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New pill shows promise for rare blood disorder

NCT ID NCT04254978

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested an experimental pill called Bomedemstat in 73 people with essential thrombocythemia, a rare blood condition causing too many platelets. Participants had already tried at least one standard treatment without success. The main goals were to check safety and see if the drug could lower platelet counts to a safer level.

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

73 people

The number who actually took part.

Started

Sep 2020

Finished

Mar 2023

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

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: * Diagnosis of Essential Thrombocythemia per World Health Organization (WHO) diagnostic criteria for myeloproliferative neoplasms. * Requires treatment in order to lower platelet count based on patient age over 60 or history of thrombosis. * Have failed at least one standard therapy * Must have discontinued ET therapy at least 1 week (4 weeks for interferon) prior to study drug initiation. Exclusion Criteria: * Has undergone major surgery ≤4 weeks prior to starting study drug or has not recovered from side effects of such surgery. * Unresolved treatment related toxicities from prior therapies (unless resolved to ≤ Grade 1). * Uncontrolled active infection. * Current use of prohibited medications * Known HIV infection or active Hepatitis B or Hepatitis C virus infection * Other hematologic/biochemistry requirements, as per protocol * Use of investigational agent within last 14 days * Pregnant or lactating females

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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.

Contacts and locations

Locations

  • CRIMM; Centro Ricerca e Innovazione delle Malattia Mieloproliferative, Azienda ospedaliera Universitaria Careggi

    Florence, 50139, Italy

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Department of Hematology and Stem Cell Transplantation, West German Cancer Center (WTZ)

    Essen, 45147, Germany

  • Guys and St Thomas Hospital

    London, SE1 9RT, United Kingdom

  • Local Institution

    Jacksonville, Florida, 32209, United States

  • Local Institution

    Ann Arbor, Michigan, 48109, United States

  • Local Institution

    New York, New York, 10021, United States

  • Local Institution

    Durham, North Carolina, 27710, United States

  • Local Institution

    Pittsburgh, Pennsylvania, 15232, United States

  • Local Institution

    Seattle, Washington, 98109, United States

  • Local Institution

    Herston, Brisbane, Australia

  • Local Institution

    Camperdown, New South Wales, Australia

  • Local Institution

    St Leonards, New South Wales, 2065, Australia

  • Local Institution

    Southport, Queensland, Australia

  • Local Institution

    Clayton, Victoria, 3168, Australia

  • Local Institution

    Jena, Germany

  • Local Institution

    Hong Kong, Hong Kong

  • Local Institution

    Alessandria, Italy

  • Local Institution

    Bologna, Italy

  • Local Institution

    Varese, Italy

  • Local Institution

    Auckland, New Zealand

  • Local Institution

    London, NW1, United Kingdom

  • Local Institution

    London, W12, United Kingdom

  • Local Institution

    Oxford, United Kingdom

  • Middlemore Hospital

    Auckland, New Zealand

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

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