Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

This study has been completed.

Sponsor:

Collaborator:

BioMarin/Genzyme LLC

Information provided by:

Genzyme

ClinicalTrials.gov Identifier:

NCT00258011

First received: November 22, 2005

Last updated: July 8, 2009

Last verified: May 2009

History of Changes

Results First Received: January 13, 2009

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Mucopolysaccharidosis I Hurler Syndrome Hurler-Scheie Syndrome Scheie Syndrome
Intervention:	Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

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
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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
Aldurazyme (Laronidase) Treatment	Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.

Participant Flow: Overall Study

	Aldurazyme (Laronidase) Treatment
STARTED	3
COMPLETED	3
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Aldurazyme (Laronidase) Treatment	Patients received weekly infusions of Aldurazyme (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight (labeled dose) for up to 73 weeks.

Baseline Measures

	Aldurazyme (Laronidase) Treatment
Number of Participants [units: participants]	3
Age [units: years] Mean ± Standard Deviation	12.2 ± 12.57
Gender [units: participants]
Female	3
Male	0
Race/Ethnicity, Customized [units: participants]
Asian (Japanese)	3

Outcome Measures

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1. Primary:

Safety Evaluation [ Time Frame: Up to 73 Weeks ]

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2. Secondary:

Urinary Glycosaminoglycan (GAG) Excretion [ Time Frame: Up to 73 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: In multi-site studies, PI can publish after Genzyme publishes or 18 months after study completion. PI gives Genzyme a draft 60 days before publication. Genzyme can ask that confidential information be removed, and can defer publication another 60 days upon notifying PI that it will file a patent application on inventions contained in the draft.

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
Due to the rarity of the disease, this study is limited by its small sample size, lack of a control group and patients had variable treatment durations.

Results Point of Contact:

Name/Title: Genzyme Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00258011 History of Changes
Other Study ID Numbers:	ALID02205, JC0498
Study First Received:	November 22, 2005
Results First Received:	January 13, 2009
Last Updated:	July 8, 2009
Health Authority:	Japan: Ministry of Health, Labor and Welfare

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