Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
Conditions
Brief summary
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
Interventions
DRUGlaronidase
dose of 0.58mg/kg body weight IV every week
Sponsors
BioMarin/Genzyme LLC
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (\<10% of the lower limit of normal). * For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion. * Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years.
Exclusion criteria
* Have previously received Aldurazyme without the collection of baseline samples as specified. * Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution. * Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant. * Are receiving chronic immunosuppressant therapy. * Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities. * Are pregnant or lactating * Have received investigational drug within 30 days prior to study enrollment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Urinary GAG (glycosaminoglycans) | Up to 4 years |
| Immunogenicity Testing | Up to 4 years |
Secondary
| Measure | Time frame |
|---|---|
| Safety | Up to 4 years |
Countries
United States
Outcome results
None listed