21 completed, 7 recruiting, 6 terminated, 3 active not recruiting. 19 Phase 2, 11 Phase 1, 6 Not applicable, 3 Phase 3.
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 |
|---|---|---|---|---|---|---|
| Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD Lentiviral TYF-ARSA correction of patient's autologous HSCs - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-06-01 | 2026-09-01 |
| Direct Lentiviral Injection Gene Therapy for MLD Intrathecal and intravenous LV gene therapy - Metachromatic Leukodystrophy (MLD) | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 10 | 2027-05-31 | 2026-09-01 |
| A Study to Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain No Intervention - Metachromatic Leukodystrophy (MLD) | Withdrawn | Takeda | 2023-11-30 | 2024-01-12 | ||
| 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 | |
| Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. collection of data - Inherited Disease, Rare Diseases | Recruiting | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | 100 | 2021-03-01 | 2024-11-19 | |
| The Natural History of Metachromatic Leukodystrophy Study (HOME Study) Metachromatic Leukodystrophy | Completed | National Organization for Rare Disorders | 21 | 2020-10-01 | 2025-06-08 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| Comparison of Acute Effect of Different Manual Techniques on Muscle Fascia Stiffness Exercise + Manual Lymphatic Drainage, Exercise + Swedish massage - Manual Lymphatic Drainage, Swedish Massage | Unknown | Marmara University | 30 | 2020-04-01 | 2020-03-27 | |
| A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 Safety and efficacy assessments - Inherited Metabolic Disorders (IMD) | Terminated | Magenta Therapeutics, Inc. | 3 | 2019-05-21 | 2021-03-15 | |
| A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy SHP611 - Metachromatic Leukodystrophy (MLD) | Phase 2 | Completed | Shire | 36 | 2019-05-13 | 2026-07-10 |
| 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 | |
| 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 |
| 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 |
| Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Umbilical cord blood transplantation with HSC835 - Inherited Metabolic Disorders IMD | Phase 1 / Phase 2 | Withdrawn | Novartis Pharmaceuticals | 2017-10-10 | 2018-11-28 | |
| 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 | |
| Natural History Study of Children With Metachromatic Leukodystrophy Lipid Metabolism Disorders, Metachromatic Leukodystrophy (MLD) | Terminated | Shire | 1 | 2015-11-02 | 2021-03-17 |
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Leukodystrophy, Globoid Cell (19) · Adrenoleukodystrophy (17) · Mucopolysaccharidosis I (15) · Mucopolysaccharidosis II (9) · Mucopolysaccharidosis VI (9) · Wolman Disease (9) · Metabolism, Inborn Errors (8) · alpha-Mannosidosis (8) · Fucosidosis (7) · Gaucher Disease (7) · Lysosomal Storage Diseases (7) · Peroxisomal Disorders (7) · Sphingolipidoses (7) · Mucopolysaccharidoses (6)
Brain Diseases, Metabolic (49) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Lipid Metabolism, Inborn Errors (49) · Lipidoses (49) · Lysosomal Storage Diseases, Nervous System (49) · Nutritional and Metabolic Diseases (49) · Brain Diseases (48) · Hereditary Central Nervous System Demyelinating Diseases (48) · Lipid Metabolism Disorders (48) · Sulfatidosis (48) · Brain Diseases, Metabolic, Inborn (47) · Central Nervous System Diseases (47) · Demyelinating Diseases (47) · Metabolic Diseases (47) · Leukoencephalopathies (46)
Stem Cell Transplantation (6) · Alemtuzumab (5) · Busulfan (3) · Cyclophosphamide (3) · Melphalan (3) · Bone Marrow Transplantation (2) · Clofarabine (2) · Cyclosporine (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2) · Mycophenolic Acid (2) · Whole-Body Irradiation (2) · AP 1903 reagent (1) · Antilymphocyte Serum (1) · Data Collection (1)
Shire (7) · Masonic Cancer Center, University of Minnesota (6) · Assistance Publique - Hôpitaux de Paris (3) · Orchard Therapeutics (3) · Talaris Therapeutics Inc. (3) · Children's Hospital of Philadelphia (2) · Magenta Therapeutics, Inc. (2) · Paul Szabolcs (2) · Shenzhen Geno-Immune Medical Institute (2) · Albert Einstein College of Medicine (1) · Aldagen (1) · Bellicum Pharmaceuticals (1) · Centre Hospitalier Universitaire de Liege (1) · Fairview University Medical Center (1) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1)
United States (28) · France (8) · Denmark (6) · Italy (5) · Germany (4) · Japan (4) · Belgium (3) · Brazil (3) · China (3) · Argentina (2) · Australia (2) · Canada (2) · Spain (2) · Czechia (1) · Greece (1)
Paul Orchard, MD (4) · Joanne Kurtzberg, MD (3) · Adeline Vanderver, MD (2) · Paul Szabolcs, MD (2) · Suzanne T Ildstad, MD (2) · Caroline Sevin, MD, PhD (1) · Caroline Sevin, MD-PhD (1) · Curt Scharfe, MD, PhD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1)
PMID 21586746, PMID 33332761, PMID 40267426, PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770, PMID 25487283, PMID 25758611, PMID 26000324, PMID 27289174, PMID 31315600, PMID 32342562, PMID 32634238, PMID 32749716, PMID 32750127, PMID 32792226, PMID 35065785, PMID 35142618, PMID 37782421, PMID 41667276
2008 (1), 2017 (1), 2018 (1), 2019 (1), R01HD073292 (1), U01NS106845 (1), U54NS115052 (1)