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

Phase 1

Completed

• Conditions: Mucopolysaccharidosis I
• Interventions: Drug: AGT-181

• Registration Number: NCT03071341
• Lead Sponsor: ArmaGen, Inc

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2016-10
• Study First Submitted: 2017-02-27
• Study First Submitted QC: 2017-03-01
• Study First Posted: 2017-03-06
• Primary Completion: 2018-02-01
• Study Completion: 2018-02-01
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-13
• Last Update Posted: 2023-03-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 19

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AGT-181	AGT-181	Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein

Primary Outcome Measures

Name	Time	Method
number of patients with adverse events as a measure of safety and tolerability	24 months	Incidence and prevalence of adverse events

Secondary Outcome Measures

Name	Time	Method
Plasma heparan sulfate and dermatan sulfate	24 months	Change in plasma heparan sulfate and dermatan sulfate
CSF heparan sulfate and dermatan sulfate	24 months	Change in CSF heparan sulfate and dermatan sulfate
Total urinary glycosaminoglycans (GAGs)	24 months	Change in total urinary glycosaminoglycans (GAGs)
Urinary heparan sulfate and dermatan sulfate	24 months	Change in urinary heparan sulfate and dermatan sulfate
liver and/or spleen volume	24 months	Change in liver and/or spleen volume measured by MRI

Trial Locations

Locations (1)

HCPA - Hospital das Clinicas de Porto Alegre; 🇧🇷 Porto Alegre, RS, Brazil