NCT03071341

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: ArmaGen, Inc · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Detailed description

AGT-181-101E is a 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 be collected as well as longitudinal safety information.

Conditions

• Mucopolysaccharidosis I

Interventions

Type	Name	Description
DRUG	AGT-181	Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

Timeline

• Start date: 2016-10-01
• Primary completion: 2018-02-01
• Completion: 2018-02-01
• First posted: 2017-03-06
• Last updated: 2023-03-15

Locations

1 site across 1 country: Brazil

Regulatory

• FDA-regulated drug study