A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

This study has been completed.

Sponsor:

Information provided by:

Genzyme

ClinicalTrials.gov Identifier:

NCT00196716

First received: September 12, 2005

Last updated: August 11, 2009

Last verified: April 2007

History of Changes

Results First Received: December 5, 2008

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Fabry Disease
Intervention:	Biological: Fabrazyme (agalsidase beta)

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
Fabrazyme	Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months.

Participant Flow for 2 periods

Period 1: 1.0 mg/kg Period

	Fabrazyme
STARTED	21
COMPLETED	21
NOT COMPLETED	0

Period 2: 0.3 mg/kg Period

	Fabrazyme
STARTED	21
COMPLETED	21
NOT COMPLETED	0

Baseline Characteristics

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Reporting Groups

	Description
Fabrazyme	Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months.

Baseline Measures

	Fabrazyme
Number of Participants [units: participants]	21
Age [units: years] Mean ± Standard Deviation	34.0 ± 10.0
Gender [units: Participants]
Female	0
Male	21
Race/Ethnicity, Customized [units: Participants]
Caucasian	21

Outcome Measures

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1. Primary:

Globotriaosylceramide (GL-3) Clearance in Kidney Interstitial Capillary Endothelium [ Time Frame: Throughout study; 96 weeks ]

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2. Secondary:

Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium [ Time Frame: Throughout study ; 96 weeks ]

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3. Secondary:

Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Throughout study; 96 weeks ]

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4. Secondary:

Plasma Globotriaosylceramide (GL-3) [ Time Frame: Throughout study; 96 weeks ]

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5. Secondary:

Urine Globotriaosylceramide (GL-3) [ Time Frame: Throughout study, 96 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
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Results Point of Contact:

Name/Title: Genzyme MedInfo
Organization: Genzyme Corporation
phone: 800-745-4447
e-mail: medinfo@genzyme.com

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00196716 History of Changes
Other Study ID Numbers:	AGAL-017-01
Study First Received:	September 12, 2005
Results First Received:	December 5, 2008
Last Updated:	August 11, 2009
Health Authority:	Estonia: The State Agency of Medicine Czech Republic: State Institute for Drug Control Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Slovakia: State Institute for Drug Control

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