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A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

NCT00074984 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 82

Last updated 2013-12-27

Study results available
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Summary

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) 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 ("globotriaosylceramide" 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

BIOLOGICAL

Fabrazyme (agalsidase beta)

1mg/kg Fabrazyme (agalsidase beta) every 2 weeks

BIOLOGICAL

Placebo

1 mg/kg placebo intravenously every 2 weeks

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme Coorporation

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2001-02-28
Primary Completion
2004-01-31
Completion
2004-01-31

Countries

  • United States
  • Canada
  • Czechia
  • Hungary
  • Poland
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
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 NCT00074984 on ClinicalTrials.gov