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

This Tabular View shows the required WHO registration data elements as marked by ^†

Tracking Information
First Received Date ^†	December 24, 2003
Last Updated Date	July 16, 2007
Start Date ^†	October 1999
Current Primary Outcome Measures ^† (submitted: July 16, 2007)	Safety and efficacy Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Original Primary Outcome Measures ^† (submitted: June 23, 2005)	Safety and efficacy Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Change History	Complete list of historical versions of study NCT00074971 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^† (submitted: July 16, 2007)	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
Original Secondary Outcome Measures ^† (submitted: June 23, 2005)	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

Descriptive Information
Brief Title ^†	A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
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
Brief Summary	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.
Detailed Description
Study Phase	Phase III
Study Type ^†	Interventional
Study Design ^†	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Condition ^†	Fabry Disease
Intervention ^†	Drug: Fabrazyme (agalsidase beta)
Study Arms / Comparison Groups
Publications *
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status ^†	Completed
Enrollment ^†	58
Completion Date	December 2004
Primary Completion Date
Eligibility 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
Gender	Both
Ages	16 Years and older
Accepts Healthy Volunteers	No
Contacts ^††
Location Countries ^†	United States, France, Netherlands, Puerto Rico, United Kingdom
Expanded Access Status

Administrative Information
NCT ID ^†	NCT00074971
Responsible Party
Secondary IDs ^††
Study Sponsor ^†	Genzyme
Collaborators ^††
Investigators ^†
Information Provided By	Genzyme
Verification Date	July 2005
^† Required WHO trial registration data element. ^†† WHO trial registration data element that is required only if it exists.