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

This study has been completed.

Study NCT00081497 Information provided by Genzyme

First Received on April 14, 2004. Last Updated on August 18, 2010 History of Changes

Results First Received: December 17, 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: 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
Only those patients who successfully completed the AGAL-008-00 (NCT00074984) trial were eligible for participation in the AGAL02503 (NCT00081497) extension study.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups

	Description
Placebo/Fabrazyme	Patients who had been randomized to placebo during AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry to AGAL02503 (NCT00081497).
Fabrazyme/Fabrazyme	Patients who had been randomized to Fabrazyme in AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry into AGAL02503 (NCT00081497).

Participant Flow: Overall Study

	Placebo/Fabrazyme	Fabrazyme/Fabrazyme
STARTED	28 ^[1]	39 ^[2]
COMPLETED	25	37
NOT COMPLETED	3	2
Adverse Event	2	0
Death	0	1
Withdrawal by Subject	1	0
Non-compliance	0	1

[1]	Patients who were randomized to placebo in AGAL-008-00 (NCT00074984).
[2]	Patients who were randomized to Fabrazyme in AGAL-008-00 (NCT00074984).

Baseline Characteristics

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Reporting Groups

	Description
Placebo/Fabrazyme	Patients who had been randomized to placebo during AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry to AGAL02503 (NCT00081497).
Fabrazyme/Fabrazyme	Patients who had been randomized to Fabrazyme in AGAL-008-00 (NCT00074984) and were then transitioned to open-label Fabrazyme (1.0 mg/kg every 2 weeks) prior to or at entry into AGAL02503 (NCT00081497).

Baseline Measures

	Placebo/Fabrazyme	Fabrazyme/Fabrazyme	Total
Number of Participants [units: participants]	28	39	67
Age, Customized [units: participants]
<40 years	8	7	15
≥40 years	20	32	52
Age [units: years] Mean ± Standard Deviation	43.5 ± 9.31	46.6 ± 9.72	45.3 ± 9.60
Gender [units: participants]
Female	4	5	9
Male	24	34	58
Race/Ethnicity [units: participants]
Caucasian	24	35	59
Black	0	0	0
Hispanic	2	3	5
Asian	1	1	2
Other	1	0	1

Outcome Measures

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1. Primary:

Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods [ Time Frame: Placebo period AGAL-008-00 (up to 35 months) through Fabrazyme period AGAL02503 (18 months) ]

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2. Secondary:

Serum Creatinine at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]

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3. Secondary:

Estimated Glomerular Filtration Rate (eGFR) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme, 6, 12, and 18 months ]

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4. Secondary:

Plasma Globotriaosylceramide (GL-3) (Normal Plasma GL-3 Level is ≤ 7.03 µg/mL) at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]

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5. Secondary:

Proteinuria at Pre-Fabrazyme and 6, 12, and 18 Months [ Time Frame: Pre-Fabrazyme and 6, 12, and 18 months ]

Show Outcome Measure 5

6. Post-Hoc:

Differences in Slopes of Estimated Glomerular Filtration Rate (eGFR) Comparing Randomized Placebo vs Fabrazyme Patients (Based on the Original Randomization in AGAL-008-00 (NCT00074984)) by Baseline eGFR Subgroups of >60 and ≤60 mL/Min/1.73 m^2. [ Time Frame: Throughout study; 18 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, Principal Investigator (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
No valid conclusions can be made from the predefined primary efficacy analysis. The sponsor believes that it is more statistically appropriate to compare placebo patients with Fabrazyme patients as they were randomized in the original Phase 4 trial.

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:	NCT00081497 History of Changes
Other Study ID Numbers:	AGAL02503
Study First Received:	April 14, 2004
Results First Received:	December 17, 2008
Last Updated:	August 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