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NCT05139316 · ClinicalTrials.gov registry record · Phase 3
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
A Phase 3 study of Glycogen Storage Disease Type Ia, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 49
- Enrollment target
- 20
- Study locations
NCT05139316 is a Phase 3 study of Glycogen Storage Disease Type Ia that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 49 participants, above the 42-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (17% higher). The trial reports 20 study locations across 15 states.
The verdict
NCT05139316, a Phase 3 study of Glycogen Storage Disease Type Ia, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 49 participants
- Enrollment target
- 20
- Study locations
Study Summary
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.
Conditions Studied
Interventions
- OTHER Placebo
- GENETIC DTX401
- DRUG Oral prednisolone
- DRUG Placebo for oral prednisolone
Study Locations (20)
Other
- University Medical Center Eppendorf - Hamburg
- University of Naples - Naples
- Kumamoto University Hospital - Kumamoto
- Osaka City General Hospital - Osaka
- Fujita Health University Hospital - Toyoake
- Groningen University - Groningen
California
- Children's Hospital of Orange County - Orange
Colorado
- Children's Hospital Colorado - Aurora
Connecticut
- University of Connecticut Health Center - Farmington
New York
- Mount Sinai - The Bronx
North Carolina
- Duke University - Durham
Ohio
- Cleveland Clinic - Cleveland
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 49 participants |
| Start Date | 2021-11-08 |
| Est. Completion | 2026-02-05 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05139316
The ClinicalTrials.gov registry entry for NCT05139316 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 49 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 42-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (17% higher). The listed sponsor is Ultragenyx Pharmaceutical, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Glycogen Storage Disease Type Ia appearing as the primary indexed condition, and to 4 interventions - of which Placebo is the first listed.
NCT05139316 reports 20 study locations spanning 15 distinct geographic areas - top geographies include Other, California, Colorado.
Frequently Asked Questions
What is clinical trial NCT05139316 about?
NCT05139316 is a clinical study titled "A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)". The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.
What is the current status of trial NCT05139316?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 49 participants. The study started on 2021-11-08. Estimated completion is 2026-02-05.
What conditions does trial NCT05139316 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type Ia.
What interventions are being tested in trial NCT05139316?
The interventions under investigation include: Placebo (OTHER), DTX401 (GENETIC), Oral prednisolone (DRUG), Placebo for oral prednisolone (DRUG).
Who is sponsoring clinical trial NCT05139316?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05139316 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, New York, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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