June 2014

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Conditions:   Sickle Cell Disease;   Transfusion Dependent Alpha- or Beta- Thalassemia;   Diamond Blackfan Anemia;   Paroxysmal Nocturnal Hemoglobinuria;   Glanzmann Thrombasthenia;   Severe Congenital Neutropenia;   Shwachman-Diamond Syndrome;   Non-Malignant Hematologic Disorders
Interventions:   Drug: Reduced Toxicity Ablative Regimen;   Drug: Reduced Intensity Preparative Regimen;   Drug: Myeloablative Preparative Regimen
Sponsor:   Masonic Cancer Center, University of Minnesota
Recruiting - verified February 2017

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Conditions:   Mucopolysaccharidosis Disorders;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Alpha-Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Glycoprotein Metabolic Disorders;   Sphingolipidoses;   Recessive Leukodystrophies;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann-Pick B;   Niemann-Pick C Subtype 2;   Sphingomyelin Deficiency;   Peroxisomal Disorders;   Adrenoleukodystrophy With Cerebral Involvement;   Zellweger Syndrome;   Neonatal Adrenoleukodystrophy;   Infantile Refsum Disease;   Acyl-CoA Oxidase D

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Conditions:   Dyskeratosis Congenita;   Aplastic Anemia
Interventions:   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Cyclophosphamide;   Radiation: Total Body Irradiation;   Biological: Stem Cell Transplant;   Drug: Anti-thymocyte globulin
Sponsor:   Masonic Cancer Center, University of Minnesota
Recruiting - verified August 2016