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.
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Dual-Action pill tame bone marrow tumors?
- Can a biologic drug outperform standard therapy for a rare blood cancer?
- New drug candidate targets Hard-to-Treat blood cancers
- Blood clot risk in rare disease: could a higher dose of clopidogrel offer better protection?
- New oral drug could tame overactive bone marrow in rare blood cancers
- New hope for rare blood cancer: targeted drug enters final testing