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A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Sponsor: ModernaTX, Inc.
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.
Official title: 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
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
INTERVENTIONAL
Enrollment
15
Start Date
2022-06-01
Completion Date
2026-11-30
Last Updated
2025-12-15
Healthy Volunteers
No
Conditions
Interventions
mRNA-3745
Sterile frozen liquid dispersion for injection
Locations (16)
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