Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

This study has been completed.

First Received: August 20, 2003 Last Updated: September 17, 2009 History of Changes

Sponsor:	BioMarin Pharmaceutical
Information provided by:	BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:	NCT00067470

Purpose

The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

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

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

Change From Baseline in 12-minute Walk Test at 24 Weeks [ Time Frame: baseline and 24 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change From Baseline in 3-minute Stair Climb at 24 Weeks [ Time Frame: baseline and 24 weeks ] [ Designated as safety issue: No ]
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks [ Time Frame: baseline and 24 weeks ] [ Designated as safety issue: No ]

Enrollment:	39
Study Start Date:	September 2003
Estimated Study Completion Date:	March 2004

Arms	Assigned Interventions
Placebo: Placebo Comparator	Drug: Placebo
rhASB: Active Comparator	Drug: N-acetylgalactosamine 4-sulfatase

Eligibility

Ages Eligible for Study:	7 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria

Patient consent
Patient must be seven years of age or older
Patient must have documented biochemical or genetic proof of MPS VI
The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
Patient refuses or is unable to complete all screening evaluations
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
The patient has clinically significant spinal cord compression
The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00067470

Locations

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

Sponsors and Collaborators

BioMarin Pharmaceutical

More Information

Additional Information:

Related Info This link exits the ClinicalTrials.gov site

No publications provided

Study ID Numbers:	ASB-03-05
Study First Received:	August 20, 2003
Results First Received:	February 4, 2009
Last Updated:	September 17, 2009
ClinicalTrials.gov Identifier:	NCT00067470 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 09, 2009