4 completed. 4 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 4 |
| Phase 3 | 1 | Completed 1 |
Follow Leukodystrophy, Globoid Cell: new trials by email Literature Review of Leukodystrophy, Globoid Cell trials Research Report on Leukodystrophy, Globoid Cell
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 |
|---|---|---|---|---|---|---|
| MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) MGTA-456 - Inherited Metabolic Disorders (IMD) | Phase 2 | Completed | Magenta Therapeutics, Inc. | 8 | 2018-02-09 | 2021-11-03 |
| Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Campath-1H, Cyclophosphamide, Busulfan - Mucopolysaccharidosis, Hurler Syndrome | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 46 | 2009-12-01 | 2018-02-05 |
| 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 Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Leukodystrophy, Metachromatic (4) · Adrenoleukodystrophy (3) · Fucosidosis (2) · Gangliosidosis, GM1 (2) · Mucopolysaccharidosis I (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Wolman Disease (2) · Aspartylglucosaminuria (1) · Gaucher Disease (1) · Metabolism, Inborn Errors (1) · Mucolipidoses (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis II (1)
Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Carbohydrate Metabolism, Inborn Errors (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Demyelinating Diseases (4) · Genetic Diseases, Inborn (4) · Hereditary Central Nervous System Demyelinating Diseases (4) · Leukoencephalopathies (4) · Lipid Metabolism Disorders (4) · Lipid Metabolism, Inborn Errors (4) · Lipidoses (4) · Lysosomal Storage Diseases (4) · Lysosomal Storage Diseases, Nervous System (4)
Alemtuzumab (2) · Busulfan (2) · Cyclophosphamide (2) · Stem Cell Transplantation (2) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Clofarabine (1) · Cyclosporine (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Melphalan (1) · Mycophenolic Acid (1) · Whole-Body Irradiation (1)
Masonic Cancer Center, University of Minnesota (3) · Magenta Therapeutics, Inc. (1)
United States (4)
Paul Orchard, MD (3)