Setrusumab vs Placebo for Osteogenesis Imperfecta
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2/PHASE3
Clinical Trials Related to Ultragenyx Pharmaceutical Inc
Related studies linked through entity extraction aliases.
Cardiovascular Structure and Function in the Mucopolysaccharidoses
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
- Status
- TERMINATED
- Phase
- N/A
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1/PHASE2
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
- Status
- TERMINATED
- Phase
- N/A
Tumor-induced Osteomalacia Disease Monitoring Program
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
- Status
- TERMINATED
- Phase
- N/A
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
- Status
- COMPLETED
- Phase
- N/A
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- N/A
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
- Status
- COMPLETED
- Phase
- N/A
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
- Status
- COMPLETED
- Phase
- N/A
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
- Status
- ENROLLING_BY_INVITATION
- Phase
- PHASE3
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
- Status
- COMPLETED
- Phase
- PHASE4
Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
- Status
- COMPLETED
- Phase
- N/A
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
- Status
- COMPLETED
- Phase
- PHASE1/PHASE2
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
- Status
- TERMINATED
- Phase
- PHASE1/PHASE2
Burosumab for CSHS
- Status
- UNKNOWN
- Phase
- EARLY_PHASE1
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
- Status
- COMPLETED
- Phase
- N/A