Status:
COMPLETED
Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
Lead Sponsor:
ArmaGen, Inc
Conditions:
Mucopolysaccharidosis II
Eligibility:
MALE
18+ years
Phase:
PHASE1
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 ...
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 1 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 27 2017
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT02262338
Start Date
April 1 2015
End Date
March 27 2017
Last Update
September 18 2018
Active Locations (5)
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1
Children's Hospital Oakland
Oakland, California, United States, 94609
2
Children's Hospital of Orange County
Orange, California, United States, 92868
3
Emory University
Decatur, Georgia, United States, 30033
4
ZKJM MC University of Mainz
Mainz, Germany