12 completed, 3 active not recruiting, 3 no longer available, 2 recruiting. 8 Phase 2, 4 Phase 1, 2 Not applicable, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Active not recruiting 1, Unknown 1 |
| Phase 1 | 4 | Completed 3, Unknown 1 |
| Not applicable | 2 | Completed 2 |
| Phase 3 | 2 | Active not recruiting 1, Completed 1 |
Follow Metachromatic Leukodystrophy: new trials by email Literature Review of Metachromatic Leukodystrophy trials Research Report on Metachromatic Leukodystrophy
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 |
|---|---|---|---|---|---|---|
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| 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 |
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia Blood sample collection - Adrenoleukodystrophy, Adrenomyeloneuropathy | Recruiting | Assistance Publique - Hôpitaux de Paris | 100 | 2021-08-30 | 2025-09-24 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| The Natural History of Metachromatic Leukodystrophy Study (HOME Study) Metachromatic Leukodystrophy | Completed | National Organization for Rare Disorders | 21 | 2020-10-01 | 2025-06-08 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) OTL-200 - Lysosomal Storage Disease, Metachromatic Leukodystrophy | Phase 2 | Completed | Orchard Therapeutics | 10 | 2018-01-25 | 2026-03-16 |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy intracerebral administration of AAVrh.10cuARSA - Metachromatic Leukodystrophy | Phase 1 / Phase 2 | Completed | Institut National de la Santé Et de la Recherche Médicale, France | 5 | 2014-06-01 | 2026-03-02 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Gene Therapy for Metachromatic Leukodystrophy (MLD) OTL-200 Gene Therapy - Lysosomal Storage Disease, Metachromatic Leukodystrophy | Phase 1 / Phase 2 | Completed | Orchard Therapeutics | 20 | 2010-04-09 | 2025-12-05 |
| Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation rhARSA - Metachromatic Leukodystrophy | Phase 2 | Completed | Assistance Publique - Hôpitaux de Paris | 1 | 2008-10-01 | 2026-03-27 |
| The Natural History of Metachromatic Leukodystrophy Metachromatic Leukodystrophy | Withdrawn | University of Pittsburgh | 2008-03-11 | 2024-05-30 | ||
| Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy Warfarin - Metachromatic Leukodystrophy | Not applicable | Completed | The Cooper Health System | 10 | 2007-06-01 | 2011-03-21 |
| 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 |
| Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation I Cell Disease, Fucosidosis | Unknown | Fairview University Medical Center | 10 | 1999-08-01 | 2005-06-24 | |
| Stem Cell Transplantation (SCT) for Genetic Diseases Stem Cell Transplantation - Thrombocytopenia, Metachromatic Leukodystrophy | Not applicable | Completed | National Center for Research Resources (NCRR) | 1995-01-01 | 2005-06-24 |
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Leukodystrophy, Metachromatic (24) · Adrenoleukodystrophy (9) · Leukodystrophy, Globoid Cell (8) · Wolman Disease (7) · Mucopolysaccharidosis I (6) · Mucopolysaccharidosis VI (6) · Gaucher Disease (5) · Metabolism, Inborn Errors (5) · Fucosidosis (4) · Lysosomal Storage Diseases (4) · Mucopolysaccharidosis II (4) · Mucopolysaccharidosis VII (4) · Niemann-Pick Diseases (4) · Peroxisomal Disorders (4) · Rare Diseases (4)
Brain Diseases (24) · Brain Diseases, Metabolic (24) · Brain Diseases, Metabolic, Inborn (24) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Demyelinating Diseases (24) · Hereditary Central Nervous System Demyelinating Diseases (24) · Lipid Metabolism Disorders (24) · Lipid Metabolism, Inborn Errors (24) · Lipidoses (24) · Lysosomal Storage Diseases, Nervous System (24) · Metabolic Diseases (24) · Nutritional and Metabolic Diseases (24) · Sulfatidosis (24) · Central Nervous System Diseases (23) · Nervous System Diseases (23)
Stem Cell Transplantation (3) · AP 1903 reagent (1) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Clofarabine (1) · Cyclophosphamide (1) · Hematopoietic Stem Cell Transplantation (1) · Hydroxyurea (1) · Injections (1) · Melphalan (1) · Regimen B (1) · Warfarin (1) · Whole-Body Irradiation (1)
Masonic Cancer Center, University of Minnesota (3) · Orchard Therapeutics (3) · Assistance Publique - Hôpitaux de Paris (2) · Children's Hospital of Philadelphia (2) · Talaris Therapeutics Inc. (2) · Albert Einstein College of Medicine (1) · Bellicum Pharmaceuticals (1) · Centre Hospitalier Universitaire de Liege (1) · Fairview University Medical Center (1) · Institut National de la Santé Et de la Recherche Médicale, France (1) · National Center for Research Resources (NCRR) (1) · National Organization for Rare Disorders (1) · New York Medical College (1) · RTI International (1) · Shenzhen Second People's Hospital (1)
United States (15) · France (3) · Italy (3) · Belgium (1) · China (1)
Adeline Vanderver, MD (2) · Paul Orchard, MD (2) · Caroline Sevin, MD-PhD (1) · Curt Scharfe, MD, PhD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1) · Jiacai Zhuo (1) · Joanne Kurtzberg, MD (1) · K. Scott Baker (1) · Laurent Servais (1) · Melissa Wasserstein, MD (1)
PMID 21586746, PMID 40267426, PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 25758611, PMID 27289174, PMID 31315600, PMID 32342562, PMID 32749716, PMID 35065785, PMID 35142618
2017 (1), 2019 (1), R01HD073292 (1), U01NS106845 (1), U54NS115052 (1)