N/A
N=12
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
Glycogen Storage Disease Type IA · Von Gierke's Disease (GSD Type Ia)
Bottom Line
View on ClinicalTrials.gov: NCT03970278 ↗Enrolled (actual)
12
Serious AEs
58.3%
Results posted
Mar 2026
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs — 3; 3; 3; 3 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- No intervention (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Primary completion
- Feb 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs |
3; 3; 3; 3; 0; 2 | — |
| SECONDARY Change From Baseline in Time to First Hypoglycemic Event During a Controlled Fasting Challenge Over Time |
4.19; -1.78; -0.59; 3.64; 3.06; -1.78 | — |
Summary
The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.
Eligibility Criteria
Inclusion Criteria
- Received DTX401 in study 401GSDIA01.
- Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
- Willing and able to comply with all scheduled study visits, procedures, and requirements.
Exclusion Criteria
- Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
- Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.
Data sourced from ClinicalTrials.gov (NCT03970278). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.