New pill shows promise for rare blood disorder
NCT ID NCT04254978
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
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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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73 people
The number who actually took part.
- Started
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Sep 2020
- Finished
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Mar 2023
- Lead sponsor
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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
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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: * 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
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CRIMM; Centro Ricerca e Innovazione delle Malattia Mieloproliferative, Azienda ospedaliera Universitaria Careggi
Florence, 50139, Italy
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Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Department of Hematology and Stem Cell Transplantation, West German Cancer Center (WTZ)
Essen, 45147, Germany
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Guys and St Thomas Hospital
London, SE1 9RT, United Kingdom
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Local Institution
Jacksonville, Florida, 32209, United States
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Local Institution
Ann Arbor, Michigan, 48109, United States
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Local Institution
New York, New York, 10021, United States
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Local Institution
Durham, North Carolina, 27710, United States
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Local Institution
Pittsburgh, Pennsylvania, 15232, United States
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Local Institution
Seattle, Washington, 98109, United States
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Local Institution
Herston, Brisbane, Australia
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Local Institution
Camperdown, New South Wales, Australia
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Local Institution
St Leonards, New South Wales, 2065, Australia
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Local Institution
Southport, Queensland, Australia
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Local Institution
Clayton, Victoria, 3168, Australia
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Local Institution
Jena, Germany
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Local Institution
Hong Kong, Hong Kong
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Local Institution
Alessandria, Italy
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Local Institution
Bologna, Italy
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Local Institution
Varese, Italy
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Local Institution
Auckland, New Zealand
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Local Institution
London, NW1, United Kingdom
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Local Institution
London, W12, United Kingdom
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Local Institution
Oxford, United Kingdom
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Middlemore Hospital
Auckland, New Zealand
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Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
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Other studies related to the condition(s) this trial covers.
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