Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
← catalyst calendarNCT05345171 · readout ≤ 407 d
Sponsored by Ultragenyx Pharmaceutical Inc (industry) · RARE — their whole pipeline →.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
37actual
Sites
16
Countries
Argentina, Brazil, Canada +7
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-10-18 | actual | When the study began enrolling |
| Primary completion | Sep 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2031 | estimated | The whole study's end, after follow-up |
| First posted | 2022-04-25 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-02 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- OTC Deficiency
Interventions
- Genetic: DTX301 — also filed as avalotcagene ontaparvovec
- Other: Placebo
- Drug: Oral Corticosteroids — also filed as Prednisolone
- Drug: Placebo for oral corticosteroids
- Drug: Sodium Acetate
Primary outcomes
what the primary-completion date above is the date OFPlasma Ammonia as Measured by 24-Hour Ammonia (AUC0-24)
measured Week 36
Complete Responder Rate at the Final Study Visit After DTX301 Exposure
measured Up to 64 Weeks Post DTX301 Infusion
Publications
- PMID 38171926 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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