# Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

> **NCT03970278** · — · COMPLETED · sponsor: **Ultragenyx Pharmaceutical Inc** · enrollment: 12 (actual)

## Conditions studied

- Glycogen Storage Disease Type IA
- Von Gierke's Disease (GSD Type Ia)

## Interventions

- **OTHER:** No intervention

## Key facts

- **NCT ID:** NCT03970278
- **Lead sponsor:** Ultragenyx Pharmaceutical Inc
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** COMPLETED
- **Start date:** 2019-07-15
- **Primary completion:** 2025-02-25
- **Final completion:** 2025-02-25
- **Target enrollment:** 12 (ACTUAL)
- **Last updated:** 2026-03-18


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03970278

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03970278, "Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia". Retrieved via AI Analytics 2026-07-20 from https://api.ai-analytics.org/clinical/NCT03970278. Licensed CC0.

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