A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Public ClinicalTrials.gov record NCT05139316. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05139316
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 52 participants
Conditions and interventions
Conditions
Interventions
- DTX401 Genetic
- Placebo Other
- Oral prednisolone Drug
- Placebo for oral prednisolone Drug
Genetic · Other · Drug
Eligibility (public fields only)
- Age range
- 8 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 8, 2021 (Actual)
- Primary completion
- Feb 20, 2024 (Actual)
- Completion
- Feb 20, 2026 (Actual)
- Last update posted
- Oct 14, 2026
2021 – 2026
United States locations
- U.S. sites
- 9
- U.S. states
- 9
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital of Orange County | Orange | California | 92868 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| University of Connecticut Health Center | Farmington | Connecticut | 06030 | — |
| Mount Sinai | The Bronx | New York | 10467 | — |
| Duke University | Durham | North Carolina | 27710 | — |
| Cleveland Clinic | Cleveland | Ohio | 44195 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Texas | Houston | Texas | 77030 | — |
| Primary Children's Hospital | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 11 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05139316, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 14, 2026 · Synced Oct 23, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05139316 live on ClinicalTrials.gov.