Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
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
1 other identifier
interventional
3
0 countries
N/A
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Nov 2015
Typical duration for phase_1
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2015
CompletedFirst Submitted
Initial submission to the registry
November 3, 2015
CompletedFirst Posted
Study publicly available on registry
November 5, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 19, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
August 2, 2018
CompletedSeptember 25, 2018
September 1, 2018
2.4 years
November 3, 2015
September 21, 2018
Conditions
Outcome Measures
Primary Outcomes (1)
Number of participants with adverse events as a measure of safety and tolerability
104 weeks (2 years)
Secondary Outcomes (4)
changes in urinary or plasma glycosaminoglycans (GAGs)
104 weeks (2 years)
change in liver size
104 weeks (2 years)
change in spleen size
104 weeks (2 years)
change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid
104 weeks (2 years)
Study Arms (1)
AGT-181
EXPERIMENTALAGT-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
Interventions
intravenous infusion over 3-4 hours
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- ArmaGen, Inclead
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Patrice Rioux, MD PhD
ArmaGen, Inc
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Masking Details
- Open Label
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 3, 2015
First Posted
November 5, 2015
Study Start
November 1, 2015
Primary Completion
March 19, 2018
Study Completion
August 2, 2018
Last Updated
September 25, 2018
Record last verified: 2018-09