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

March 13, 2023 updated by: ArmaGen, Inc

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

Intervention / Treatment

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

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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
Time Frame: 24 months
Incidence and prevalence of adverse events
24 months

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ArmaGen, Inc

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

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

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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