4 completed, 2 not yet recruiting. 6 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 6 | Completed 4, Not yet recruiting 2 |
Follow Sulfatidosis: new trials by email Literature Review of Sulfatidosis trials Research Report on Sulfatidosis
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 | 2026-11-02 |
| 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 | 2026-11-02 |
| Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Campath-1H, Clofarabine, Melphalan - Lysosomal Storage Disease, Peroxisomal Disorder | Not applicable | Completed | Masonic Cancer Center, University of Minnesota | 3 | 2012-07-11 | 2017-12-05 |
| Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy High-field MRI (3 Teslas) - Late Infantile Metachromatic Leukodystrophy | Not applicable | Completed | Assistance Publique - Hôpitaux de Paris | 29 | 2010-11-01 | 2025-11-20 |
| 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 |
| 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 |
Leukodystrophy, Metachromatic (6) · Adrenoleukodystrophy (1) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Demyelinating Diseases (1) · Fanconi Anemia (1) · Gangliosidosis, GM1 (1) · Genetic Diseases, Inborn (1) · Hematologic Diseases (1) · Leukodystrophy, Globoid Cell (1) · Lysosomal Storage Diseases (1) · Metabolism, Inborn Errors (1) · Mucolipidoses (1) · Mucopolysaccharidosis III (1) · Peroxisomal Disorders (1)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Hereditary Central Nervous System Demyelinating Diseases (6) · Leukoencephalopathies (6) · Lipid Metabolism Disorders (6) · Lipid Metabolism, Inborn Errors (6) · Lipidoses (6) · Lysosomal Storage Diseases, Nervous System (6) · Metabolic Diseases (6) · Nervous System Diseases (6) · Nutritional and Metabolic Diseases (6)
Alemtuzumab (1) · Clofarabine (1) · Cyclosporine (1) · Hematopoietic Stem Cell Transplantation (1) · Melphalan (1) · Mycophenolic Acid (1) · Radiotherapy, Intensity-Modulated (1) · Stem Cell Transplantation (1) · Warfarin (1) · Whole-Body Irradiation (1)
Shenzhen Geno-Immune Medical Institute (2) · Assistance Publique - Hôpitaux de Paris (1) · Masonic Cancer Center, University of Minnesota (1) · National Center for Research Resources (NCRR) (1) · The Cooper Health System (1)
United States (3) · China (2) · France (1)
Caroline Sevin, MD, PhD (1) · Lung-Ji Chang, Ph.D (1) · Mitra Assadi, M.D. (1) · Paola Leone, Ph.D. (1) · Stephen A. Feig (1) · Weston Miller, MD (1)