New pill tested for rare blood disorder that defies standard treatment
NCT ID NCT05828108
Summary
This study tested a new oral drug called bitopertin for adults with a rare, inherited blood disorder called Diamond-Blackfan anemia (DBA) that no longer responds to steroid treatment. Fifteen participants took the pill daily for eight months, starting with a low dose that could be increased over time. The main goal was to see if the drug could increase hemoglobin levels or reduce the need for blood transfusions.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for STEROID-REFRACTORY DIAMOND-BLACKFAN ANEMIA (DBA) are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
Explore the condition pages connected to this study.