Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
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ClinicalTrials.gov Identifier: NCT02262338 |
Recruitment Status :
Completed
First Posted : October 13, 2014
Last Update Posted : September 18, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Mucopolysaccharidosis II | Drug: AGT-182 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 6 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Masking Description: | Open Label |
Primary Purpose: | Treatment |
Official Title: | 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) |
Actual Study Start Date : | April 2015 |
Actual Primary Completion Date : | March 27, 2017 |
Actual Study Completion Date : | March 27, 2017 |

Arm | Intervention/treatment |
---|---|
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.
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Drug: AGT-182
Recombinant HIRMAb-IDS |
- 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) ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.)
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02262338
United States, California | |
Children's Hospital Oakland | |
Oakland, California, United States, 94609 | |
Children's Hospital of Orange County | |
Orange, California, United States, 92868 | |
United States, Georgia | |
Emory University | |
Decatur, Georgia, United States, 30033 | |
Germany | |
ZKJM MC University of Mainz | |
Mainz, Germany | |
Philippines | |
Institute of Human Genetics, National Inst of Health, University of the Philippines | |
Manila, Philippines |
Study Director: | Patrice Rioux, MD PhD | ArmaGen, Inc |
Responsible Party: | ArmaGen, Inc |
ClinicalTrials.gov Identifier: | NCT02262338 |
Other Study ID Numbers: |
AGT-182-101 |
First Posted: | October 13, 2014 Key Record Dates |
Last Update Posted: | September 18, 2018 |
Last Verified: | September 2018 |
MPS II Hunter Syndrome |
Mucopolysaccharidosis II Mucopolysaccharidoses Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases |
Metabolic Diseases Mental Retardation, X-Linked Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System |