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

Tracking Information

First Received Date ^ICMJE

April 14, 2004

Last Updated Date

September 26, 2008

Start Date ^ICMJE

January 2004

Primary Completion Date

September 2005 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

Original Primary Outcome Measures ^ICMJE

stabliization of renal function by estimating the difference within placebo patients' inverse serum creatinine slope in AGAL-008-00 versus the inverse serum creatinine slope while in study, AGAL02503

Change History

Complete list of historical versions of study NCT00081497 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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 ]

Original Secondary Outcome Measures ^ICMJE

Serum creatinine,
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],
plasma GL-3
proteinuria

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

Brief Summary

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.

Detailed Description

Study Phase

Phase IV

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Fabry Disease

Intervention ^ICMJE

Biological: Fabrazyme (agalsidase beta)

Study Arms / Comparison Groups

Experimental: Open-Label extension study to AGAL-008-00. All patients received Fabrazyme treatment.

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 ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

December 2005

Primary Completion Date

September 2005 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

16 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States, Canada, Hungary, Poland, United Kingdom

Administrative Information

NCT ID ^ICMJE

NCT00081497

Responsible Party

Medical Monitor, Genzyme Corporation

Study ID Numbers ^ICMJE

AGAL02503

Study Sponsor ^ICMJE

Genzyme

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director:

Bernard Bénichou, M.D.

Genzyme

Information Provided By

Genzyme

Verification Date

December 2005

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP