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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00074971
• Other study ID #: AGAL-005-99
• Status: Completed
• Phase: Phase 3
• First received: December 24, 2003
• Last updated: December 2, 2013
• Start date: October 1999
• Est. completion date: December 2004

Study information

• Verified date: December 2013
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 58
• Est. completion date: December 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• Fabrazyme (agalsidase beta)

Locations

Country	Name	City	State
France	Hospital Edouard Herriot	Lyon
France	Hospital Europeen Georges Pompidou	Paris
Netherlands	Academisch Medisch Centrum	Amsterdam
Puerto Rico	University of Puerto Rico	San Juan
United Kingdom	National Hospital for Neurology and Neurosurgery	London
United Kingdom	Hope Hospital	Manchester
United States	University of New Mexico	Albuquerque	New Mexico
United States	Greater Baltimore Medical Center	Baltimore	Maryland
United States	Fetal Diagnostic and Imaging Center	Billings	Montana
United States	Beth Israel Deaconess Medical Center	Boston	Massachusetts
United States	Children's Memorial Hospital	Chicago	Illinois
United States	Northwest Oncology & Hematology Associates	Coral Springs	Florida
United States	Hart Family Practice	Hickory	North Carolina
United States	University of Iowa Hospital and Clinics	Iowa City	Iowa
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	Mount Sinai School of Medicine	New York	New York
United States	University of Rochester School of Medicine	Rochester	New York
United States	Hematology/Oncology Associates of South Texas	San Antonio	Texas
United States	University of California San Fransisco	San Francisco	California
United States	University of Washington School of Medicine	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  France,  Netherlands,  Puerto Rico,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and efficacy
Primary	Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
Secondary	Changes in McGill Pain Questionnaire
Secondary	Autonomic status
Secondary	Glomerular filtration
Secondary	Functional assessment of urinary protein excretion Ophthalmic changes
Secondary	SF-36 Health Survey
Secondary	Physician's assessment of Fabry Symptoms and pain medication