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NCT03636438ACTIVE NOT RECRUITING

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Study Summary

Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.

Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions / Drugs
No Intervention
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment11 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045605+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 11 participants
Sponsor Catalyst Profile

Ultragenyx Pharmaceutical (RARE) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.