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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
19
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein
HCPA - Hospital das Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
number of patients with adverse events as a measure of safety and tolerability
Incidence and prevalence of adverse events
Time frame: 24 months
Total urinary glycosaminoglycans (GAGs)
Change in total urinary glycosaminoglycans (GAGs)
Time frame: 24 months
Urinary heparan sulfate and dermatan sulfate
Change in urinary heparan sulfate and dermatan sulfate
Time frame: 24 months
Plasma heparan sulfate and dermatan sulfate
Change in plasma heparan sulfate and dermatan sulfate
Time frame: 24 months
CSF heparan sulfate and dermatan sulfate
Change in CSF heparan sulfate and dermatan sulfate
Time frame: 24 months
liver and/or spleen volume
Change in liver and/or spleen volume measured by MRI
Time frame: 24 months
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