3 terminated. 3 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Terminated 3 |
| Phase 1 | 1 | Terminated 1 |
Follow Leukodystrophy, Metachromatic: new trials by email Literature Review of Leukodystrophy, Metachromatic trials Research Report on Leukodystrophy, Metachromatic
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 |
|---|---|---|---|---|---|---|
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| 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 |
| Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD HGT-1111 - Late Infantile Metachromatic Leukodystrophy | Phase 2 | Terminated | Shire | 11 | 2008-02-20 | 2021-06-08 |
Leukodystrophy, Globoid Cell (2) · Mucopolysaccharidosis I (2) · alpha-Mannosidosis (2) · Adrenoleukodystrophy (1) · Aspartylglucosaminuria (1) · Fucosidosis (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis III (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Niemann-Pick Disease, Type B (1) · Niemann-Pick Disease, Type C (1) · Niemann-Pick Diseases (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Demyelinating Diseases (3) · Genetic Diseases, Inborn (3) · Hereditary Central Nervous System Demyelinating Diseases (3) · Leukoencephalopathies (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3) · Metabolic Diseases (3)
Stem Cell Transplantation (2) · Alemtuzumab (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1)
Masonic Cancer Center, University of Minnesota (1) · Shire (1) · Talaris Therapeutics Inc. (1)
United States (2) · Denmark (1)
Paul Orchard, MD (1) · Suzanne T Ildstad, MD (1)
2008 (1)