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

Sponsored by:	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

Genetics Home Reference related topics: cholesteryl ester storage disease Fabry disease Farber lipogranulomatosis L1 syndrome long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency

Drug Information available for: Agalsidase alfa alpha-Galactosidase

U.S. FDA Resources

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

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

Show 24 Study Locations

Sponsors and Collaborators

Genzyme

Investigators

Study Director:

Bernard Bénichou, M.D.

Genzyme

More Information

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

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
Health Authority:	United States: Food and Drug Administration

Keywords provided by Genzyme:

alpha-galactosidase A
a-GAL
r-haGAL

Fabry
GL-3
Fabrazyme

Study placed in the following topic categories:

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

Ceramide trihexosidosis
Genetic Diseases, Inborn
Fabry Disease
Genetic Diseases, X-Linked
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Lysosomal Storage Diseases, Nervous System
Nervous System Diseases

ClinicalTrials.gov processed this record on January 16, 2009