Moderna tests mRNA therapy for rare, devastating metabolic disease
NCT ID NCT04899310
Summary
This study is testing an experimental mRNA therapy called mRNA-3705 for people with a rare genetic disorder called methylmalonic acidemia (MMA). The main goals are to see if the treatment is safe and if it can lower the high levels of a harmful acid in the blood that causes the disease. Participants receive the therapy through an IV infusion.
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Contacts and locations
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Locations
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Erasmus MC
Rotterdam, 3015 AA, Netherlands
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Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
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Hospital Universitario Cruces
Barakaldo, 48903, Spain
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Hôpital Necker - Enfants Malades
Paris, 75015, France
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Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304, United States
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
Conditions
Explore the condition pages connected to this study.