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Phase 2 × Congenital amegakaryocytic thrombocytopenia × treosulfan × Clear all

NCT04965597 2026-03-09

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Congenital amegakaryocytic thrombocytopenia, Bone Marrow Failure Disorders, Shwachman-Diamond Syndrome, Anemia, Hypochromic, Hemoglobinuria, Paroxysmal, Anemia, Diamond-Blackfan