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:	NCT00074971

Purpose

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove 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	Drug: Fabrazyme (agalsidase beta)	Phase III

Genetics Home Reference related topics: Chanarin-Dorfman syndrome 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 succinic semialdehyde dehydrogenase deficiency

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Multi-Center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease

Further study details as provided by Genzyme:

Primary Outcome Measures:

Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney

Secondary Outcome Measures:

Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
Physician's assessment of Fabry Symptoms and pain medication

Estimated Enrollment:	58
Study Start Date:	October 1999
Estimated Study Completion Date:	December 2004

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-1-002-98)
Patients must provide written informed consent prior to study participation
Female patients must have a negative pregnancy test prior to each dosing and use a medically accepted method of contraception throughout the study

Exclusion criteria:

Patient has undergone kidney transplant or is currently on dialysis
Patient is pregnant or lactating
Patient is unwilling to comply with the requirements of the protocol
Patient has a clinically significant organic disease (with the exception of symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would preclude participation in the study

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00074971

Show 20 Study Locations

Sponsors and Collaborators

Genzyme

More Information

Additional Information:

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

No publications provided

Study ID Numbers:	AGAL-005-99
Study First Received:	December 24, 2003
Last Updated:	July 16, 2007
ClinicalTrials.gov Identifier:	NCT00074971 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Genzyme:

a-Galactosidase A
aGAL
r-haGAL

Fabry
GL-3
Fabrazyme

Study placed in the following topic categories:

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

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

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 May 06, 2009