New hope for rare blood disorder: experimental pill reduces need for transfusions
NCT ID NCT05828108
First seen Nov 01, 2025 · Last updated May 09, 2026 · Updated 22 times
Summary
This study tested a new daily pill, bitopertin, in 15 adults with Diamond-Blackfan anemia (DBA) that no longer responds to steroids. DBA is a rare inherited condition causing severe anemia, often requiring frequent blood transfusions. The goal was to see if bitopertin could boost red blood cell production and reduce or eliminate the need for transfusions over 8 months, with an option to continue for up to 3 more years if it worked.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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