First-in-human Study of UX016 in GNEM
- Status
- NOT_YET_RECRUITING
- Phase
- PHASE1/PHASE2
Clinical Trials Related to Ultragenyx Pharmaceutical Inc
Related studies linked through entity extraction aliases.
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
- Status
- RECRUITING
- Phase
- N/A
Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
- Status
- RECRUITING
- Phase
- PHASE2
Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
- Status
- RECRUITING
- Phase
- PHASE2
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
- Status
- RECRUITING
- Phase
- N/A
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Long-term Extension of GTX-102 in Angelman Syndrome
- Status
- ENROLLING_BY_INVITATION
- Phase
- PHASE3
Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
- Status
- RECRUITING
- Phase
- PHASE1
Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
- Status
- SUSPENDED
- Phase
- PHASE2
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome
- Status
- COMPLETED
- Phase
- N/A
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
- Status
- COMPLETED
- Phase
- PHASE3
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
- Status
- ENROLLING_BY_INVITATION
- Phase
- N/A
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
- Status
- TERMINATED
- Phase
- PHASE2
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
- Status
- TERMINATED
- Phase
- N/A
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
- Status
- COMPLETED
- Phase
- PHASE3