A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

This study has been completed.

Sponsor:

Information provided by:

BioMarin Pharmaceutical

ClinicalTrials.gov Identifier:

NCT00299000

First received: March 2, 2006

Last updated: July 19, 2011

Last verified: July 2011

History of Changes

Results First Received: May 7, 2010

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Prevention
Conditions:	Mucopolysaccharidosis VI Maroteaux-Lamy Syndrome
Intervention:	Drug: Naglazyme

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
Global study sites were hospitals. First Enrollment: 08MAY2006 Last Dose: 30APR2009

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

Global study sites were hospitals.

First Enrollment: 08MAY2006

Last Dose: 30APR2009

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
Naglazyme, 1.0 mg/kg	weekly infusions for minimum of 52 weeks
Naglazyme, 2.0 mg/kg	weekly infusions for minimum of 52 weeks

Participant Flow: Overall Study

	Naglazyme, 1.0 mg/kg	Naglazyme, 2.0 mg/kg
STARTED	2	2
COMPLETED	2	2
NOT COMPLETED	0	0

Baseline Characteristics

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Reporting Groups

	Description
Naglazyme, 1.0 mg/kg	weekly infusions for minimum of 52 weeks
Naglazyme, 2.0 mg/kg	weekly infusions for minimum of 52 weeks
Total	Total of all reporting groups

Baseline Measures

	Naglazyme, 1.0 mg/kg	Naglazyme, 2.0 mg/kg	Total
Number of Participants [units: participants]	2	2	4
Age [units: participants]
<=18 years	2	2	4
Between 18 and 65 years	0	0	0
>=65 years	0	0	0
Age [units: months] Mean ± Standard Deviation	6.05 ± 3.89	12.4 ± 0.42	9.23 ± 4.31
Gender [units: participants]
Female	0	0	0
Male	2	2	4
Race/Ethnicity, Customized [units: participants]
White, non-Hispanic	1	1	2
Hispanic/Latino	1	0	1
Other	0	1	1
Region of Enrollment [units: participants]
United States	2	0	2
France	0	1	1
Portugal	0	1	1

Outcome Measures

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1. Primary:

Change in Height [ Time Frame: 52 weeks ]

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2. Primary:

Change in Weight [ Time Frame: 52 weeks ]

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3. Primary:

Change in Haed Circumference [ Time Frame: 52 weeks ]

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4. Secondary:

Change in Urinary Glycosaminoglycan Levels [ Time Frame: minimum 52 weeks of dosing ]

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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 the Sponsor in a joint publication. If such a multicenter publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from 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
Given the small number of patients (4) represented in this study, the outcomes observed in this study may not reflect or predict outcomes observed by physicians in clinical practice.

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

Responsible Party:	Celeste Decker, MD, BioMarin Pharmaceutical Inc.
ClinicalTrials.gov Identifier:	NCT00299000 History of Changes
Other Study ID Numbers:	ASB-008
Study First Received:	March 2, 2006
Results First Received:	May 7, 2010
Last Updated:	July 19, 2011
Health Authority:	United States: Food and Drug Administration

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