Moderna tests mRNA shot to fight rare, Life-Threatening metabolic disease

NCT ID NCT04899310

Ongoing Disease control Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

Summary

This study is testing an experimental mRNA therapy called mRNA-3705 for people with a rare, inherited metabolic disorder called methylmalonic acidemia (MMA). The main goals are to see if the treatment is safe and if it can lower the levels of harmful acids that build up in the blood. Participants receive the therapy through an IV infusion, and the study will measure changes in their health and quality of life.

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Contacts and locations

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Locations

Erasmus MC

Rotterdam, 3015 AA, Netherlands
Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada
Hospital Universitario 12 de Octubre

Madrid, 28041, Spain
Hospital Universitario Cruces

Barakaldo, 48903, Spain
Hôpital Necker - Enfants Malades

Paris, 75015, France
Lucile Packard Children's Hospital at Stanford

Palo Alto, California, 94304, United States
Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom
Stollery Children's Hospital University of Alberta

Edmonton, Alberta, T6G 2R7, Canada
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
UCLA Medical Center

Los Angeles, California, 90095, United States
Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

Conditions

Explore the condition pages connected to this study.

METHYLMALONIC ACIDEMIA