Overview
The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.
Description
The study includes a single ascending dose (SAD) stage and a multiple ascending dose (MAD) stage. Participants enrolled in the MAD stage have the option to continue treatment in an open-label extension (OLE) period that will assess long-term safety and clinical activity of mRNA-3745.
Eligibility
Inclusion Criteria:
- Documented GSD1a with confirmation by genetic testing
- Documented history of a symptomatic hypoglycemic event with blood glucose <60 mg/dL (<3.3 mmol/L)
- Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening
Exclusion Criteria:
- Solid organ transplant
- Received gene therapy for GSD1a
- Presence of liver adenoma >5 centimeters (cm) in size
- Diagnosis of type 1 or type 2 diabetes mellitus
- Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenomas, in the previous 2 years
- Requirement for continuous feeds via gastrostomy or nasogastric tubes
Note: Additional inclusion/exclusion criteria may apply, per protocol.