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.
Detailed Summary:
Sponsor: ArmaGen, Inc
Current Primary Outcome: number of patients with adverse events as a measure of safety and tolerability [ Time Frame: 24 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- change in total urinary glycosaminoglycans (GAGs) [ Time Frame: 24 months ]
- change in urinary heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
- change in plasma heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
- change in CSF heparan sulfate and dermatan sulfate [ Time Frame: 24 months ]
- change in liver and/or spleen volume (measured by MRI) [ Time Frame: 24 months ]
Original Secondary Outcome: Same as current
Information By: ArmaGen, Inc
Dates:
Date Received: February 27, 2017
Date Started: October 2016
Date Completion: October 2019
Last Updated: March 1, 2017
Last Verified: February 2017
