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

Tracking Information
First Received Date ^ICMJE	August 20, 2003
Last Updated Date	September 17, 2009
Start Date ^ICMJE	September 2003
Primary Completion Date
Current Primary Outcome Measures ^ICMJE (submitted: September 17, 2009)	Change From Baseline in 12-minute Walk Test at 24 Weeks [ Time Frame: baseline and 24 weeks ] [ Designated as safety issue: No ]
Original Primary Outcome Measures ^ICMJE (submitted: June 23, 2005)	Lysosomal Storage Disease
Change History	Complete list of historical versions of study NCT00067470 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE (submitted: September 17, 2009)	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 ]
Original Secondary Outcome Measures ^ICMJE (submitted: July 6, 2009)	3-Minute Stair Climb [ Time Frame: 24 weeks ] [ Designated as safety issue: No ] Urinary GAG (uGAG) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Descriptive Information
Brief Title ^ICMJE	Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Official Title ^ICMJE
Brief Summary	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.
Detailed Description
Study Phase	Phase III
Study Type ^ICMJE	Interventional
Study Design ^ICMJE	Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study
Condition ^ICMJE	Mucopolysaccharidosis VI
Intervention ^ICMJE	Drug: Placebo Drug: N-acetylgalactosamine 4-sulfatase
Study Arms / Comparison Groups
Publications *
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Enrollment ^ICMJE	39
Completion Date	March 2004
Primary Completion Date
Eligibility Criteria ^ICMJE	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
Gender	Both
Ages	7 Years and older
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT ID ^ICMJE	NCT00067470
Responsible Party
Study ID Numbers ^ICMJE	ASB-03-05
Study Sponsor ^ICMJE	BioMarin Pharmaceutical
Collaborators ^ICMJE
Investigators ^ICMJE
Information Provided By	BioMarin Pharmaceutical
Verification Date	September 2009
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP