# Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

> **NCT05345171** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 32 (estimated)

## Conditions studied

- OTC Deficiency

## Interventions

- **GENETIC:** DTX301
- **OTHER:** Placebo
- **DRUG:** Oral Corticosteroids
- **DRUG:** Placebo for oral corticosteroids
- **DRUG:** Sodium Acetate

## Key facts

- **NCT ID:** NCT05345171
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2022-10-18
- **Primary completion:** 2027-09
- **Final completion:** 2031-03
- **Target enrollment:** 32 (ESTIMATED)
- **Last updated:** 2026-02-17


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05345171

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05345171, "Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency". Retrieved via AI Analytics 2026-06-04 from https://api.ai-analytics.org/clinical/NCT05345171. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*