Ultragenyx Pharmaceutical Inc: all 63 studies

Page 1 of 1. Open to participants first (8), then studies with results posted, then the rest; newest registration first within each.

• MPS IIIA Disease Monitoring ProgramNot yet recruitingNCT07855562
• First-in-human Study of UX016 in GNEMRecruitingNCT07511556Phase 1/2
• Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring ProgramRecruitingNCT06636383
• Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAEnrolling by invitationNCT04360265Phase 3
• Expanded Access to MepseviiAvailableNCT03775174
• Expanded Access to TriheptanoinAvailableNCT03773770
• Mucopolysaccharidosis VII Disease Monitoring ProgramRecruitingNCT03604835
• Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHRecruitingNCT02716246Phase 2/3
• Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis ImperfectaTerminatedNCT05312697Phase 2Results posted
• 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 3Results posted
• Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)TerminatedNCT04990388Phase 1/2Results posted
• Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseTerminatedNCT04088734Phase 1/2Results posted
• Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type IaCompletedNCT03970278Results posted
• Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)CompletedNCT03517085Phase 1/2Results posted
• A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804CompletedNCT03118570Phase 2Results posted
• Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) DeficiencyCompletedNCT02991144Phase 1/2Results posted
• Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)TerminatedNCT02960217Phase 3Results posted
• 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 3Results posted
• A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory ImpairmentTerminatedNCT02731690Phase 2Results posted
• Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia BTerminatedNCT02618915Phase 1/2Results posted
• Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)TerminatedNCT02599961Phase 2Results posted
• A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)CompletedNCT02432144Phase 3Results posted
• Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of AgeCompletedNCT02418455Phase 2Results posted
• Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)CompletedNCT02377921Phase 3Results posted
• A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)CompletedNCT02230566Phase 3Results posted
• Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin StudiesCompletedNCT02214160Phase 2Results posted
• Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)CompletedNCT01993186Phase 2Results posted
• A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)CompletedNCT01886378Phase 2Results posted
• An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)CompletedNCT01856218Phase 1/2Results posted
• An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) MyopathyCompletedNCT01830972Phase 2Results posted
• A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)Active, not recruitingNCT07157254Phase 2
• Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaActive, not recruitingNCT06636071Phase 3
• Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)Active, not recruitingNCT06617429Phase 3
• Long-term Extension of GTX-102 in Angelman SyndromeActive, not recruitingNCT06415344Phase 3
• A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAODActive, not recruitingNCT05933200Phase 3
• Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaActive, not recruitingNCT05768854Phase 3
• Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyActive, not recruitingNCT05345171Phase 3
• Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type IIITerminatedNCT05196165
• Setrusumab vs Placebo for Osteogenesis ImperfectaActive, not recruitingNCT05125809Phase 2/3
• Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson DiseaseTerminatedNCT04909346
• A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseActive, not recruitingNCT04884815Phase 1/2
• Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring ProgramTerminatedNCT04812106
• Tumor-induced Osteomalacia Disease Monitoring ProgramActive, not recruitingNCT04783428
• Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) DeficiencyTerminatedNCT04717453
• Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIaCompletedNCT04708015
• Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring ProgramActive, not recruitingNCT04632953
• 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
• A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult SubjectsCompletedNCT04269122
• A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeCompletedNCT04259281Phase 1/2
• Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access ProgramCompletedNCT03768817
• X-linked Hypophosphatemia Disease Monitoring ProgramActive, not recruitingNCT03651505
• Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC DeficiencyActive, not recruitingNCT03636438
• An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis ImperfectaWithdrawnNCT03216486Phase 2
• Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia BCompletedNCT02971969
• Observational Study of Males With Creatine Transporter DeficiencyTerminatedNCT02931682
• Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney DiseaseWithdrawnNCT01806610Phase 2
• GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)CompletedNCT01784679
• A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body MyopathyCompletedNCT01517880Phase 2
• Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis ImperfectaCompletedNCT01417091Phase 2
• 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
• Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)CompletedNCT01359319Phase 1

← Ultragenyx Pharmaceutical Inc overview and focus areas