Sponsor or institution
Clinical trials and research at Ultragenyx Pharmaceutical Inc
As of November 12, 2026, Find a Trial lists 4 clinical trials run by Ultragenyx Pharmaceutical Inc that are recruiting, at sites in 13 countries (most in United States (4), Spain (3), Brazil (2)). By phase: Phase 2/3 (1), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 4Clinical trials
- 13Countries
Newest trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| First-in-human test of UX016 for GNEM | Phase 1/2 | Orange, California and 1 other locations | Recruiting | Apr 6, 2026 |
| Monitoring program for patients with Glycogen Storage Disease Type Ia | — | Orange, California and 18 other locations | Recruiting | Oct 10, 2024 |
| Monitoring program for Mucopolysaccharidosis VII disease | — | Orange, California and 13 other locations | Recruiting | Jul 30, 2018 |
| Gene therapy for treating MPS IIIA | Phase 2/3 | Columbus, Ohio and 4 other locations | Recruiting | Mar 23, 2016 |
All recruiting trials (4)
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First-in-human test of UX016 for GNEM
This study will test whether UX016 is safe and can improve muscle strength in adults with GNE myopathy.
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Monitoring program for patients with Glycogen Storage Disease Type Ia
This study is tracking the long-term health and safety of a treatment called DTX401 in people with Glycogen Storage Disease Type Ia to see how it helps them over at least 10 years.
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Monitoring program for Mucopolysaccharidosis VII disease
This study is trying to understand how Mucopolysaccharidosis VII affects people over time and see if a treatment called vestronidase alfa is safe and effective for both treated and untreated patients.
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Gene therapy for treating MPS IIIA
This study is testing a new gene therapy called UX111 to see if it can help people with MPS IIIA, a serious genetic disorder.
Quick answers
How many clinical trials run by Ultragenyx Pharmaceutical Inc are recruiting right now?
4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 13 countries. The most sites are in United States (4 trials), Spain (3 trials), Brazil (2 trials), Germany (2 trials), Netherlands (2 trials). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 2/3: 1, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.