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

Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

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


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.

Primary Investigator

Deepa Soundara Rajan, MD

Contact Information

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