🏛️ Verified NIH Record • Updated 2025-12-09
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a) (NCT05095727)
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
🔵 Active, Not Recruiting Phase1 / Phase2 💊 Investigational Drug / Biologic Sponsor: ModernaTX, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of Connecticut Health Center — Farmington, Connecticut
Age range: 6 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- University of Connecticut Health Center — Farmington, Connecticut
- Boston Children's Hospital — Boston, Massachusetts
- Duke University Medical Center — Durham, North Carolina
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- The University of Texas Health Science Center at Houston — Houston, Texas
- Baylor College of Medicine — Houston, Texas
- University of Utah — Salt Lake City, Utah
- Stollery Children's Hospital University of Alberta — Edmonton, Alberta
- The Hospital for Sick Children — Toronto, Ontario
- AP-HP - Hôpital Antoine Béclère — Clamart,
- CHRU Tours - Hopital Clocheville — Tours,
- Universitair Medisch Centrum Groningen — Groningen,
- Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw,
- Hospital Universitario 12 de Octubre — Madrid,
- Hospital Regional Universitario de Malaga — Málaga,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Glycogen Storage Disease
- Rare Glycogen Storage Diseases Natural History Study — Durham, North Carolina
Explore by condition: All Glycogen Storage Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.