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

NCT04965597 · clinicaltrials.gov ↗

PHASE2

Phase

COMPLETED

Status

40

Enrollment

OTHER

Sponsor class

Conditions

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

Interventions

DRUG: Treosulfan
DRUG: Fludarabine Phosphate
DRUG: Tacrolimus
DRUG: Methotrexate
BIOLOGICAL: Lapine T-Lymphocyte Immune Globulin
PROCEDURE: Peripheral Blood Stem Cell Transplantation
PROCEDURE: Allogeneic Bone Marrow Transplantation
OTHER: Quality-of-Life Assessment
PROCEDURE: Echocardiography
PROCEDURE: Multigated Acquisition Scan
PROCEDURE: Bone Marrow Biopsy
PROCEDURE: Bone Marrow Aspiration
PROCEDURE: Biospecimen Collection
PROCEDURE: X-Ray Imaging
PROCEDURE: Computed Tomography

Sponsor

Fred Hutchinson Cancer Center

Collaborators

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