Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

September 21, 2018 updated by: ArmaGen, Inc

An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102

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 study is an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Study Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 1

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

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Must have completed clinical trial AGT-181-102
  • Voluntary written consent by patient or legally responsible representative
  • All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
  • Negative pregnancy test (females)

Exclusion Criteria:

  • Refusal to complete screening evaluations.
  • Any medical condition or other circumstances that may significantly interfere with study compliance
  • Patient is pregnant or lactating
  • Clinically significant spinal cord compression, evidence of cervical instability.
  • Subject developed clinically relevant hypersensitivity 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: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: AGT-181
AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study
intravenous infusion over 3-4 hours
Other Names:
  • fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase
  • HIRMAb-IDUA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with adverse events as a measure of safety and tolerability
Time Frame: 104 weeks (2 years)
104 weeks (2 years)

Secondary Outcome Measures

Outcome Measure
Time Frame
changes in urinary or plasma glycosaminoglycans (GAGs)
Time Frame: 104 weeks (2 years)
104 weeks (2 years)
change in liver size
Time Frame: 104 weeks (2 years)
104 weeks (2 years)
change in spleen size
Time Frame: 104 weeks (2 years)
104 weeks (2 years)
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
Time Frame: 104 weeks (2 years)
104 weeks (2 years)

Collaborators and Investigators

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

Sponsor

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)

November 1, 2015

Primary Completion (ACTUAL)

March 19, 2018

Study Completion (ACTUAL)

August 2, 2018

Study Registration Dates

First Submitted

November 3, 2015

First Submitted That Met QC Criteria

November 3, 2015

First Posted (ESTIMATE)

November 5, 2015

Study Record Updates

Last Update Posted (ACTUAL)

September 25, 2018

Last Update Submitted That Met QC Criteria

September 21, 2018

Last Verified

September 1, 2018

More Information

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.

Clinical Trials on Mucopolysaccharidosis I

Clinical Trials on AGT-181

3
Subscribe