US-based ArmaGen has started dosing in a Phase II proof-of-concept (POC) clinical trial of an investigational enzyme replacement therapy (ERT), AGT-181, to treat pediatric patients with Hurler syndrome in Brazil.
The trial is being carried out after a Phase I study was successfully completed for adult patients.
Hurler syndrome is the most severe form of mucopolysaccharidosis type I (MPS I), an uncommon, hereditary lysosomal storage disorder that affects the brain and spinal cord in children, and results in a wide range of debilitating symptoms.
The few commercial treatments available for Hurler syndrome do not penetrate the blood-brain barrier (BBB), and therefore fail to address neurological complications caused by the disease.
AGT-181 uses the body to transport products across the BBB, targeting the receptor that delivers insulin to body cells.
The Phase II POC study will include children aged two or older with Hurler or Hurler-Scheie syndrome and central nervous system involvement.
ArmaGen senior vice president of global clinical development Patrice Rioux said: "We are extremely pleased to be working with Dr. Roberto Giugliani and his team at Hospital de Clínicas, as this site has a world-class reputation for clinical research and has contributed to numerous successful ERT clinical studies over the years.
"This POC trial joins our ongoing US Phase I/IIa trial in adults and will contribute important data on safety and tolerability for our platform technology.
"Additionally, this study will allow us to capture biomarker data to help assess the potential impact of treatment on both peripheral and central nervous system symptoms of Hurler syndrome."
The Phase II POC trial's primary objective is to determine the safety and tolerability of weekly infusions of 1.0 or 3.0 mg/kg of AGT-181 in children with Hurler or Hurler-Scheie syndrome.
According to the company, the Phase II POC study is expected to be completed by the end of the year.