Expired Study
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Novato, California 94949


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.


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)

Study is Available At:

Original ID:




Secondary ID:

Study Acronym:

Brief Title:

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Official Title:

Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Overall Status:


Study Phase:

Phase 1



Minimum Age:


Maximum Age:


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

BioMarin Pharmaceutical

Oversight Authority:

United States: Food and Drug Administration

Reasons Why Stopped:

Study Type:


Study Design:

Allocation: Randomized, Endpoint Classification:

Number of Arms:


Number of Groups:


Total Enrollment:


Enrollment Type:

Overall Contact Information

Official Name:Stuart J Swiedler, MD, Ph.D.
Study Director
BioMarin Pharmaceutical

Study Dates

Start Date:September 2000
Completion Date:November 2005
Verification Date:November 2006
Last Changed Date:November 2, 2006
First Received Date:November 4, 2002

Study Outcomes

Outcome Type:Primary Outcome
Measure:Lysosomal storage disease
Safety Issues:False

Study Interventions

Intervention Type:Drug
Name:N-acetylgalactosamine 4-sulfatase

Study Arms

There are no available Study Arms

Study Agencies

Agency Class:Industry
Agency Type:Lead Sponsor
Agency Name:BioMarin Pharmaceutical

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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