One-Shot gene therapy tested in babies with devastating brain disease
NCT ID NCT04411654
Summary
This study is testing a single-dose gene therapy called LY3884961 in infants with Type 2 Gaucher disease, a severe and fatal genetic disorder that damages the brain and body. The main goals are to see if the treatment is safe and if it can improve symptoms like movement and thinking skills. Seven infants will be followed for about five years to monitor their health and development.
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Contacts and locations
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Locations
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Children's Hospital of Pittsburgh, 4401 Penn Avenue
Pittsburgh, Pennsylvania, 15224, United States
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Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030, United States
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Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road
Manchester, M13 9WL, United Kingdom
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UCSF Benioff Children's Hospital, 5700 Martin Luther King Jr Way
Oakland, California, 94609, United States
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University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
Minneapolis, Minnesota, 55454, United States
Conditions
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