Mucopolysaccharidosis VI
Conditions
Brief summary
The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)
Interventions
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient consent * Patient must be five years of age or older * Patient must have documented biochemical or genetic proof of MPS VI * Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test * If female of childbearing potential, patient must have a negative pregnancy test
Exclusion criteria
* Patient is under consideration for or has undergone a successful bone marrow transplant (BMT). * Pregnant or lactating patient * Patient has received an investigational drug within 30 days prior to study enrollment * Patient has been previously treated with rhASB * Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance * Patient has a known hypersensitivity to rhASB or to components of the study drug * History of cancer (except low grade and fully resolved skin malignancy)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 12-minute walk test | weeks 6, 12, 24, 48, 96 and 144 |
| urinary glycoaminoglycan(uGAG)levels | weeks 1, 4, 6, 8, 12 |
Countries
United States
Outcome results
None listed