Last updated on February 2020

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

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: GSD1
  • Age: Between 18 - 100 Years
  • Gender: Male or Female

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

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 ULN, alkaline phosphatase >2.5 ULN

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

Recruitment Status: Open

Brief Description Eligibility Contact Research Team

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