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Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome | Clinical Trials | Clareo Health

COMPLETEDPHASE1

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)

NCT02262338•ArmaGen, Inc

View on ClinicalTrials.gov

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.

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.

Eligibility

Age

18 - No limit years

Sex

MALE

Healthy Volunteers

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

Locations (5)

Children's Hospital Oakland

Oakland, California, United States

Children's Hospital of Orange County

Orange, California, United States

Emory University

Decatur, Georgia, United States

ZKJM MC University of Mainz

Mainz, Germany

Institute of Human Genetics, National Inst of Health, University of the Philippines

Manila, Philippines

Key Dates

Start Date

April 1, 2015

Primary Completion Date

March 27, 2017

Completion Date

March 27, 2017

Last Updated

September 18, 2018

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis II

Interventions

AGT-182

DRUG

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NCT02254863

AdrenoleukodystrophyBatten Disease+10 more

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ACTIVE_NOT_RECRUITINGPHASE1

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

NCT05422482

Mucopolysaccharidosis IIHunter Syndrome

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 18, 2018Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Inclusion Criteria

Exclusion Criteria

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

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