Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Completed · Phase 1
Conditions studied: Mucopolysaccharidosis VI
In brief
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.
Key facts
- Study ID
- NCT00048620
- Run by
- BioMarin Pharmaceutical
- Starts
- 2000-09-01
- Expected to finish
- 2005-11-01
- Last updated by the study team
- 2006-11-03
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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)
Where it is running
- BioMarin Pharmaceutical Inc. — Novato, California, United States
Full record on ClinicalTrials.gov
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