Ultragenyx Pharmaceutical is the lead sponsor of 16 current clinical trials in our USA and UK registers. 4 are recruiting patients now. The most common phase is Observational. The drugs named most often in its trials are Apazunersen and Setrusumab. The most-studied condition is Angelman Syndrome.
| Study | Phase | Status | Start | Registers |
|---|---|---|---|---|
|
First-in-human Study of UX016 in GNEM
NCT07511556 · GNE Myopathy
|
Phase 1/2 | Recruiting | 2026-10 | USA |
|
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT02716246 · ClinicalTrials.gov ↗ · MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
|
Phase 2/3 | Recruiting | 2016-04 | USA |
|
Mucopolysaccharidosis VII Disease Monitoring Program
NCT03604835 · ClinicalTrials.gov ↗ · Mucopolysaccharidosis VII, MPS VII, MPS 7
|
Observational | Recruiting | 2018-01 | USA |
|
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
NCT06636383 · ClinicalTrials.gov ↗ · Glycogen Storage Disease Type Ia
|
Observational | Recruiting | 2024-11 | USA |
|
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
NCT04360265 · ClinicalTrials.gov ↗ · Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome
|
Phase 3 | By invitation | 2020-09 | USA |
|
Long-term Extension of GTX-102 in Angelman Syndrome
NCT06415344 · Angelman Syndrome
Drug: Apazunersen |
Phase 3 | Active, closed | 2024-07 | UK, USA |
|
X-linked Hypophosphatemia Disease Monitoring Program
NCT03651505 · X-linked Hypophosphatemia, Hypophosphatemic Rickets
|
Observational | Active, closed | 2018-07 | USA |
|
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
NCT05768854 · Osteogenesis Imperfecta
Drug: Setrusumab |
Phase 3 | Active, closed | 2023-06 | USA |
|
Setrusumab vs Placebo for Osteogenesis Imperfecta
NCT05125809 · ClinicalTrials.gov ↗ · Osteogenesis Imperfecta
Drug: Setrusumab |
Phase 2/3 | Active, closed | 2022-02 | UK, USA |
|
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429 · Angelman Syndrome
Drug: Apazunersen |
Phase 3 | Active, closed | 2024-12 | USA |
|
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT04884815 · ClinicalTrials.gov ↗ · Wilson Disease
|
Phase 1/2 | Active, closed | 2021-09 | UK, USA |
|
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
NCT03636438 · ClinicalTrials.gov ↗ · Ornithine Transcarbamylase (OTC) Deficiency
|
Observational | Active, closed | 2018-08 | UK, USA |
|
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254 · ClinicalTrials.gov ↗ · Angelman Syndrome
Drug: Apazunersen |
Phase 2 | Active, closed | 2025-10 | UK, USA |
|
Tumor-induced Osteomalacia Disease Monitoring Program
NCT04783428 · ClinicalTrials.gov ↗ · Tumor-induced Osteomalacia (TIO)
|
Observational | Active, closed | 2022-01 | USA |
|
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
NCT05345171 · ClinicalTrials.gov ↗ · OTC Deficiency
|
Phase 3 | Active, closed | 2022-10 | USA |
|
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
NCT04632953 · ClinicalTrials.gov ↗ · Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
|
Observational | Active, closed | 2021-11 | USA |
Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.
Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.
| Register | Trials | Recruiting |
|---|---|---|
| Clinical trials in USA | 16 | 4 |
| Clinical trials in UK | 5 | 0 |
A trial with sites in more than one country appears in each of those registers.
| Sponsor | Trials |
|---|---|
| Ionis Pharmaceuticals | 2 |
Sponsors with the most current trials registered for this condition.
Ultragenyx Pharmaceutical is the lead sponsor of 16 current trials on ClinicalTrials.gov in our USA and UK registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.
Yes. 4 of its 16 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Its current trials name 2 drugs and biologicals as interventions. The most frequent are Apazunersen (3) and Setrusumab (2). The number in brackets is how many of its trials name each drug.
Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.
Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.
This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.