Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
Public ClinicalTrials.gov record NCT04990388. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III
Study identification
- NCT ID
- NCT04990388
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Enrollment
- 9 participants
Conditions and interventions
Conditions
Interventions
- Antipyretic Drug
- H1 Blocker Drug
- H2 Blocker Drug
- Placebo Other
- UX053 Biological
Drug · Other · Biological
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 17, 2021
- Primary completion
- Mar 19, 2023
- Completion
- Mar 19, 2023
- Last update posted
- Apr 15, 2024
2021 – 2023
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Irvine | Orange | California | 92868 | — |
| Rare Disease Research | Atlanta | Georgia | 30329 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Texas, Health Science Center of Houston | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04990388, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 15, 2024 · Synced May 4, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04990388 live on ClinicalTrials.gov.