low to intermediate * De novo or therapy related MDS * Availability of bone marrow sample for central revision * Treatment with eltrombopag, in monotherapy or associated with IST, as first -line therapy or as a subsequent therapy to the first line Exclusion Criteria: * Unavailability of bone marrow sample for central revision

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Conditions

The condition(s) this trial relates to.

myelodysplastic syndrome myelodysplastic syndrome with excess blasts Myelodysplastic Syndromes thrombocytopenia

As listed by the trial registrant

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

Myelodysplastic neoplasm Myelodysplastic syndrome

Contacts and locations

Locations

  • Comprehensive Cancer Center, Spedali Civili di Brescia

    Brescia, Italy

  • Ematologia, Dipartimento di Medicina, Chirurgia e Farmacia, Università degli Studi di Sassari

    Sassari, Italy

  • Hematology Unit, IRCCS Istituto Romagnolo per lo Studio dei Tumori Dino Amadori

    Meldola (FC), Italy

  • Hematology Unit, Ospedale di Circolo e Fondazione Macchi, ASST Sette Laghi

    Varese, Italy

  • Istituto di Ematologia L. e A. Seràgnoli, Policlinico di S. Orsola, IRCCS AOU di Bologna

    Bologna, Italy

  • MDS Unit, AOU Careggi

    Florence, Italy

  • Oncoematologia, PO Fatebenefratelli, ASST Fatebenefratelli Sacco

    Milan, Italy

  • Oncologia Medica, INOC

    Candiolo, Italy

  • SC Ematologia U, AOU Città della Salute e della Scienza, Università degli Studi di Torino

    Torino, Italy

  • SC Ematologia, Dipartimento di Oncologia, AOU Città della Salute e della Scienza

    Torino, Italy

  • SC Immunoematologia e Medicina Trasfusionale, Ospedale Carlo Poma, ASST di Mantova

    Mantua, Italy

  • SC Medicina II, PO San Paolo, ASST Santi Paolo e Carlo

    Milan, Italy

  • SCDU Ematologia e Terapie cellulari, AO Ordine Mauriziano, Università degli Studi di Torino

    Torino, Italy

  • SCDU Medicina interna ad indirizzo ematologico, AOU San Luigi Gonzaga, Università degli Studi di Torino

    Orbassano, Italy

  • SCDU di Ematologia, AO SS. Antonio e Biagio e Cesare Arrigo

    Alessandria, Italy

  • UO Ematologia con Trapianto, Dipartimento di Medicina di Precisione e Rigenerativa e Area Ionica, AOU Consorziale Policlinico, Università degli Studi Aldo Moro

    Bari, Italy

  • UO Ematologia e CTMO, Ospedale Guglielmo da Saliceto

    Piacenza, Italy

  • UO Ematologia e Immunologia clinica, Dipartimento di Medicina, AOU di Padova

    Padova, Italy

  • UO Ematologia e Terapie Cellulari, IRCCS Ospedale Policlinico San Martino

    Genova, Italy

  • UOC Ematologia e CTMO, AOU Parma

    Parma, Italy

  • UOC Ematologia e TMO, Pia Fondazione di Culto e Religione Card. G. Panico

    Tricase, Italy

  • UOC Ematologia, Dipartimento di Medicina Clinica e Chirurgia, Università di Napoli Federico II

    Naples, Italy

  • UOC Ematologia, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

    Milan, Italy

  • UOC Ematologia, Ospedale Maggiore, ASU Giuliano Isontina

    Trieste, Italy

  • UOC Ematologia, Ospedale S. Eugenio, ASL Roma 2

    Roma, Italy

  • Unità di Ematologia e TMO, IRCCS Ospedale San Raffaele

    Milan, Italy

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