A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCT05139316 · clinicaltrials.gov ↗

PHASE3

Phase

COMPLETED

Status

49

Enrollment

INDUSTRY

Sponsor class

Conditions

Glycogen Storage Disease Type IA

Interventions

GENETIC: DTX401
OTHER: Placebo
DRUG: Oral prednisolone
DRUG: Placebo for oral prednisolone

Sponsor

Ultragenyx Pharmaceutical Inc