| Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) (NCT05330039) | Emma Marie Caroline Slack | 2021-11-30 |
| Protecting Brains and Saving Futures - the PBSF Protocol (NCT03786497) | Protecting Brains Saving Futures | 2021-01-01 |
| Utilizing Augmented Artificial Intelligence for Aminoacidopathies (NCT05437445) | Aga Khan University Hospital, Pakistan | 2019-08-01 |
| Biomarkers for Inborn Errors of Metabolism (NCT04098198) | CENTOGENE GmbH Rostock | 2019-08-01 |
| Induced Pluripotent Stem Cells for the Development of Novel Drug Therapies for Inborn Errors of Metabolism (iPSC-IEM) (NCT04097275) | CENTOGENE GmbH Rostock | 2019-08-01 |
| Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism (NCT04248062) | University Children's Hospital, Zurich | 2019-08-01 |
| Market Research - Acceptability Study for a New PKU Protein Substitute (NCT04318509) | Dr. Schär AG / SPA | 2019-02-04 |
| North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 (NCT03548779) | University of North Carolina, Chapel Hill | 2018-09-28 |
| Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute (NCT04309331) | Dr. Schär AG / SPA | 2018-07-01 |
| Retrospective Study of Adult Patients With Inborn Errors of Metabolism in Switzerland (NCT03534752) | University of Lausanne | 2018-04-01 |
| Evaluation of PKU Explore (NCT03168399) | Vitaflo International, Ltd | 2017-06-08 |
| Evaluation of PKU Start (NCT03058848) | Vitaflo International, Ltd | 2017-03-06 |
| Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases (NCT02349906) | medac GmbH | 2015-04-01 |
| Maternal Inborn Errors of Metabolism in Pregnancy: A Pregnancy Registry Protocol (NCT02322177) | National Human Genome Research Institute (NHGRI) | 2014-12-19 |
| Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC (NCT02356653) | Children's Hospital of Philadelphia | 2013-12-01 |
| A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD (NCT03911089) | Nutricia Research | 2013-05-28 |
| CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant (NCT01049854) | New York Medical College | 2011-09-01 |
| Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate (NCT01341379) | Children's Hospital of Philadelphia | 2010-12-01 |
| Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases (NCT01003912) | Joanne Kurtzberg, MD | 2009-10-01 |
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders (NCT00744692) | Duke University | 2008-10-01 |
| N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia (NCT00843921) | Mendel Tuchman | 2008-08-01 |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (NCT00654433) | Aldagen | 2008-03-01 |
| Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids (NCT00328159) | Assistance Publique - Hôpitaux de Paris | 2006-06-01 |
| The Early History of Universal Screening for Metabolic Disorders (NCT00309400) | University of Miami | 2006-01-01 |
| Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome (NCT00001596) | William Gahl, M.D. | 2005-09-01 |
| Stem Cell Transplantation (SCT) for Genetic Diseases (NCT00004378) | National Center for Research Resources (NCRR) | 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 | |