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NCT Number: NCT03071341

Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

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 is a long term safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will also be collected.

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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

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

HCPA - Hospital das Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

About this study

AGT-181-101E is a safety and tolerability study of AGT-181 in patients with MPS I who completed the previous 26-week study, AGT-181-101. Information on the biological activity of the investigational drug will be collected as well as longitudinal safety information.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • 2 years of age or older (and less than 18)
  • Must have been previously enrolled in study AGT-181-101
  • Written consent and assent as required
  • Female patients must not be pregnant, willing to utilize appropriate birth control methods and undergo pregnancy testing during the study

Exclusion criteria

  • Refusal to complete all assessments
  • Pregnant or Lactating
  • Received investigational drug within 1 year prior to study enrollment
  • Medical condition or extenuating circumstance that, in the opinion of the investigator, may interfere with study compliance
  • Clinically significant spinal cord compression or evidence of cervical instability (i.e. expected to require intervention during study participation)
  • Has developed clinically relevant hypersensitivity/anaphylactoid reactions to AGT-181

Treatment and study plan

AGT-181

Drug

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

Primary outcomes

  1. number of patients with adverse events as a measure of safety and tolerability

    Time frame: 24 months

    Incidence and prevalence of adverse events

Secondary outcomes

  1. Total urinary glycosaminoglycans (GAGs)

    Time frame: 24 months

    Change in total urinary glycosaminoglycans (GAGs)

  2. Urinary heparan sulfate and dermatan sulfate

    Time frame: 24 months

    Change in urinary heparan sulfate and dermatan sulfate

  3. Plasma heparan sulfate and dermatan sulfate

    Time frame: 24 months

    Change in plasma heparan sulfate and dermatan sulfate

  4. CSF heparan sulfate and dermatan sulfate

    Time frame: 24 months

    Change in CSF heparan sulfate and dermatan sulfate

  5. liver and/or spleen volume

    Time frame: 24 months

    Change in liver and/or spleen volume measured by MRI

Sponsors and collaborators

Lead sponsor

ArmaGen, Inc

Industry

Registry information

Official study title

An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Mar 6, 2017
Registry last updated
Mar 15, 2023

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.

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