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

• Status: Completed
• Trial end date: 2021-11-02
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ultragenyx Pharmaceutical Inc

Criteria

Key Inclusion Criteria:

• Males and females ≥18 years of age

• Documented GSDIa with confirmation by molecular testing

• Documented history of ≥1 hypoglycemic event with blood glucose <60 mg/dL (<3.33 mmol/L)

• Patient's GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit

Key Exclusion Criteria:

• Anti-AAV8 neutralizing antibody titer ≥1:5

• Screening or Baseline (Day 0) blood glucose level <60 mg/dL (<3.33 mmol/L)

• Liver transplant, including hepatocyte cell therapy/transplant

• Presence of liver adenoma >5 cm in size

• Presence of liver adenoma >3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year

• Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN), total bilirubin > 1.5 x ULN, or alkaline phosphatase > 2.5 x ULN

Note additional inclusion/exclusion criteria may apply, per protocol.