Mucopolysaccharidosis VI Clinical Trial
Official title:
Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Verified date | November 2006 |
Source | BioMarin Pharmaceutical |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 0 |
Est. completion date | November 2005 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
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) |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double-Blind, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | BioMarin Pharmaceutical Inc. | Novato | California |
Lead Sponsor | Collaborator |
---|---|
BioMarin Pharmaceutical |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Lysosomal storage disease |
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