A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

This study has been completed.

Study NCT00074984 Information provided by Genzyme

First Received on December 24, 2003. Last Updated on November 18, 2010 History of Changes

Results First Received: July 21, 2010

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Fabry Disease
Interventions:	Biological: Fabrazyme (agalsidase beta) Biological: Placebo

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
A total of 252 patients were screened for entry into the study and of these, 82 patients were eligible to be enrolled.

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
Placebo	Patients randomized to placebo are administered 1 mg/kg placebo intravenously every 2 weeks.
Fabrazyme (Agalsidase Beta)	Patients randomized to Fabrazyme (agalsidase beta) are administered 1mg/kg Fabrazyme (agalsidase beta) every 2 weeks.

Participant Flow: Overall Study

	Placebo	Fabrazyme (Agalsidase Beta)
STARTED	31	51
COMPLETED	28	43
NOT COMPLETED	3	8
Death	1	2
Withdrawal by Subject	2	3
Patient Withdrawn Per Protocol	0	3

Baseline Characteristics

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Reporting Groups

	Description
Placebo	Patients randomized to placebo are administered 1 mg/kg placebo intravenously every 2 weeks.
Fabrazyme (Agalsidase Beta)	Patients randomized to Fabrazyme (agalsidase beta) are administered 1mg/kg Fabrazyme (agalsidase beta) every 2 weeks.

Baseline Measures

	Placebo	Fabrazyme (Agalsidase Beta)	Total
Number of Participants [units: participants]	31	51	82
Age [units: participants]
<=18 years	0	0	0
Between 18 and 65 years	31	49	80
>=65 years	0	2	2
Age, Customized [units: participants]
<40 years	8	11	19
≥40 years	23	40	63
Age [units: years] Mean ± Standard Deviation	44.3 ± 9.23	46.9 ± 9.75	45.9 ± 9.58
Gender [units: participants]
Female	4	6	10
Male	27	45	72
Race/Ethnicity [units: participants]
Caucasian	27	45	72
Black	0	1	1
Hispanic	2	3	5
Asian	1	1	2
Other	1	1	2

Outcome Measures

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1. Primary:

Number of Participants Experiencing a Clinically Significant Renal, Cardiac or Cerebrovascular Event and/or Death in Fabrazyme (Agalsidase Beta) Patients as Compared to Placebo Patients [ Time Frame: up to 35 months ]

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2. Secondary:

Number of Participants Experiencing a Renal Event in Fabrazyme (Agalsidase Beta) Patients as Compared to Placebo Patients [ Time Frame: up to 35 months ]

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3. Secondary:

Slope of Estimated Glomerular Filtration Rate (eGFR) Comparing Placebo vs Fabrazyme (Agalsidase Beta) Patients [ Time Frame: up to 35 months ]

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4. Secondary:

Slope of Inverse Serum Creatinine Values Comparing Placebo vs Fabrazyme (Agalsidase Beta)Patients [ Time Frame: up to 35 months ]

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5. Secondary:

Neuropathic Pain as Assessed by Question 12 of the Brief Pain Inventory (BPI) Questionnaire (Pain at Its Worst) [ Time Frame: at 24 months ]

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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 Medical Information
Organization: Genzyme Corporation
phone: 800-745-4447

No publications provided by Genzyme

Publications automatically indexed to this study:

Banikazemi M, Bultas J, Waldek S, Wilcox WR, Whitley CB, McDonald M, Finkel R, Packman S, Bichet DG, Warnock DG, Desnick RJ; Fabry Disease Clinical Trial Study Group. Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. Ann Intern Med. 2007 Jan 16;146(2):77-86. Epub 2006 Dec 18.

Responsible Party:	Medical Monitor, Genzyme Coporation
ClinicalTrials.gov Identifier:	NCT00074984 History of Changes
Other Study ID Numbers:	AGAL-008-00
Study First Received:	December 24, 2003
Results First Received:	July 21, 2010
Last Updated:	November 18, 2010
Health Authority:	United States: Food and Drug Administration; Canada: Health Canada; Hungary: National Institute of Pharmacy; Czech Republic: State Institute for Drug Control; Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; United Kingdom: Medicines and Healthcare Products Regulatory Agency