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NCT03517085 · ClinicalTrials.gov registry record · Phase 1
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
A Phase 1 study of GSD1, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 6
- Study locations
NCT03517085 is a Phase 1 study of GSD1 that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 12 participants. The trial reports 6 study locations across 6 states.
The verdict
NCT03517085, a Phase 1 study of GSD1, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 6
- Study locations
Study Summary
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
Conditions Studied
Interventions
- GENETIC DTX401
- DRUG steroid regimen
Study Locations (6)
Connecticut
- UCONN Health - Farmington
Michigan
- Michigan Medicine University of Michigan - Ann Arbor
Texas
- UT Health - McGovern Medical School - Houston
Quebec
- Montreal Children Hospital, McGill University Health Centre - Montreal
Other
- University Medical Center Groningen - Groningen
A Coruna
- Complejo Hospitalario Universitario de Santiago - Santiago de Compostela
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2018-05-18 |
| Est. Completion | 2021-11-02 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03517085
The ClinicalTrials.gov registry entry for NCT03517085 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 12 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 GSD1 appearing as the primary indexed condition, and to 2 interventions - of which DTX401 is the first listed.
NCT03517085 reports 6 study locations spanning 6 distinct geographic areas - top geographies include Connecticut, Michigan, Texas.
Frequently Asked Questions
What is clinical trial NCT03517085 about?
NCT03517085 is a clinical study titled "Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)". The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
What is the current status of trial NCT03517085?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2018-05-18. Estimated completion is 2021-11-02.
What conditions does trial NCT03517085 study?
This clinical trial studies the following conditions: GSD1.
What interventions are being tested in trial NCT03517085?
The interventions under investigation include: DTX401 (GENETIC), steroid regimen (DRUG).
Who is sponsoring clinical trial NCT03517085?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03517085 being conducted?
This trial has 6 study locations across Connecticut, Michigan, Texas, Quebec, A Coruna. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Related
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