Clinical Trial: Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

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




Official Title: An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dos

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 study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug