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)

Status: Completed
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Healthy Volunteers: No
View:

• 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.

Locations
United States
California
Children's Hospital Oakland
Oakland
Children's Hospital of Orange County
Orange
Georgia
Emory University
Decatur
Other Locations
Germany
ZKJM MC University of Mainz
Mainz
Philippines
Institute of Human Genetics, National Inst of Health, University of the Philippines
Manila
Time Frame
Start Date: April 2015
Completion Date: March 27, 2017
Participants
Target number of participants: 6
Treatments
Experimental: Treated subjects
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.
Authors
William R Wilcox, Chester B Whitley, Raymond Y Wang, David H Viskochil, Paul Harmatz, Ozlem Goker-Alpan, Barbara K Burton, Julia Hennermann, JMP van den Hout, Jerry Vockley, Mary Anne Chiong
Sponsors
Leads: ArmaGen, Inc

This content was sourced from clinicaltrials.gov

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