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

NCT03517085 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

12

Enrollment

INDUSTRY

Sponsor class

Conditions

GSD1

Interventions

GENETIC: DTX401
DRUG: steroid regimen

Sponsor

Ultragenyx Pharmaceutical Inc