A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease - Full Text View

Sponsor:	Genzyme
Information provided by:	Genzyme
ClinicalTrials.gov Identifier:	NCT00081497

Purpose

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and removes certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.

Condition	Intervention	Phase
Fabry Disease	Biological: Fabrazyme (agalsidase beta)	Phase IV

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Fabry disease Farber lipogranulomatosis L1 syndrome primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

stabilization of renal function with Fabrazyme by estimating the difference within placebo patients' inverse serum creatinine slope while in AGAL-008-00 versus the inverse serum creatinine slope while in the extension study (AGAL02503) [ Time Frame: throughout study 18 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Serum creatinine, [ Time Frame: throughout study 18 months ] [ Designated as safety issue: No ]
estimated glomerular filtration rate, GFR [as estimated by the Modification of Diet in Renal Disease (MDRD)Study Group equation incorporating: serum creatinine, age, gender, race] [ Time Frame: throughout study 18 months ] [ Designated as safety issue: No ]
plasma GL-3 [ Time Frame: throughout study 18 months ] [ Designated as safety issue: No ]
proteinuria [ Time Frame: throughout study 18 months ] [ Designated as safety issue: No ]

Enrollment:	70
Study Start Date:	January 2004
Study Completion Date:	December 2005
Primary Completion Date:	September 2005 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental Open-Label extension study to AGAL-008-00. All patients received Fabrazyme treatment.	Biological: Fabrazyme (agalsidase beta) 1 mg/kg every 2 weeks

Eligibility

Ages Eligible for Study:	16 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients must have successfully completed the previous double-blind study (AGAL-008-00)
Patients must provide written informed consent prior to study participation
Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study

Exclusion Criteria:

The patient was unable to complete AGAL-008-00
The patient has undergone kidney transplantation or is currently on dialysis
The patient has diabetes mellitus or presence of confounding renal disease
The patient has a clinically significant organic disease or an unstable condition that precludes participation
The patient is unwilling to comply with the protocol requirements

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00081497

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Locations

United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294-0006
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
University of San Francisco
San Francisco, California, United States, 94143
United States, Connecticut
University of Connecticut Health Partners
West Hartford, Connecticut, United States, 06119
United States, Florida
Oncology Hematology Association
Coral Springs, Florida, United States, 33065
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160-7233
United States, Minnesota
Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics
Minneapolis, Minnesota, United States, 55455
United States, New York
Children's Hospital
Buffalo, New York, United States, 14209
Mount Sinai School of Medicine
New York, New York, United States, 10029
University of Rochester School of Medicine
Rochester, New York, United States, 14642
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15261
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Washington
University of Washington School of Medicine
Seattle, Washington, United States, 98195
Canada, Nova Scotia
Queen Elizabeth II Health Center
Halifax, Nova Scotia, Canada, B3H 1V8
Canada, Ontario
North York General Hospital
Toronto, Ontario, Canada, M2K 1E1
Canada, Quebec
Hopital du Sacre-Coeur de Montreal
Montreal, Quebec, Canada, H4J 1C5
Hungary
Sopron Megyei Jogu Varos Erzsebet Korhaz
Sopron, Hungary, 9400
Poland
Klinika Chorob Metabolicznych Instytut
Warsaw, Poland, 04-730
United Kingdom
Hope Hospital
Manchester, United Kingdom, M6 8HD

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Bernard Bénichou, M.D.

Genzyme

More Information

Additional Information:

US FDA Approved Full Prescribing Information for Fabrazyme® This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Genzyme Corporation ( Medical Monitor )
Study ID Numbers:	AGAL02503
Study First Received:	April 14, 2004
Last Updated:	September 26, 2008
ClinicalTrials.gov Identifier:	NCT00081497 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Genzyme:

alpha-galactosidase A
a-GAL
r-haGAL

Fabry
GL-3
Fabrazyme

Additional relevant MeSH terms:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases

Metabolism, Inborn Errors
Genetic Diseases, Inborn
Fabry Disease
Genetic Diseases, X-Linked
Brain Diseases, Metabolic, Inborn
Lipidoses
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on November 27, 2009