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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-31

Study Completion Date

2018-02-01

Brief Summary

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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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Detailed Description

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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.

Conditions

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Mucopolysaccharidosis I

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101)

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AGT-181

Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein

Group Type EXPERIMENTAL

AGT-181

Intervention Type DRUG

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

Interventions

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AGT-181

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

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No