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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Glycogen Storage Disease

In brief

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.

Key facts

Study ID

NCT05095727

Run by

ModernaTX, Inc.

People needed

15

Starts

2022-06-01

Expected to finish

2026-11-30

Last updated by the study team

2025-12-15

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (G6PC) mutations by genetic testing.
• Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening

You may not qualify if…

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

Where it is running

• University of Connecticut Health Center — Farmington, Connecticut, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• The University of Texas Health Science Center at Houston — Houston, Texas, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada
• The Hospital for Sick Children — Toronto, Ontario, Canada
• AP-HP - Hôpital Antoine Béclère — Clamart, France
• CHRU Tours - Hopital Clocheville — Tours, France
• Universitair Medisch Centrum Groningen — Groningen, Netherlands
• Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw, Poland
• Hospital Universitario 12 de Octubre — Madrid, Spain
• Hospital Regional Universitario de Malaga — Málaga, Spain

Full record on ClinicalTrials.gov

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