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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 the therapy using ABO-102 is safe and potentially effective in pediatric cases with middle and advanced stages of MPS IIIA. The disease 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 inclusion and exclusion criteria, this study is enrolling participants ages 2 months through 17 years who have been diagnosed with MPS IIIA in middle and advanced stages.
Males and Females: Ages 2 months through 17 years
Following screening visits, eligible participants will receive a single intravascular 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 schedule over a 2-year period.
Visits: Up to 17, including hospitalization
Duration: 2 years
Study Description at National Institutes of Health
Neurodevelopment in Rare Disorders Research
Maria L. Escolar, MD, MS
For more information about the study or enrollment, please contact:
Laura Mathews, Clinical Research Coordinator
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