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The purpose of this study is to assess the safety and potential effectiveness of a gene transfer therapy (drug ABO-102) injected intravenously through a peripheral limb vein to help patients with mucopolysaccharidosis type IIIA (MPS IIIA), also called Sanfilippo type A, a progressive disease that primarily affects the brain. Through this study, researchers will determine if a therapy using ABO-102 is safe at three different dose levels. MPS IIIA is caused by the accumulation of glycosaminoglycans (GAGs) in the body due to an enzyme called N-sulfoglucosamine-sulfohydrolase (SGSH) not working properly. In lab tests, researchers have demonstrated potential for a functional copy of the SGSH gene to be transferred to replace SGSH genes that contain errors.
Subject to certain exclusion criteria, this study is enrolling participants ages 6 months and older who have been diagnosed with MPS IIIA.
Males and Females: Ages 6 months and older
Following screening visits, eligible participants will receive a single intravenous infusion of ABO-102 during a 48-hour inpatient stay at UPMC Children’s Hospital of Pittsburgh. After that, participants will return to the hospital over a 2-year period for examinations, testing, imaging, and blood draws in accordance with the protocol.
Visits: Up to 17, including hospitalization
Duration: 2 years
Ultragenyx Pharmaceutical Inc.
Study Description at National Institutes of Health
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
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
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