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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02262338
• Date of registration: 02/10/2014
• Prospective Registration: Yes
• Primary sponsor: ArmaGen, Inc
• Public title: Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
• Scientific 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)
• Date of first enrolment: April 2015
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02262338
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Germany, Netherlands, Philippines, United States

Contacts

• Name: Patrice Rioux, MD PhD
• Affiliation: ArmaGen, Inc

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis II

Intervention(s)

Drug: AGT-182

Primary Outcome(s)

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(s)

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)]

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)]

Secondary ID(s)

AGT-182-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors