Back to resultsExtension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
Primary Purpose
Mucopolysaccharidosis I
Status
Completed
Phase
Phase 1
Locations
Brazil
Study Type
Interventional
Intervention
AGT-181
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring MPS I; Hurler Syndrome
Eligibility Criteria
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
Sites / Locations
- HCPA - Hospital das Clinicas de Porto Alegre
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AGT-181
Arm Description
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein
Outcomes
Primary Outcome Measures
number of patients with adverse events as a measure of safety and tolerability
Incidence and prevalence of adverse events
Secondary Outcome Measures
Total urinary glycosaminoglycans (GAGs)
Change in total urinary glycosaminoglycans (GAGs)
Urinary heparan sulfate and dermatan sulfate
Change in urinary heparan sulfate and dermatan sulfate
Plasma heparan sulfate and dermatan sulfate
Change in plasma heparan sulfate and dermatan sulfate
CSF heparan sulfate and dermatan sulfate
Change in CSF heparan sulfate and dermatan sulfate
liver and/or spleen volume
Change in liver and/or spleen volume measured by MRI
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03071341
Brief Title
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
Official Title
An Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Patients With Mucopolysaccharidosis I Who Were Previously Enrolled in Studies With AGT-181
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
October 2016 (Actual)
Primary Completion Date
February 1, 2018 (Actual)
Study Completion Date
February 1, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ArmaGen, Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
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 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.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I
Keywords
MPS I; Hurler Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Patients will be assigned to 1.0, 3.0 or 6.0 mg/kg (same dose taken during the previous study, AGT-181-101)
Masking
None (Open Label)
Allocation
N/A
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AGT-181
Arm Type
Experimental
Arm Description
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) fusion protein
Intervention Type
Drug
Intervention Name(s)
AGT-181
Intervention Description
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein
Primary Outcome Measure Information:
Title
number of patients with adverse events as a measure of safety and tolerability
Description
Incidence and prevalence of adverse events
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Total urinary glycosaminoglycans (GAGs)
Description
Change in total urinary glycosaminoglycans (GAGs)
Time Frame
24 months
Title
Urinary heparan sulfate and dermatan sulfate
Description
Change in urinary heparan sulfate and dermatan sulfate
Time Frame
24 months
Title
Plasma heparan sulfate and dermatan sulfate
Description
Change in plasma heparan sulfate and dermatan sulfate
Time Frame
24 months
Title
CSF heparan sulfate and dermatan sulfate
Description
Change in CSF heparan sulfate and dermatan sulfate
Time Frame
24 months
Title
liver and/or spleen volume
Description
Change in liver and/or spleen volume measured by MRI
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Patrice P Rioux, MD PhD
Organizational Affiliation
ArmaGen, Inc
Official's Role
Study Director
Facility Information:
Facility Name
HCPA - Hospital das Clinicas de Porto Alegre
City
Porto Alegre
State/Province
RS
ZIP/Postal Code
90035-903
Country
Brazil
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
29976218
Citation
Giugliani R, Giugliani L, de Oliveira Poswar F, Donis KC, Corte AD, Schmidt M, Boado RJ, Nestrasil I, Nguyen C, Chen S, Pardridge WM. Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial. Orphanet J Rare Dis. 2018 Jul 5;13(1):110. doi: 10.1186/s13023-018-0849-8.
Results Reference
result
Learn more about this trial
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
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