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NCT05345171 · ClinicalTrials.gov registry record · Phase 3
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
A Phase 3 study of OTC Deficiency, sponsored by Ultragenyx Pharmaceutical.
- Active
- Registry status
- Phase 3
- Development phase
- 32
- Enrollment target
- 16
- Study locations
NCT05345171: Active Phase 3 study of OTC Deficiency, sponsored by Ultragenyx Pharmaceutical.
NCT05345171 is a Phase 3 study of OTC Deficiency that is active but no longer recruiting, run by Ultragenyx Pharmaceutical. The registered enrollment target is 32 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). The trial reports 16 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05345171, a Phase 3 study of OTC Deficiency, is active but no longer recruiting, sponsored by Ultragenyx Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 32 participants
- Enrollment target
- 16
- Study locations
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 |
What the registry record for NCT05345171 still lists
NCT05345171 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower).
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.
NCT05345171 lists 16 locations in 6 states (Other, California, Colorado).
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.
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).
Who is sponsoring clinical trial NCT05345171?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05345171's enrollment target sits among peer trials
32 1909th of 2000 higher than 87 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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