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

Study of a Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients with Mucopolysaccharidosis Type I

Protocol Description

Among other factors, this study evaluates biological activity of the investigational drug AGT-181, for adults with mucopolysaccharidosis type I (MPS I), which includes Hurler, Hurler-Scheie and Scheie syndromes. People with MPS I lack normal amounts of a certain enzyme. Aldurazyme is an approved treatment to replace this enzyme, but is blocked by a kind of wall between the brain and blood system. The antibody AGT-181 tricks the body, enabling it to carry the enzyme to the brain and elsewhere. Researchers believe that once delivered, the drug may be able to help treat MPS I symptoms present in the brain and the rest of the body.

Study Level: Phase I

Eligibility Criteria

Subject to exclusion criteria, this study is accepting men and women ages 18 and older.
Men and Women: Ages 18 and older


Following the initial screening visit and exam, participants will receive 8 weeks of weekly infusions of the study drug at one of two dosing levels, 1 mg/kg or 3 mg/kg. During certain visits, additional testing will be done. Participants who have had prior enzyme replacement therapy can opt to wait 6 weeks for all prior medications to clear. The waiting period is called the washout. There will be one final visit with study doctors one week after the last infusion.
Visits: 12
Duration: 15 weeks, inclusive of the 6-week washout period

Status: Open to Enrollment

Source(s) of Support

ArmaGen (AGT-181-102)

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

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