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

Trying treosulfan before transplant for bone marrow failure study.

Recruiting
1-49 years
All
Phase 2

This study looks at a new way to prepare patients for a blood or bone marrow transplant. The plan uses three drugs: **treosulfan**, **fludarabine**, and **rabbit antithymocyte globulin (rATG)**. Treosulfan is a chemotherapy drug that helps stop unwanted cell growth. Fludarabine works by blocking enzymes the body needs to grow cancer cells. **rATG** helps lower the body’s immune response, which can help the bone marrow work better. Together, these drugs may cause fewer problems after a transplant.

  • The study lasts for about one year and includes regular follow-ups after the transplant.
  • Patients may take part if they are older than 1 year but younger than 50 years.
  • Patients receive treosulfan and fludarabine by IV (intravenous) and undergo several tests.

There are some risks and not everyone can join; for example, those with prior transplants or certain infections are not eligible. Compensation is not mentioned in the study details. Participants will need to sign a consent form before starting the treatment. You might want to discuss with a doctor if this is the right choice for you.

Study details
    Bone Marrow Failure Syndrome
    Congenital Amegakaryocytic Thrombocytopenia
    Diamond-Blackfan Anemia
    Hereditary Sideroblastic Anemia
    Paroxysmal Nocturnal Hemoglobinuria
    Shwachman-Diamond Syndrome
    Hematologic Neoplasm With Germline GATA2 Mutation
    Hematologic Neoplasm With Germline SAMD9 Mutation
    Hematologic Neoplasm With Germline SAMD9L Mutation

NCT04965597

Fred Hutchinson Cancer Center

12 October 2024

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