Completed

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

What is being tested

Data Collection

Who is being recruted

Fabry Disease+19

+ Brain Diseases

+ Brain Diseases, Metabolic

Over 16 Years

+7 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 3

Interventional

Study Start: October 1999

See protocol details

Summary

Principal SponsorGenzyme, a Sanofi Company

Last updated: January 27, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: October 1, 1999

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 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.

Official TitleA 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

NCT00074971

Principal SponsorGenzyme, a Sanofi Company

Last updated: January 27, 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

58 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 ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesSphingolipidosesVascular DiseasesLysosomal Storage DiseasesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornGenetic Diseases, InbornGenetic Diseases, X-LinkedLipid Metabolism DisordersCerebral Small Vessel Diseases

Criteria

3 inclusion criteria required to participate

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

4 exclusion criteria prevent from participating

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

Study Plan

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

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 20 locations

Suspended

Cedars-Sinai Medical Center

Los Angeles, United StatesOpen Cedars-Sinai Medical Center in Google Maps

Suspended

University of California San Fransisco

San Francisco, United States

Suspended

Northwest Oncology & Hematology Associates

Coral Springs, United States

Suspended

Children's Memorial Hospital

Chicago, United States

Completed20 Study Centers