Sanfilippo B Syndrome Treatment with SBC-103 – Phase I/II

Study of Pediatric Patients with MPS IIIB to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

Protocol Description

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.

Eligibility Criteria

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

Status: Open for Enrollment

Source(s) of Support

Synageva BioPharma Corp.

Primary Investigator

Maria L. Escolar, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Program Coordinator Mary Brannaman, MPA, BS