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

December 2, 2013 updated by: Genzyme, a Sanofi Company

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment

58

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Lyon, France, Cedex 03
        • Hospital Edouard Herriot
      • Paris, France, Cedex 15
        • Hospital Europeen Georges Pompidou
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academisch Medisch Centrum
  • Puerto Rico
      • San Juan, Puerto Rico, 00935
        • University of Puerto Rico
  • United Kingdom
      • London, United Kingdom, WC1N 3BG
        • National Hospital For Neurology and Neurosurgery
      • Manchester, United Kingdom, M6 8HD
        • Hope Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center
      • San Francisco, California, United States, 94143
        • University of California San Fransisco
    • Florida
      • Coral Springs, Florida, United States, 33065
        • Northwest Oncology & Hematology Associates
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospital and Clinics
    • Maryland
      • Baltimore, Maryland, United States, 21204
        • Greater Baltimore Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center
    • Montana
      • Billings, Montana, United States, 59101
        • Fetal Diagnostic and Imaging Center
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131
        • University of New Mexico
    • New York
      • New York, New York, United States, 10029
        • Mount Sinai School of Medicine
      • Rochester, New York, United States, 14642
        • University of Rochester School of Medicine
    • North Carolina
      • Hickory, North Carolina, United States, 28601
        • Hart Family Practice
    • Texas
      • San Antonio, Texas, United States, 78229
        • Hematology/Oncology Associates of South Texas
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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

Exclusion criteria:

  • 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

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

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Safety and efficacy
Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney

Secondary Outcome Measures

Outcome Measure
Changes in McGill Pain Questionnaire
Autonomic status
Glomerular filtration
Functional assessment of urinary protein excretion Ophthalmic changes
SF-36 Health Survey
Physician's assessment of Fabry Symptoms and pain medication

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 1999

Primary Completion

December 6, 2022

Study Completion

December 1, 2004

Study Registration Dates

First Submitted

December 24, 2003

First Submitted That Met QC Criteria

December 24, 2003

First Posted (Estimate)

December 25, 2003

Study Record Updates

Last Update Posted (Estimate)

December 4, 2013

Last Update Submitted That Met QC Criteria

December 2, 2013

Last Verified

December 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGAL-005-99

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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