| OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) (NCT04283227) | Orchard Therapeutics | 2022-01-17 |
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (NCT04925349) | Assistance Publique - Hôpitaux de Paris | 2021-08-30 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (NCT05368038) | Albert Einstein College of Medicine | 2021-05-10 |
| The Natural History of Metachromatic Leukodystrophy Study (HOME Study) (NCT04628364) | National Organization for Rare Disorders | 2020-10-01 |
| Early Check: Expanded Screening in Newborns (NCT03655223) | RTI International | 2018-10-15 |
| A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (NCT03392987) | Orchard Therapeutics | 2018-01-25 |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies (NCT02699190) | Children's Hospital of Philadelphia | 2017-01-06 |
| The Myelin Disorders Biorepository Project (NCT03047369) | Children's Hospital of Philadelphia | 2016-12-08 |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy (NCT02559830) | Shenzhen Second People's Hospital | 2015-01-01 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis (NCT02171104) | Masonic Cancer Center, University of Minnesota | 2014-07-10 |
| Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy (NCT01801709) | Institut National de la Santé Et de la Recherche Médicale, France | 2014-06-01 |
| Human Placental-Derived Stem Cell Transplantation (NCT01586455) | New York Medical College | 2013-04-01 |
| Gene Therapy for Metachromatic Leukodystrophy (MLD) (NCT01560182) | Orchard Therapeutics | 2010-04-09 |
| Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation (NCT01303146) | Assistance Publique - Hôpitaux de Paris | 2008-10-01 |
| The Natural History of Metachromatic Leukodystrophy (NCT00639132) | University of Pittsburgh | 2008-03-11 |
| Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy (NCT00683189) | The Cooper Health System | 2007-06-01 |
| HSCT for High Risk Inherited Inborn Errors (NCT00383448) | Masonic Cancer Center, University of Minnesota | 2006-09-01 |
| Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (NCT00005900) | Fairview University Medical Center | 1999-08-01 |
| Stem Cell Transplantation (SCT) for Genetic Diseases (NCT00004378) | National Center for Research Resources (NCRR) | 1995-01-01 |
| Stem Cell Transplant for Inborn Errors of Metabolism (NCT00176904) | Masonic Cancer Center, University of Minnesota | 1995-01-01 |
| BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study (NCT03639844) | Bellicum Pharmaceuticals | |
| Single Patient Expanded Access Protocol: Metabolic Boost (NCT02021266) | Talaris Therapeutics Inc. | |
| Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use) (NCT02084121) | Talaris Therapeutics Inc. | |