🏛️ Verified NIH Record • Updated 2026-05-28
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency (NCT05345171)
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Ultragenyx Pharmaceutical Inc
🩺 Plain-English Summary (8th-Grade Level)
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
Who can join: Review the eligibility criteria below.
Location: University of California — Los Angeles, California
Age range: 12 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- University of California — Los Angeles, California
- University of Colorado — Aurora, Colorado
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- University Hospitals Cleveland Medical Center — Cleveland, Ohio
- Hospital Italiano de Buenos Aires — Buenos Aires,
- Clinica Universitaria Reina Fabiola — Córdoba,
- Hospital de Clinicas de Porto Alegre — Porto Alegre,
- The Hospital for Sick Children — Toronto, Ontario
- 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,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in OTC Deficiency
- A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD — Chevy Chase, Maryland
Explore by condition: All OTC Deficiency clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.