The primary objective of this Phase 1/2 study is to determine the safety of single doses of the gene therapy DTX401 (AAV8G6PC, pariglasgene brecaparvovec), including the incidence of dose-limiting toxicities at each dose level, in adults with glycogen storage disease type Ia (GSDIa). A steroid regimen is given alongside the treatment. Eligibility requires being aged 18 or older, GSDIa documented by molecular testing, a documented history of at least one hypoglycemic event with blood glucose below 60 mg/dL, and stable disease without hospitalization for severe hypoglycemia.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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.