Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
NCT05345171 · View on ClinicalTrials.gov ↗
Study Summary
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
Conditions Studied
Interventions
- OTHER Placebo
- GENETIC DTX301
- DRUG Oral Corticosteroids
- DRUG Placebo for oral corticosteroids
- DRUG Sodium Acetate
Study Locations (16)
Other
- Hospital Italiano de Buenos Aires — Buenos Aires
- Clinica Universitaria Reina Fabiola — Córdoba
- Hospital de Clinicas de Porto Alegre — Porto Alegre
- Hopital Femme Mere Enfant — Bron
- Necker-Enfants Maladas Hospital — Paris
- Universitatsklinikum Heidelberg — Heidelberg
- Kumamoto University Hospital — Kumamoto
- Fujita Health University Hospital — Toyoake
- Erasmus Universitair Medisch Centrum Rotterrdam — Rotterdam
- Centro Hospitalar Universitario de Sao Joao — Porto
- Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca — Barcelona
California
- University of California — Los Angeles
Colorado
- University of Colorado — Aurora
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Ohio
- University Hospitals Cleveland Medical Center — Cleveland
Ontario
- The Hospital for Sick Children — Toronto
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2022-10-18 |
| Est. Completion | 2031-03 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05345171
The ClinicalTrials.gov registry entry for NCT05345171 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 23 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with OTC Deficiency appearing as the primary indexed condition, and to 5 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05345171 reports 16 study locations spanning 6 distinct geographic areas — top geographies include Other, California, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05345171 about?
NCT05345171 is a clinical study titled "Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency". The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
What is the current status of trial NCT05345171?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 32 participants. The study started on 2022-10-18. Estimated completion is 2031-03.
What conditions does trial NCT05345171 study?
This clinical trial studies the following conditions: OTC Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05345171?
The interventions under investigation include: Placebo (OTHER), DTX301 (GENETIC), Oral Corticosteroids (DRUG), Placebo for oral corticosteroids (DRUG), Sodium Acetate (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05345171?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 23 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05345171 being conducted?
This trial has 16 study locations across California, Colorado, Illinois, Ohio, Ontario. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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