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

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

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.

Study Overview

• Status: Completed
• Conditions: Mucopolysaccharidosis I
• Intervention / Treatment: Drug: AGT-181

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 Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Brazil
  • RS
    • Porto Alegre, RS, Brazil, 90035-903
      • HCPA - Hospital das Clinicas de Porto Alegre

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AGT-181; Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein	Drug: AGT-181; Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
number of patients with adverse events as a measure of safety and tolerability	Incidence and prevalence of adverse events	24 months

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: ArmaGen, Inc
• Investigators: Study Director: Patrice P Rioux, MD PhD, ArmaGen, Inc

Publications and helpful links

General Publications

• Giugliani R, Giugliani L, de Oliveira Poswar F, Donis KC, Corte AD, Schmidt M, Boado RJ, Nestrasil I, Nguyen C, Chen S, Pardridge WM. Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial. Orphanet J Rare Dis. 2018 Jul 5;13(1):110. doi: 10.1186/s13023-018-0849-8.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2016
• Primary Completion (Actual): February 1, 2018
• Study Completion (Actual): February 1, 2018

Study Registration Dates

• First Submitted: February 27, 2017
• First Submitted That Met QC Criteria: March 1, 2017
• First Posted (Actual): March 6, 2017

Study Record Updates

• Last Update Posted (Actual): March 15, 2023
• Last Update Submitted That Met QC Criteria: March 13, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: MPS I; Hurler Syndrome
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Mucopolysaccharidosis I
• Other Study ID Numbers: AGT-181-101E

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No