Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
- Registration Number
- NCT02597114
- Lead Sponsor
- ArmaGen, Inc
- 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 3
Inclusion Criteria
- Must have completed clinical trial AGT-181-102
- Voluntary written consent by patient or legally responsible representative
- All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
- Negative pregnancy test (females)
Exclusion Criteria
- Refusal to complete screening evaluations.
- Any medical condition or other circumstances that may significantly interfere with study compliance
- Patient is pregnant or lactating
- Clinically significant spinal cord compression, evidence of cervical instability.
- Subject developed clinically relevant hypersensitivity to AGT-181
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description AGT-181 AGT-181 AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study
- Primary Outcome Measures
Name Time Method Number of participants with adverse events as a measure of safety and tolerability 104 weeks (2 years)
- Secondary Outcome Measures
Name Time Method changes in urinary or plasma glycosaminoglycans (GAGs) 104 weeks (2 years) change in spleen size 104 weeks (2 years) change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid 104 weeks (2 years) change in liver size 104 weeks (2 years)