Mucopolysaccharidosis I
Conditions
Keywords
MPS I; Hurler Syndrome
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.
Interventions
DRUGAGT-181
Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein
Sponsors
ArmaGen, Inc
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention 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)
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| number of patients with adverse events as a measure of safety and tolerability | 24 months | Incidence and prevalence of adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Total urinary glycosaminoglycans (GAGs) | 24 months | Change in total urinary glycosaminoglycans (GAGs) |
| Urinary heparan sulfate and dermatan sulfate | 24 months | Change in urinary heparan sulfate and dermatan sulfate |
| Plasma heparan sulfate and dermatan sulfate | 24 months | Change in plasma heparan sulfate and dermatan sulfate |
| CSF heparan sulfate and dermatan sulfate | 24 months | Change in CSF heparan sulfate and dermatan sulfate |
| liver and/or spleen volume | 24 months | Change in liver and/or spleen volume measured by MRI |
Countries
Brazil
Outcome results
None listed