Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Public ClinicalTrials.gov record NCT05345171. 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 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency
Study identification
- NCT ID
- NCT05345171
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Enrollment
- 32 participants
Conditions and interventions
Conditions
Interventions
- DTX301 Genetic
- Oral Corticosteroids Drug
- Placebo Other
- Placebo for oral corticosteroids Drug
- Sodium Acetate Drug
Genetic · Drug · Other
Eligibility (public fields only)
- Age range
- 12 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, 2022
- Primary completion
- Aug 31, 2027
- Completion
- Feb 28, 2031
- Last update posted
- Feb 16, 2026
2022 – 2031
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California | Los Angeles | California | 90095 | — |
| University of Colorado | Aurora | Colorado | 80045 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | — |
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 12 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05345171, 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 Feb 16, 2026 · Synced May 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05345171 live on ClinicalTrials.gov.