Completed

Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
What is being tested

Fabrazyme (agalsidase beta)

+ Placebo

Biological
Who is being recruted

Fabry Disease+17

+ Brain Diseases

+ Brain Diseases, Metabolic

Over 16 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 4
Interventional
Study Start: February 2001
See protocol details

Summary

Principal SponsorGenzyme, a Sanofi Company
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: February 1, 2001

Actual date on which the first participant was enrolled.

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.

Official TitleMulti-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease 
NCT00074984
Principal SponsorGenzyme, a Sanofi Company
Last updated: January 14, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

82 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.



Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 16 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Fabry DiseaseBrain DiseasesBrain Diseases, MetabolicCardiovascular DiseasesCentral Nervous System DiseasesCerebrovascular DisordersLipid Metabolism, Inborn ErrorsLipidosesMetabolic DiseasesMetabolism, Inborn ErrorsNervous System DiseasesSphingolipidosesVascular DiseasesLysosomal Storage DiseasesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornGenetic Diseases, InbornGenetic Diseases, X-LinkedLipid Metabolism DisordersCerebral Small Vessel Diseases

Criteria

Inclusion Criteria: * Patients must provide written informed consent * Patients must be at least 16 years old * Patients must have a current diagnosis of Fabry disease and have a clinical presentation consistent of Fabry disease (decreased sweating, Fabry pain, angiokeratoma, etc.) * Patients may not have received enzyme replacement therapy as a treatment for Fabry disease * Patients must have a documented plasma a-galactosidase A (aGAL) activity of \< 1.5 nmol/hr/mL or a documented leukocyte aGAL activity of \< 4 nmol/hr/mg * Patients must have one or more of the following: a serum creatinine measurement of 1.2 to 3 mg/dL (106.1 to 265 umol/L) OR estimated creatinine clearance \< 80 mL/min only if the patient's serum creatinine measurement is \< 1.2 mg/dL * 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 Exclusion Criteria: * Patient has undergone or is currently scheduled for kidney transplantation or is currently on dialysis * Patient has acute renal failure * Patient has participated in a study employing an investigational drug within 30 days of study entry * Patient has diabetes mellitus or presence of confounding renal disease * Patient has a history of transient ischemic attack (TIA) or ischemic stroke within 3 months of study entry documented by mild-to-moderate neurological deficit * Patient has critical coronary disease * Patient has congestive heart failure * Patient has severe residual neurological deficit that will confound the detection of new events as determined by an attending neurologist and/or Principal Investigator * Patient is unwilling to comply with the requirements of the protocol or the patient has a medical condition, serious intercurrent illness, or extenuating circumstances that would significantly decrease study compliance, including prescribed follow-up

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Active Comparator
Patients randomized to Fabrazyme (agalsidase beta).

Group II

Placebo
Patients randomized to placebo

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 26 locations

Suspended

University of Alabama at Birmingham

Birmingham, United StatesSee the location
Suspended

Cedars-Sinai Medical Center

Los Angeles, United States
Suspended

University of San Francisco

San Francisco, United States
Suspended

University of Connecticut Health Partners

Farmington, United States
Completed26 Study Centers