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This Phase 3 study evaluates the efficacy of DTX401 (pariglasgene brecaparvovec) in patients with glycogen storage disease type Ia (GSDIa). Its main objectives are to reduce or eliminate dependence on exogenous glucose replacement therapy for maintaining normal blood sugar and to maintain or improve the quality of glucose control; the study also includes placebo, oral prednisolone, and placebo for oral prednisolone. Eligibility requires documented GSDIa confirmed by molecular testing or enzymatic activity on liver biopsy, and a current cornstarch (or equivalent) regimen following international guidance with stable nutritional, glycemic, and clinical status.
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: * Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy * Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status. * Willing and able to complete the informed consent process and to comply with study procedures and visit schedule * Females of childbearing potential and fertile males must consent to use highly effective contraception from the period following informed consent through the duration of the 144-week study and in cases of early withdrawal at least 48 weeks after the last dose of investigational product (IP). Female subjects must agree not to become pregnant. Male subjects must agree not to father a child or donate sperm Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV8 capsid * History of liver transplant, including hepatocyte cell therapy/ transplant * History of liver disease * 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 * Non-fasting triglycerides ≥1000 mg/dL * Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study. * Current or previous participation in another gene transfer study * History of illicit drug use within 60 days prior to screening or positive results from an 8-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart Note: additional inclusion/exclusion criteria may apply, per protocol