NCT05095727

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

A Phase 1/2, Adaptive, Open-label, Single Ascending Dose to Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a), Followed by an Open-label Extension

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: ModernaTX, Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Glycogen Storage Disease

Interventions

Type	Name	Description
DRUG	mRNA-3745	Sterile frozen liquid dispersion for injection

Timeline

• Start date: 2022-06-01
• Primary completion: 2026-11-30
• Completion: 2026-11-30
• First posted: 2021-10-27
• Last updated: 2025-12-15

Locations

16 sites across 6 countries: United States, Canada, France, Netherlands, Poland, Spain

Regulatory

• FDA-regulated drug study