MPS I (Hurler, Hurler-Scheie, Scheie) Long-term Treatment with AGT-181 – Phase I

Evaluating Safety and Glycosaminoglycans in Adult Patients with Hurler-Scheie or Scheie Syndrome who have Completed 8-Weeks of Dosing with AGT-181 in Study AGT-181-102

Protocol Description

Through this study, researchers will be studying longer-term effects of the investigational drug AGT-181 on the levels of certain markers of the disease mucopolysaccharidosis type I (MPS I), which includes Hurler, Hurler-Scheie and Scheie syndromes. These markers, called glycosaminoglycans (GAGs), are found in urine, blood and cerebral spinal fluid and can accumulate in people who have MPS I. Decreasing levels of GAGs in body fluids may indicate that the medicine has activity in MPS I treatment.

Study Level: Phase I

Eligibility Criteria

Subject to exclusion criteria, this study is accepting men and women ages 18 and older with MPS I who have completed 8 weeks of participation in study AGT-181-102.
Men and Women: Ages 18 and older


Participants will continue to receive weekly intravenous doses of the study drug for 24 more weeks following successful completion of the AGT-181-102 study. Dosages will be at the same levels as before, either 1 mg/kg or 3 mg/kg, until all safety data from the AGT-181-102 study is assessed, at which point the dosage may be increased. Physicals, and blood/urine/spinal fluid testing as well as MRI scans, will be performed at study intervals. There will be one final visit with study doctors one week after the last infusion.
Visits: 25
Duration: 25 weeks

Status: Enrolling by Invitation

Source(s) of Support

ArmaGen (Study AGT 181-103)

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC