| First-in-human Study of UX016 in GNEM (NCT07511556) | Ultragenyx Pharmaceutical Inc | 2026-10-01 |
| A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) (NCT07157254) | Ultragenyx Pharmaceutical Inc | 2025-10-13 |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) (NCT06617429) | Ultragenyx Pharmaceutical Inc | 2024-12-03 |
| Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program (NCT06636383) | Ultragenyx Pharmaceutical Inc | 2024-11-04 |
| Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta (NCT06636071) | Ultragenyx Pharmaceutical Inc | 2024-10-25 |
| Long-term Extension of GTX-102 in Angelman Syndrome (NCT06415344) | Ultragenyx Pharmaceutical Inc | 2024-07-31 |
| Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta (NCT05768854) | Ultragenyx Pharmaceutical Inc | 2023-06-14 |
| A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD (NCT05933200) | Ultragenyx Pharmaceutical Inc | 2023-02-28 |
| Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency (NCT05345171) | Ultragenyx Pharmaceutical Inc | 2022-10-18 |
| Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program (NCT04812106) | Ultragenyx Pharmaceutical Inc | 2022-07-25 |
| Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III (NCT05196165) | Ultragenyx Pharmaceutical Inc | 2022-05-20 |
| Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta (NCT05312697) | Ultragenyx Pharmaceutical Inc | 2022-04-28 |
| Setrusumab vs Placebo for Osteogenesis Imperfecta (NCT05125809) | Ultragenyx Pharmaceutical Inc | 2022-02-21 |
| Tumor-induced Osteomalacia Disease Monitoring Program (NCT04783428) | Ultragenyx Pharmaceutical Inc | 2022-01-31 |
| Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (NCT04632953) | Ultragenyx Pharmaceutical Inc | 2021-11-30 |
| Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease (NCT04909346) | Ultragenyx Pharmaceutical Inc | 2021-06-23 |
| Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease (NCT04531189) | Ultragenyx Pharmaceutical Inc | 2020-12-11 |
| Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III) (NCT04574830) | Ultragenyx Pharmaceutical Inc | 2020-11-23 |
| Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa (NCT04708015) | Ultragenyx Pharmaceutical Inc | 2020-11-05 |
| Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency (NCT04717453) | Ultragenyx Pharmaceutical Inc | 2020-10-06 |
| Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA (NCT04360265) | Ultragenyx Pharmaceutical Inc | 2020-09-28 |
| A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (NCT04259281) | Ultragenyx Pharmaceutical Inc | 2020-02-24 |
| Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease (NCT04088734) | Ultragenyx Pharmaceutical Inc | 2019-09-18 |
| A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects (NCT04269122) | Ultragenyx Pharmaceutical Inc | 2019-08-02 |
| Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia (NCT03970278) | Ultragenyx Pharmaceutical Inc | 2019-07-15 |
| Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program (NCT03768817) | Ultragenyx Pharmaceutical Inc | 2019-01-30 |
| Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency (NCT03636438) | Ultragenyx Pharmaceutical Inc | 2018-08-30 |
| X-linked Hypophosphatemia Disease Monitoring Program (NCT03651505) | Ultragenyx Pharmaceutical Inc | 2018-07-16 |
| Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) (NCT03517085) | Ultragenyx Pharmaceutical Inc | 2018-05-18 |
| Mucopolysaccharidosis VII Disease Monitoring Program (NCT03604835) | Ultragenyx Pharmaceutical Inc | 2018-01-29 |
| A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (NCT03118570) | Ultragenyx Pharmaceutical Inc | 2017-09-11 |