1 active not recruiting, 1 completed, 1 terminated. 3 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Active not recruiting 1, Completed 1, Terminated 1 |
| Phase 2 | 1 | Completed 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 |
|---|---|---|---|---|---|---|
| OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) OTL-200 - Lysosomal Storage Diseases, Metachromatic Leukodystrophy | Phase 3 | Active not recruiting | Orchard Therapeutics | 6 | 2022-01-17 | 2025-09-05 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
| 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 |
Adrenoleukodystrophy (2) · Fucosidosis (2) · Gangliosidosis, GM1 (2) · Leukodystrophy, Globoid Cell (2) · Lysosomal Storage Diseases (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · Canavan Disease (1) · Gaucher Disease (1) · Metabolism, Inborn Errors (1) · Mucolipidoses (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (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, Nervous System (3) · Metabolic Diseases (3) · Nervous System Diseases (3)
Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Stem Cell Transplantation (1)
Aldagen (1) · Masonic Cancer Center, University of Minnesota (1) · Orchard Therapeutics (1)
United States (2) · Italy (1)
James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Paul Orchard, MD (1)