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

This study has been completed.

Study NCT00067470 Information provided by BioMarin Pharmaceutical

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

Study Type:	Interventional
Study Design:	Randomized, Double-Blind, Placebo Control, Parallel Assignment
Condition:	Mucopolysaccharidosis VI
Interventions:	Drug: Placebo Drug: N-acetylgalactosamine 4-sulfatase

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Study Locations:Oakland, CA, USA Mainz, Germany Manchester, England Porto Alegre, Brazil Lyon, France Porto, Portugal Study Duration: 24 weeks First enrollment: 21 July 2003 Last Dose Given: 1 April 2004 Last Assessment: 8 April 2004

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations

Study Locations:Oakland, CA, USA Mainz, Germany Manchester, England Porto Alegre, Brazil Lyon, France Porto, Portugal

Study Duration: 24 weeks

First enrollment: 21 July 2003

Last Dose Given: 1 April 2004

Last Assessment: 8 April 2004

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups

	Description
rhASB	Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
Placebo	Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB

Participant Flow: Overall Study

Baseline Characteristics

Reporting Groups

	Description
rhASB	Intravenous (IV) recombinant human arylsulfatase(rhASB) at 1.0 mg/kg
Placebo	Intravenous (IV) placebo solution at a volume equivalent to that needed for a 1.0 mg/kg dose of rhASB

Baseline Measures

	rhASB	Placebo	Total
Number of Participants [units: participants]	19	20	39
Age [units: years] Mean ± Standard Deviation	13.7 ± 6.47	10.7 ± 4.35	12.2 ± 5.62
Gender [units: participants]
Female	12	14	26
Male	7	6	13
Race/Ethnicity, Customized [units: participants]
White, non-Hispanic	15	9	24
Hispanic	1	3	4
Black	1	2	3
Asian	1	1	2
Indigenous	1	1	2
Other	0	4	4
Region of Enrollment [units: participants]
United States	2	4	6
Germany	4	4	8
United Kingdom	3	3	6
Brazil	4	4	8
France	2	3	5
Portugal	4	2	6

Outcome Measures

1. Primary:

Change From Baseline in 12-minute Walk Test at 24 Weeks [ baseline and 24 weeks ]

Show Outcome Measure 1

2. Secondary:

Change From Baseline in 3-minute Stair Climb at 24 Weeks [ baseline and 24 weeks ]

Show Outcome Measure 2

3. Secondary:

Change From Baseline in Urinary GAG (uGAG) at 24 Weeks [ baseline and 24 weeks ]

Show Outcome Measure 3