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

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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
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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

	rhASB	Placebo
STARTED	19	20
COMPLETED	19	19
NOT COMPLETED	0	1
Withdrawal by Subject	0	1

Baseline Characteristics

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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

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1. Primary:

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

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Measure Type	Primary
Measure Title	Change From Baseline in 12-minute Walk Test at 24 Weeks
Measure Description	Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Time Frame	baseline and 24 weeks
Safety Issue	No

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The efficacy analysis included all subjects except one from the placebo group who withdrew from the study prior to the week 24 assessment.

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

Measured Values

	rhASB	Placebo
Number of Participants Analyzed [units: participants]	19	19
Change From Baseline in 12-minute Walk Test at 24 Weeks [units: meters] Mean ± Standard Deviation	109 ± 154	26 ± 122

Statistical Analysis 1 for Change From Baseline in 12-minute Walk Test at 24 Weeks

Groups ^[1]	All groups
Method ^[2]	Regression, Linear
P Value ^[3]	0.025
Mean Difference (Net) ^[4]	92
Standard Error of the mean	± 40
95% Confidence Interval	( 11 to 172 )

[1]	Additional details about the analysis, such as null hypothesis and power calculation:
	No text entered.
[2]	Other relevant information, such as adjustments or degrees of freedom:
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[3]	Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
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[4]	Other relevant estimation information:
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2. Secondary:

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

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3. Secondary:

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

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The first publication of the results shall be made by Sponsor in a joint publication. If such a multi-center publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from their site individually, subject however to comply with the other terms of the agreement.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Not Applicable

Results Point of Contact:

Name/Title: Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
phone: 800-983-4587
e-mail: medinfo@bmrn.com

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