A Study of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a)

• Status: Not yet recruiting
• Trial end date: 2024-01-19
• Target enrollment: 0
• Participant gender: All

Summary

The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in participants with GSD1a.

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

Details

• Lead Sponsor: ModernaTX, Inc.

Criteria

Inclusion Criteria:

• Documented GSD1a with confirmation by genetic testing

• Documented history of ≥1 hypoglycemic event with blood glucose <60 milligrams/deciliter (mg/dL) (<3.3 millimoles/liter [mmol/L]) and symptoms of hypoglycemia in the absence of acute illness, with at least one such event in the 4 weeks before signing the Informed Consent.

Exclusion Criteria:

• Liver transplant, including hepatocyte cell therapy/transplant

• Received gene therapy for GSD1a

• Presence of liver adenoma >5 centimeters (cm) in size

• Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenoma, in the previous 2 years.

Note: Additional inclusion/exclusion criteria may apply, per protocol.