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NCT02991144 · ClinicalTrials.gov registry record · Phase 1
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
A Phase 1 study of Ornithine Transcarbamylase (OTC) Deficiency, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 16
- Enrollment target
- 9
- Study locations
NCT02991144: Completed Phase 1 study of Ornithine Transcarbamylase (OTC) Deficiency, sponsored by Ultragenyx Pharmaceutical.
NCT02991144 is a Phase 1 study of Ornithine Transcarbamylase (OTC) Deficiency that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 16 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (73% lower). The trial reports 9 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02991144, a Phase 1 study of Ornithine Transcarbamylase (OTC) Deficiency, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 16 participants
- Enrollment target
- 9
- Study locations
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.
Primary Outcome
AE: any untoward medical occurrence regardless of its causal relationship to study product. TEAE: any event not present before exposure to study product or any event already present that worsens in either intensity or frequency after exposure to study product. SAE: any event that results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anom
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 |
What the finished NCT02991144 record still lists
NCT02991144 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (73% lower).
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.
NCT02991144 lists 9 locations in 9 states (Colorado, Massachusetts, New York).
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.
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).
Who is sponsoring clinical trial NCT02991144?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
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
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Where NCT02991144's enrollment target sits among peer trials
16 1786th of 2000 higher than 203 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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