Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Public ClinicalTrials.gov record NCT02991144. 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, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency
Study identification
- NCT ID
- NCT02991144
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Enrollment
- 16 participants
Conditions and interventions
Conditions
Interventions
- Prophylactic Corticosteroid Taper Regimen Drug
- Reactive Corticosteroid Taper Regimen Drug
- scAAV8OTC Genetic
Drug · Genetic
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
- Jul 30, 2017
- Primary completion
- Dec 15, 2021
- Completion
- Dec 15, 2021
- Last update posted
- Jan 25, 2023
2017 – 2021
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| The Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029-6508 | — |
| University Hospital Cleveland Medical Center/Case Western Reserve University | 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 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02991144, 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 Jan 25, 2023 · Synced May 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02991144 live on ClinicalTrials.gov.