Clinical Trial Update: Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) [Phase 2]

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Summary

This update reports the completion of a Phase 2 study evaluating a treosulfan-based conditioning regimen before bone marrow or blood stem cell transplantation f…

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Diseases / indications

• Congenital Amegakaryocytic Thrombocytopenia
• Diamond-blackfan Anemia
• Hematologic Neoplasm with Germline Gata2 Mutation
• Hematologic Neoplasm with Germline Samd9 Mutation
• Hematologic Neoplasm with Germline Samd9l Mutation
• Hereditary Sideroblastic Anemia
• Paroxysmal Nocturnal Hemoglobinuria
• Shwachman-diamond Syndrome

Drugs mentioned

• Treosulfan (treosulfan)

Snippet

Sponsor: Fred Hutchinson Cancer Center | Conditions: Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia | Phase 2 | Status: COMPLETED