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

Study DTX401-CL301 is a phase 3 study to determine the efficacy and confirm the safety of DTX401 in patients 8 years and older with glycogen storage disease type Ia (GSDIa). Participants were randomized 1:1 to DTX401 or placebo group and followed closely for 48 weeks. At week 48 eligible participants crossed over and received DTX401 if they had previously received placebo or placebo if they had previously received DTX401 and were followed closely for an additional 96 weeks. After completion of week 144 or early withdrawal, participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion. In Japan, there was a single open label study arm and all participants were treated with DTX401. At week 48, Japanese participants were offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.