A Study of Fabrazyme in Pediatric Patients With Fabry Disease - Study Results

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

	Fabrazyme
STARTED	16
COMPLETED	15
NOT COMPLETED	1
Adverse Event	1

Baseline Characteristics

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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

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1. Primary:

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

Measure Type	Primary
Measure Title	Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
Measure Description	Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Time Frame	Baseline, Week 24 and Week 48
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.
Intent to Treat (ITT) population – male patients only. 14 patients had skin biopsies performed at Baseline and Week 24 but only 5 patients had skin biopsies performed at Week 48.

Reporting Groups

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

Measured Values

	Fabrazyme
Number of Participants Analyzed [units: participants]	14
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin [units: patients]
None/Trace (0) Score at Baseline	2
None/Trace (0) Score at Week 24	14
None/Trace (0) Score at Week 48	5
Mild (1) Score at Baseline	0
Mild (1) Score at Week 24	0
Mild (1) Score at Week 48	0
Moderate (2) Score at Baseline	11
Moderate (2) Score at Week 24	0
Moderate (2) Score at Week 48	0
Severe (3) Score at Baseline	1
Severe (3) Score at Week 24	0
Severe (3) Score at Week 48	0

No statistical analysis provided for Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin

2. Secondary:

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

Measure Type	Secondary
Measure Title	Plasma GL-3
Measure Description	Plasma GL-3 values at Baseline, Week 24, and Week 48. Normal plasma GL-3 level is ≤ 7.03 µg/mL.
Time Frame	Baseline, Week 24 and Week 48
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.
ITT population. 16 male patients had plasma GL-3 values at Baseline and Week 24, while 15 male patients had plasma GL-3 values at Week 48. 2 female patients had plasma GL-3 values at Baseline, Week 24 and Week 48.

Reporting Groups

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

Measured Values

	Fabrazyme
Number of Participants Analyzed [units: participants]	16
Plasma GL-3 [units: µg/mL] Mean ± Standard Deviation
Male Patients at Baseline	15.4 ± 4.10
Female Patients at Baseline	4.9 ± 0.64
Overall at Baseline	14.1 ± 5.25
Males at Week 24	5.5 ± 0.95
Females at Week 24	3.6 ± 0.57
Overall at Week 24	5.2 ± 1.10
Males at Week 48	5.0 ± 1.00
Females at Week 48	4.4 ± 1.27
Overall at Week 48	4.9 ± 1.01

No statistical analysis provided for Plasma GL-3

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
The trial was not powered to demonstrate clinical benefit.

Results Point of Contact:

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

No publications provided by Genzyme

Publications automatically indexed to this study:

Wraith JE, Tylki-Szymanska A, Guffon N, Lien YH, Tsimaratos M, Vellodi A, Germain DP. Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease. J Pediatr. 2008 Apr;152(4):563-70, 570.e1. Epub 2007 Dec 3.

Responsible Party:	Genzyme Corporation ( Medical Monitor )
Study ID Numbers:	AGAL-016-01
Study First Received:	December 24, 2003
Results First Received:	March 3, 2009
Last Updated:	August 11, 2009
ClinicalTrials.gov Identifier:	NCT00074958 History of Changes
Health Authority:	United States: Food and Drug Administration