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Ultragenyx Pharmaceutical Inc clinical trials
The registry lists 63 studies whose lead sponsor is Ultragenyx Pharmaceutical Inc. 8 are recruiting or about to start. Per ClinicalTrials.gov, last updated Oct 20, 2026.
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Each name exactly as ClinicalTrials.gov lists it, and how it relates to Ultragenyx Pharmaceutical Inc.
- Ultragenyx Pharmaceutical Inc63 studiesSame company
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Studies
Recruiting first, then most recently updated. Showing 50 of 63. Browse all 63 →
- Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring ProgramRecruitingNCT06636383
- First-in-human Study of UX016 in GNEMRecruitingNCT07511556Phase 1/2
- MPS IIIA Disease Monitoring ProgramNot yet recruitingNCT07855562
- Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHRecruitingNCT02716246Phase 2/3
- Mucopolysaccharidosis VII Disease Monitoring ProgramRecruitingNCT03604835
- Expanded Access to TriheptanoinAvailableNCT03773770
- Expanded Access to MepseviiAvailableNCT03775174
- Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAEnrolling by invitationNCT04360265Phase 3
- Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaActive, not recruitingNCT05768854Phase 3
- Setrusumab vs Placebo for Osteogenesis ImperfectaActive, not recruitingNCT05125809Phase 2/3
- A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)CompletedNCT05139316Phase 3
- Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaActive, not recruitingNCT06636071Phase 3
- A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseActive, not recruitingNCT04884815Phase 1/2
- A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAODActive, not recruitingNCT05933200Phase 3
- Long-term Extension of GTX-102 in Angelman SyndromeActive, not recruitingNCT06415344Phase 3
- Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyActive, not recruitingNCT05345171Phase 3
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)Active, not recruitingNCT07157254Phase 2
- X-linked Hypophosphatemia Disease Monitoring ProgramActive, not recruitingNCT03651505
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)Active, not recruitingNCT06617429Phase 3
- Tumor-induced Osteomalacia Disease Monitoring ProgramActive, not recruitingNCT04783428
- Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring ProgramActive, not recruitingNCT04632953
- Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC DeficiencyActive, not recruitingNCT03636438
- Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type IaCompletedNCT03970278
- A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeCompletedNCT04259281Phase 1/2
- Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis ImperfectaTerminatedNCT05312697Phase 2
- Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIaCompletedNCT04708015
- Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)TerminatedNCT04990388Phase 1/2
- Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type IIITerminatedNCT05196165
- Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin StudiesCompletedNCT02214160Phase 2
- Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseTerminatedNCT04088734Phase 1/2
- A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804CompletedNCT03118570Phase 2
- An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis ImperfectaWithdrawnNCT03216486Phase 2
- Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson DiseaseTerminatedNCT04909346
- Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)TerminatedNCT02736188Phase 3
- Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) DeficiencyCompletedNCT02991144Phase 1/2
- Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring ProgramTerminatedNCT04812106
- Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)CompletedNCT03517085Phase 1/2
- Observational Study of Males With Creatine Transporter DeficiencyTerminatedNCT02931682
- Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)CompletedNCT01406977Phase 2
- Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral DensityCompletedNCT01406548Phase 2
- Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney DiseaseWithdrawnNCT01806610Phase 2
- Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)CompletedNCT04574830
- Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson DiseaseCompletedNCT04531189
- Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) DeficiencyTerminatedNCT04717453
- Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia BCompletedNCT02971969
- Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis ImperfectaCompletedNCT01417091Phase 2
- A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)CompletedNCT01886378Phase 2
- Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access ProgramCompletedNCT03768817
- A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)CompletedNCT02432144Phase 3
- A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)CompletedNCT02230566Phase 3