Researchers are conducting a clinical trial of AGT-181, an investigational treatment for people with attenuated (less severe) MPS I (Scheie and Hurler-Scheie). AGT-181 is an enzyme replacement therapy (ERT) designed to treat both the body-related (somatic) and central nervous system (CNS) symptoms and complications of MPS I.
Currently approved treatments for MPS I are unable to treat the brain (CNS) because of the enzyme’s inability to cross the blood-brain barrier (BBB). The BBB is a filter that protects the brain from toxins but allows vital nutrients like insulin to cross from the blood into the brain. As a result, available therapies do not address many of the severe and progressive neurological complications of the MPS I. AGT-181 is designed to cross the BBB in the same way insulin does.
This clinical trial is monitored by an independent Data Monitoring Committee (DMC) and safety will be reported to an institutional review board to ensure the safety of the participants.