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

NCT00074971 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 58

Last updated 2013-12-04

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Fabrazyme (agalsidase beta)

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme Coorporation

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1999-10-31
Completion
2004-12-31

Countries

  • United States
  • France
  • Netherlands
  • Puerto Rico
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00074971 on ClinicalTrials.gov