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This study will evaluate the effects and effectiveness of SBC-103, the recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) when intravenously provided to pediatric patients for the treatment of mucopolysaccharidosis IIIB (MPS IIIB), also known as Sanfilippo B syndrome. Sanfilippo syndrome is a rare, inherited genetic disorder that primarily affects the central nervous system and for which there presently is no cure.
Subject to certain exclusion criteria, this study is open to children of both genders, ages 2 to 12 years of age, who have a confirmed diagnosis of Sanfilippo B syndrome.
Boys: 2 to 12 years old
Girls: 2 to 12 years old
Following the initial screening, eligible patients will receive SBC-103 every other week for 24 weeks. Participants will have a 4-week interruption of therapy prior to being considered for a second arm of the study, extended therapy with SBC-103 for up to 3 years.
Visits: Approximately 25 for the base study
Duration: 1 year for the base study; up to 4 years if enrolled in the extended study
Synageva BioPharma Corp.
Study Description at National Institutes of Health
Maria L. Escolar, MD, MS
For more information about the study or enrollment, please contact:
Program Coordinator Mary Brannaman, MPA, BS
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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