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Trial record 1 of 1 for:    AGT-182
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Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02262338
Recruitment Status : Completed
First Posted : October 13, 2014
Last Update Posted : September 18, 2018
Information provided by (Responsible Party):
ArmaGen, Inc

Brief 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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis II Drug: AGT-182 Phase 1

Detailed Description:
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.

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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.
Drug: AGT-182
Recombinant HIRMAb-IDS

Primary Outcome Measures :
  1. number of participants with adverse events as a measure of safety and tolerability [ Time Frame: 8 weeks (ERT-naive) or 13 weeks (ERT) ]

Secondary Outcome Measures :
  1. 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) ]
  2. change in urinary or plasma glycosaminoglycans (GAGs) [ Time Frame: 8 weeks (ERT-naive) or 13 weeks (ERT) ]
  3. change in liver or spleen size [ Time Frame: 8 weeks (ERT-naive) or 13 weeks (ERT) ]
  4. change in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs) [ Time Frame: 8 weeks (ERT-naive) or 13 weeks (ERT) ]

Information from the National Library of Medicine

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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.)
  • 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

Information from the National Library of Medicine

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 identifier (NCT number): NCT02262338

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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
ZKJM MC University of Mainz
Mainz, Germany
Institute of Human Genetics, National Inst of Health, University of the Philippines
Manila, Philippines
Sponsors and Collaborators
ArmaGen, Inc
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Study Director: Patrice Rioux, MD PhD ArmaGen, Inc
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Responsible Party: ArmaGen, Inc 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
Keywords provided by ArmaGen, Inc:
Hunter Syndrome
Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
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