Ultragenyx Pharmaceutical
Trial Pipeline
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
NCT06636383
Mucopolysaccharidosis VII Disease Monitoring Program
NCT03604835
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT02716246
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
NCT05768854
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
NCT05345171
Setrusumab vs Placebo for Osteogenesis Imperfecta
NCT05125809
Tumor-induced Osteomalacia Disease Monitoring Program
NCT04783428
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
NCT04632953
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT04884815
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
NCT03636438
X-linked Hypophosphatemia Disease Monitoring Program
NCT03651505
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
NCT05139316
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
NCT04269122
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
NCT03970278
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
NCT03768817
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
NCT03517085
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
NCT02991144
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
NCT02971969
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
NCT01886378
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 Myopathy
NCT01517880
Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
NCT01417091
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 3 |
| Phase 2 | 6 |
| Phase 3 | 4 |
Therapeutic Areas
What the Pipeline for Ultragenyx Pharmaceutical Shows
According to the ClinicalTrials.gov registry, Ultragenyx Pharmaceutical is linked to 23 US clinical trials across every stage of research activity. Of those, 4 studies are currently recruiting — about 17% of the sponsor's indexed portfolio — and 10 are already marked complete, representing roughly 43% of the total. Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
The phase mix for Ultragenyx Pharmaceutical reports 4 late-stage studies (Phase 3 and Phase 4 combined) and 9 earlier-phase studies (Phase 1 and Phase 2). A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work — safety, dosing, and early signal detection — and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
The top therapeutic focus area indexed for Ultragenyx Pharmaceutical is Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) with 3 linked trials, and 9 other condition areas appear in the top list above. That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice — verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology — how this data is sourced, computed, and verified.