Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

This study has been completed.

First Received: November 6, 2002 Last Updated: August 24, 2009 History of Changes

Sponsor:	BioMarin Pharmaceutical
Information provided by:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT00048711

Purpose

The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)

Condition	Intervention	Phase
Mucopolysaccharidosis VI	Drug: N-acetylgalactosamine 4-sulfatase	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

12-minute walk test [ Time Frame: weeks 6, 12, 24, 48, 96 and 144 ] [ Designated as safety issue: No ]
urinary glycoaminoglycan(uGAG)levels [ Time Frame: weeks 1, 4, 6, 8, 12 ] [ Designated as safety issue: No ]

Study Start Date:	March 2002
Study Completion Date:	June 2006

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patient consent
Patient must be five years of age or older
Patient must have documented biochemical or genetic proof of MPS VI
Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
If female of childbearing potential, patient must have a negative pregnancy test

Exclusion Criteria:

Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
Pregnant or lactating patient
Patient has received an investigational drug within 30 days prior to study enrollment
Patient has been previously treated with rhASB
Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
Patient has a known hypersensitivity to rhASB or to components of the study drug
History of cancer (except low grade and fully resolved skin malignancy)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00048711

Locations

United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

Study Director:

Stuart J Sweidler, MD, Ph.D.

BioMarin Pharmaceutical

More Information

Additional Information:

Related Info This link exits the ClinicalTrials.gov site

No publications provided

Study ID Numbers:	ASB-01-04
Study First Received:	November 6, 2002
Last Updated:	August 24, 2009
ClinicalTrials.gov Identifier:	NCT00048711 History of Changes
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Metabolism, Inborn Errors
Mucopolysaccharidoses
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases

Connective Tissue Diseases
Mucinoses
Carbohydrate Metabolism, Inborn Errors
Mucopolysaccharidosis VI

ClinicalTrials.gov processed this record on November 27, 2009