Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
NCT02991144 · View on ClinicalTrials.gov ↗
Study Summary
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
Conditions Studied
Interventions
- GENETIC scAAV8OTC
- DRUG Reactive Corticosteroid Taper Regimen
- DRUG Prophylactic Corticosteroid Taper Regimen
Study Locations (9)
Colorado
- The Children's Hospital Colorado — Aurora
Massachusetts
- Boston Children's Hospital — Boston
New York
- Icahn School of Medicine at Mount Sinai — New York
Ohio
- University Hospital Cleveland Medical Center/Case Western Reserve University — Cleveland
Alberta
- Alberta's Children's Hospital — Calgary
A Coruna
- Hospital Clinico Universitario de Santiago — Santiago de Compostela
Vizcaya
- Hospital Universitario de Cruzes — Barakaldo
London City
- National Hospital for Neurology & Neurosurgery — London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2017-07-31 |
| Est. Completion | 2021-12-16 |
| Phase | Phase 1 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02991144
The ClinicalTrials.gov registry entry for NCT02991144 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 16 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 23 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Ornithine Transcarbamylase (OTC) Deficiency appearing as the primary indexed condition, and to 3 interventions — of which scAAV8OTC is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02991144 reports 9 study locations spanning 9 distinct geographic areas — top geographies include Colorado, Massachusetts, New York. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT02991144 about?
NCT02991144 is a clinical study titled "Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency". This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
What is the current status of trial NCT02991144?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 16 participants. The study started on 2017-07-31. Estimated completion is 2021-12-16.
What conditions does trial NCT02991144 study?
This clinical trial studies the following conditions: Ornithine Transcarbamylase (OTC) Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02991144?
The interventions under investigation include: scAAV8OTC (GENETIC), Reactive Corticosteroid Taper Regimen (DRUG), Prophylactic Corticosteroid Taper Regimen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02991144?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 23 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02991144 being conducted?
This trial has 9 study locations across Colorado, Massachusetts, New York, Ohio, Alberta. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.