Moderna tests mRNA therapy for rare metabolic disease

NCT ID NCT05095727

Summary

This early-stage study is testing a new mRNA-based drug called mRNA-3745 for people with a rare inherited condition called glycogen storage disease type 1a (GSD1a). The main goal is to see if the drug is safe and well-tolerated when given through an IV. Researchers will also check if it helps patients better control their blood sugar levels during periods of fasting.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for GLYCOGEN STORAGE DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • AP-HP - Hôpital Antoine Béclère

    Clamart, 92140, France

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHRU Tours - Hopital Clocheville

    Tours, 37000, France

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27713, United States

  • Hospital Regional Universitario de Malaga

    Málaga, 29011, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28026, Spain

  • Instytut Pomnik Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • Stollery Children's Hospital University of Alberta

    Edmonton, Alberta, T6G 2R7, Canada

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030-1501, United States

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ, Netherlands

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030-0001, United States

  • University of Utah

    Salt Lake City, Utah, 84132-0001, United States

Conditions

Explore the condition pages connected to this study.