# Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

> **NCT03517085** · PHASE1,PHASE2 · COMPLETED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 12 (actual)

## Conditions studied

- GSD1

## Interventions

- **GENETIC:** DTX401
- **DRUG:** steroid regimen

## Key facts

- **NCT ID:** NCT03517085
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2018-05-18
- **Primary completion:** 2021-11-02
- **Final completion:** 2021-11-02
- **Target enrollment:** 12 (ACTUAL)
- **Last updated:** 2022-11-18


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03517085

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03517085, "Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)". Retrieved via AI Analytics 2026-07-19 from https://api.ai-analytics.org/clinical/NCT03517085. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*