Clinical Trial: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional




Official Title: An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181

Brief Summary:

AGT-181 is a fusion protein containing alpha-L-iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.

This is a long term safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will also be collected.