Skip to main content
OpenTrials
Completed

NCT Number: NCT02597114

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Mucopolysaccharidosis I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Must have completed clinical trial AGT-181-102
  • Voluntary written consent by patient or legally responsible representative
  • All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
  • Negative pregnancy test (females)

Exclusion criteria

  • Refusal to complete screening evaluations.
  • Any medical condition or other circumstances that may significantly interfere with study compliance
  • Patient is pregnant or lactating
  • Clinically significant spinal cord compression, evidence of cervical instability.
  • Subject developed clinically relevant hypersensitivity to AGT-181

Treatment and study plan

AGT-181

Drug

intravenous infusion over 3-4 hours

Other names: HIRMAb-IDUA, fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase

Primary outcomes

  1. Number of participants with adverse events as a measure of safety and tolerability

    Time frame: 104 weeks (2 years)

Secondary outcomes

  1. changes in urinary or plasma glycosaminoglycans (GAGs)

    Time frame: 104 weeks (2 years)

  2. change in liver size

    Time frame: 104 weeks (2 years)

  3. change in spleen size

    Time frame: 104 weeks (2 years)

  4. change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid

    Time frame: 104 weeks (2 years)

Sponsors and collaborators

Lead sponsor

ArmaGen, Inc

Industry

Registry information

Official study title

An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
Nov 5, 2015
Registry last updated
Sep 25, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.