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.
- 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
- 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)