| Active Not Recruiting | Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy NCT05003648 | Massachusetts General Hospital | Phase 4 |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Recruiting | Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adu NCT04925349 | Assistance Publique - Hôpitaux de Paris | — |
| Completed | Plasma Exchange With Albumin in AMN Patients NCT04303416 | Pujol, Aurora, M.D. | Phase 2 / Phase 3 |
| Recruiting | Adrenoleukodystrophy National Registry Study NCT03789721 | Masonic Cancer Center, University of Minnesota | — |
| Withdrawn | Early Diagnosis Of Childhood Cerebral ALD NCT02948062 | Masonic Cancer Center, University of Minnesota | — |
| Completed | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation NCT03513328 | University of Florida | Phase 1 / Phase 2 |
| Completed | A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. NCT03231878 | Minoryx Therapeutics, S.L. | Phase 2 / Phase 3 |
| Unknown | Precision Exercise in Children With Malignant Hemopathies NCT04090268 | University of Milano Bicocca | N/A |
| Completed | LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies NCT02699190 | Children's Hospital of Philadelphia | — |
| Recruiting | The Myelin Disorders Biorepository Project NCT03047369 | Children's Hospital of Philadelphia | — |
| Completed | Newborn Screening for Adrenoleukodystrophy NCT02952482 | National Taiwan University Hospital | — |
| Unknown | Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy NCT02559830 | Shenzhen Second People's Hospital | Phase 1 / Phase 2 |
| Completed | MD1003-AMN MD1003 in Adrenomyeloneuropathy NCT02961803 | MedDay Pharmaceuticals SA | Phase 2 / Phase 3 |
| Recruiting | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte- NCT02254863 | Joanne Kurtzberg, MD | Phase 1 |
| Completed | Human Placental-Derived Stem Cell Transplantation NCT01586455 | New York Medical College | Phase 1 |
| Completed | HSCT for High Risk Inherited Inborn Errors NCT00383448 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Unknown | Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children NCT00278044 | Far Eastern Memorial Hospital | N/A |
| Terminated | A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy NCT00545597 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Phase 3 |
| Unknown | Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoie NCT00005900 | Fairview University Medical Center | — |
| Completed | Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy NCT00004450 | FDA Office of Orphan Products Development | N/A |
| Terminated | Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy NCT00004418 | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Phase 2 |
| Completed | Stem Cell Transplant for Inborn Errors of Metabolism NCT00176904 | Masonic Cancer Center, University of Minnesota | Phase 2 / Phase 3 |
| Completed | Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid NCT00007020 | Mirum Pharmaceuticals, Inc. | Phase 3 |
| Terminated | Study of Bile Acids in Patients With Peroxisomal Disorders NCT00004442 | University of Cincinnati | N/A |