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The purpose of this study is to assess the safety and potential effectiveness of a single dose of LY3884961, a type of gene therapy, in infants with Type 2 Gaucher disease (GD2). LY3884961 is designed to deliver a normal GBA1 gene copy into the body to increase the activity of a specific protein called glucocerebrosidase or “GCase,” which is low in GD2 patients. When levels of GCase are low, the body cannot break down fatty chemicals, and they build up in the body’s cells. These processes are believed to contribute to the onset and severity of neurologic and other symptoms associated with GD2.
Subject to certain exclusion criteria, this study is enrolling participants from birth through 24 months of age who have been diagnosed with GD2. Males and Females: Birth through 24 months
Following screening visits, eligible participants will receive a single dose of PR001A during a 48-hour inpatient stay at UPMC Children’s Hospital of Pittsburgh. After that, participants will return to the hospital over a 5-year period for examinations, testing, imaging, and blood draws in accordance with the protocol. Visits: Up to 15, including hospitalization for initial drug treatment Duration: 61 months (5 years and 1 month)
Prevail Therapeutics
Study Description at National Institutes of Health Gaucher Disease Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact: Dawn Kolar, Clinical Research Coordinator 412-692-8343
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh One Children’s Hospital Way 4401 Penn Ave. Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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