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Found 4 clinical trials
Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of …

tacrolimus
fludarabine
methotrexate
bone marrow failure
platelet count
  • 0 views
  • 22 Oct, 2022
  • 17 locations
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

preparative regimen
thalassemia
fludarabine
sickle cell anemia
alemtuzumab
  • 231 views
  • 22 Apr, 2022
  • 1 location
Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study

-Blackfan anemia: pure red cell aplasia; elevated red cell adenosine deaminase. Dyskeratosis congenita: dysplastic nails, lacey pigmentation, leukoplakia; marrow failure. Shwachman-Diamond

kostmann's syndrome
bone marrow failure syndromes
dyskeratosis congenita
cancer
pure red cell aplasia
  • 43 views
  • 24 Oct, 2022
  • 2 locations
Diabetes/ Endocrine Surveillance in SDS

Shwachman-Diamond syndrome(SDS) is a rare autosomal recessive disorder involving primarily the Shwachman-Bodian-Diamond syndrome gene located on chromosome 7q11. The gene effects function of the

Accepts healthy volunteers
  • 0 views
  • 11 Jul, 2022
  • 1 location