Diamond-Blackfan anemia (DBA) is an inherited disorder that occurs when the bone marrow fails to make red blood cells, leading to anemia and bone marrow failure, with increased cancer risks.
Researchers at the National Institutes of Health want to know if the investigational drug Bitopertin is safe and effective in helping restore blood formation by targeting one of the defects found in patients with DBA. If you or someone you know has been diagnosed with DBA and have not responded to therapy, has relapsed, or is not able to tolerate current treatment; you may be eligible to participate in this study. There is no cost for tests, treatments or procedures, and travel, food and lodging may be provided.
To learn more about this study, contact the NIH Clinical Center Office of Patient Recruitment at 866-444-2214 (TTY users dial 7-1-1), ccopr@nih.gov or visit https://bit.ly/44vPqWD. Ask for study #001528-H.