AGT-182 is a fusion protein containing idursulfase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This study is a safety and dose ranging study to obtain safety and exposure data, as well as information on the biological activity of the investigational drug.
This is a sequential, open-label, dose escalation, multi-dose study in adults with Hunter syndrome. Two dose levels, assuming tolerability, are planned sequentially, with safety data from the previous cohort being reviewed prior to escalation to the next higher dose cohort. Subjects will receive weekly doses of AGT-182 for 8 weeks if ERT-naive or agreeing to a 6-week ERT washout, or for 13 weeks if currently taking ERT and not agreeing to washout.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Recombinant HIRMAb-IDS
Children's Hospital Oakland
Oakland, California, United States
Children's Hospital of Orange County
Orange, California, United States
Emory University
Decatur, Georgia, United States
ZKJM MC University of Mainz
Mainz, Germany
number of participants with adverse events as a measure of safety and tolerability
Time frame: 8 weeks (ERT-naive) or 13 weeks (ERT)
plasma pharmacokinetic parameters (maximal concentration, half-life, area under the curve, mean residence time, volume of distribution and clearance of AGT-182)
Time frame: 8 weeks (ERT-naive) or 13 weeks (ERT)
change in urinary or plasma glycosaminoglycans (GAGs)
Time frame: 8 weeks (ERT-naive) or 13 weeks (ERT)
change in liver or spleen size
Time frame: 8 weeks (ERT-naive) or 13 weeks (ERT)
change in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs)
Time frame: 8 weeks (ERT-naive) or 13 weeks (ERT)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Institute of Human Genetics, National Inst of Health, University of the Philippines
Manila, Philippines