2 completed. 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Completed 2 |
Follow Mucolipidoses: new trials by email Literature Review of Mucolipidoses trials Research Report on Mucolipidoses
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 |
|---|---|---|---|---|---|---|
| HSCT for High Risk Inherited Inborn Errors Clofarabine, Total body Irradiation, Melphalan - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 38 | 2006-09-01 | 2019-07-11 |
| Stem Cell Transplantation for Hurler Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 41 | 1999-05-01 | 2017-12-28 |
Adrenoleukodystrophy (1) · Gangliosidosis, GM1 (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Mannosidase Deficiency Diseases (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis VI (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1) · Wolman Disease (1)
Bone Diseases (2) · Bone Diseases, Metabolic (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Connective Tissue Diseases (2) · Genetic Diseases, Inborn (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2) · Metabolic Diseases (2) · Mucinoses (2) · Mucopolysaccharidoses (2)
Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Clofarabine (1) · Cyclophosphamide (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Stem Cell Transplantation (1) · Whole-Body Irradiation (1) · thymoglobulin (1)
Masonic Cancer Center, University of Minnesota (2)
United States (2)
Paul Orchard, MD (2)