A Study of Fabrazyme in Pediatric Patients With Fabry Disease

This study has been completed.

Study NCT00074958 Information provided by Genzyme

First Received: December 24, 2003 Last Updated: August 11, 2009 History of Changes

Study Type:	Interventional
Study Design:	Non-Randomized, Open Label, Uncontrolled, Single Group Assignment
Condition:	Fabry Disease
Intervention:	Biological: Fabrazyme (agalsidase beta)

Participant Flow

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	1.0 mg/kg of Fabrazyme given to the patients every 2 weeks

Participant Flow: Overall Study

Baseline Characteristics

Reporting Groups

	Description
Fabrazyme	1.0 mg/kg of Fabrazyme given to the patients every 2 weeks

Baseline Measures

	Fabrazyme
Number of Participants [units: participants]	16
Age [units: years] Mean ± Standard Deviation	12.1 ± 2.52
Gender [units: participants]
Female	2
Male	14

Outcome Measures

1. Primary:

Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin [ Baseline, Week 24 and Week 48 ]

Show Outcome Measure 1

2. Secondary:

Plasma GL-3 [ Baseline, Week 24 and Week 48 ]

Show Outcome Measure 2