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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 is a Phase 1 study of Ornithine Transcarbamylase (OTC) Deficiency that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 16 participants. The trial reports 9 study locations across 9 states.
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.
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 |
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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, categorized as Phase 1. 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 52 total studies on file at ClinicalTrials.gov.
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 reports 9 study locations spanning 9 distinct geographic areas - top geographies include 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.
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