MPS IIIA Treatment with ABO-102 – Phase I/II

Protocol Description

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.

Study Level: Phase I/II

Eligibility Criteria

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

Requirements

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

Status: Open to Enrollment

Source(s) of Support

Ultragenyx Pharmaceutical Inc.

Additional Information

Study Description at National Institutes of Health
Neurodevelopment in Rare Disorders Research

Primary Investigator

Deepa Soundara Rajan, MD

Contact Information

For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
412-692-8343