1 terminated. 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 1 | Terminated 1 |
Follow Niemann-Pick Disease Type B: new trials by email Literature Review of Niemann-Pick Disease Type B trials Research Report on Niemann-Pick Disease Type B
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Stem Cell Transplantation, Cyclophosphamide, Campath-1H - Hurler's Syndrome, Maroteaux-Lamy Syndrome | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 18 | 2008-03-01 | 2017-12-28 |
Aspartylglucosaminuria (1) · Fucosidosis (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Niemann-Pick Disease, Type B (1) · Niemann-Pick Disease, Type C (1) · Peroxisomal Disorders (1) · Sphingolipidoses (1) · Wolman Disease (1) · alpha-Mannosidosis (1)
Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Central Nervous System Diseases (1) · Cholesterol Ester Storage Disease (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Connective Tissue Diseases (1) · Demyelinating Diseases (1) · Genetic Diseases, Inborn (1) · Hemic and Lymphatic Diseases (1) · Hereditary Central Nervous System Demyelinating Diseases (1) · Histiocytosis (1) · Histiocytosis, Non-Langerhans-Cell (1) · Infant, Newborn, Diseases (1)
Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Stem Cell Transplantation (1)
Masonic Cancer Center, University of Minnesota (1)
United States (1)
Paul Orchard, MD (1)