Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome)
  • Phase

    Phase 1
  • Study Type

  • Status

    Completed No Results Posted
  • Intervention/Treatment

    agt-182 ...
  • Study Participants

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 Started
Apr 30
Primary Completion
Mar 27
Study Completion
Mar 27
Last Update
Sep 18

Drug AGT-182

Recombinant HIRMAb-IDS

Treated subjects Experimental

AGT-182 solution for infusion will be administered intravenously at doses of 1.0 mg/kg or 3.0 mg/kg weekly for 8-13 weeks.


Inclusion Criteria:

Male age 18 years or older
Diagnosis of Hunter Syndrome (documented fibroblast or leukocyte IDS enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory - or any level of enzyme deficiency together with the presence of a pathogenic mutation in the IDS gene - and documentation of normal enzymatic activity of at least 1 other sulfatase.)

Must fall into one of the following groups:

currently receiving standard enzyme replacement therapy (ERT) and be willing to discontinue it for the study duration, taking AGT-182 instead
have not received standard ERT for at least 3 months and have elevated uGAGs of at least 3.5 fold above age-related normals at study screening
have never received ERT
Voluntary written consent
Sexually mature males must be advised to use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

Refusal to complete screening/baseline evaluations
Receipt of an investigational drug within the prior 90 days
Any medical condition or other circumstances that may significantly interfere with study compliance
Clinically significant spinal cord compression, evidence of cervical instability
Known hypersensitivity to idursulfase or any of the components of AGT-182
Known to be nonresponsive to standard ERT treatment (i.e., high uGAG values despite taking full dose standard ERT)
History of diabetes mellitus or hypoglycemia
Contraindication to lumbar puncture, if the patient agrees to this optional assessment
No Results Posted