Back to resultsExtension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
Primary Purpose
Mucopolysaccharidosis I
Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
AGT-181
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I
Eligibility Criteria
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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AGT-181
Arm Description
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
Outcomes
Primary Outcome Measures
Number of participants with adverse events as a measure of safety and tolerability
Secondary Outcome Measures
changes in urinary or plasma glycosaminoglycans (GAGs)
change in liver size
change in spleen size
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02597114
Brief Title
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
November 2015 (Actual)
Primary Completion Date
March 19, 2018 (Actual)
Study Completion Date
August 2, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ArmaGen, Inc
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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 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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Masking Description
Open Label
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AGT-181
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
AGT-181
Other Intervention Name(s)
HIRMAb-IDUA, fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase
Intervention Description
intravenous infusion over 3-4 hours
Primary Outcome Measure Information:
Title
Number of participants with adverse events as a measure of safety and tolerability
Time Frame
104 weeks (2 years)
Secondary Outcome Measure Information:
Title
changes in urinary or plasma glycosaminoglycans (GAGs)
Time Frame
104 weeks (2 years)
Title
change in liver size
Time Frame
104 weeks (2 years)
Title
change in spleen size
Time Frame
104 weeks (2 years)
Title
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
Time Frame
104 weeks (2 years)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Patrice Rioux, MD PhD
Organizational Affiliation
ArmaGen, Inc
Official's Role
Study Director
12. IPD Sharing Statement
Learn more about this trial
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
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