- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02597114
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
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:
|
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
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AGT-181-103
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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