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First received: April 22, 2023

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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