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

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

OTC Deficiency

Interventions / Drugs

DTX301 Placebo Oral Corticosteroids Placebo for oral corticosteroids Sodium Acetate

View on ClinicalTrials.gov ↗

Sponsor

RARE

Ultragenyx Pharmaceutical

Rare Disease

C63/100

Study Details

Enrollment32 participants

Primary Completion—

Lead SponsorUltragenyx Pharmaceutical Inc

Data Retrieved2026-04-11 04:25:35.045675+00:00

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