Clinical Trial: Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia
Study Status: Completed
Recruit Status: RECRUITING
Condition: Steroid-refractory Diamond-Blackfan Anemia (DBA)
Study Type: INTERVENTIONAL
Official Title: A Phase I/II, Intra-Patient Dose-Escalation Study of the Selective GlyT1 Inhibitor, Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia.
Brief Summary:
Background:Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow.People with DBA have chronic anemia that can be severe.Many must have frequent transfusions of red blood cells.Current treatments for DBA all have risks of serious side effects.Better treatments are needed.Objective:To test a new drug (bitopertin) in people with DBA.Eligibility:People aged 18 or older with DBA.Design:Participants will be screened.They will have a physical exam; they will have blood tests and a test of…
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