Moderna tests mRNA therapy for rare metabolic disease
NCT ID NCT05095727
Summary
This early-stage trial is testing an experimental mRNA therapy called mRNA-3745 for people with Glycogen Storage Disease Type 1a (GSD1a), a rare genetic disorder that causes dangerous low blood sugar. The main goal is to check the safety and side effects of the treatment, given by IV infusion, in up to 15 adults and children. Researchers will also measure if the therapy helps the body better manage blood sugar levels during fasting.
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Contacts and locations
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Locations
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AP-HP - Hôpital Antoine Béclère
Clamart, 92140, France
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CHRU Tours - Hopital Clocheville
Tours, 37000, France
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center
Durham, North Carolina, 27713, United States
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Hospital Regional Universitario de Malaga
Málaga, 29011, Spain
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Hospital Universitario 12 de Octubre
Madrid, 28026, Spain
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Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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The University of Texas Health Science Center at Houston
Houston, Texas, 77030-1501, United States
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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University of Connecticut Health Center
Farmington, Connecticut, 06030-0001, United States
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University of Utah
Salt Lake City, Utah, 84132-0001, United States
Conditions
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