Mucopolysaccharidosis VI
Conditions
Brief summary
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.
Interventions
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patient consent * Patient must be five years of age or older * Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI * Leukocyte ASB enzyme activity level less than 20% of the normal range * Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly. * Ability to perform all protocol tests * Ability to stand independently for six minutes * Sexually active subjects must agree to use an adequate form of contraception
Exclusion criteria
* History of bone marrow transplantation * Pregnant or lactating patient * Use of an investigational drug or device within 30 days prior to study participation. * A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up * 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 |
|---|---|
| Lysosomal storage disease | — |
Countries
United States
Outcome results
None listed