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ClinicalTrials.gov public records Last synced Sep 8, 2026, 9:16 AM EDT

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Showing 1–3 of 3 matching trials from the live ClinicalTrials.gov search.
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Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Timeline
2018 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Conditions
OTC Deficiency
Interventions
DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more