A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
- Conditions
- Fabry Disease
- Registration Number
- NCT00074971
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 58
- 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
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Safety and efficacy Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (20)
University of Iowa Hospital and Clinics
🇺🇸Iowa City, Iowa, United States
Northwest Oncology & Hematology Associates
🇺🇸Coral Springs, Florida, United States
Hematology/Oncology Associates of South Texas
🇺🇸San Antonio, Texas, United States
Hope Hospital
🇬🇧Manchester, United Kingdom
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
Greater Baltimore Medical Center
🇺🇸Baltimore, Maryland, United States
University of California San Fransisco
🇺🇸San Francisco, California, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
University of Puerto Rico
🇵🇷San Juan, Puerto Rico
Hospital Edouard Herriot
🇫🇷Lyon, France
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
University of Rochester School of Medicine
🇺🇸Rochester, New York, United States
Hospital Europeen Georges Pompidou
🇫🇷Paris, France
Hart Family Practice
🇺🇸Hickory, North Carolina, United States
Academisch Medisch Centrum
🇳🇱Amsterdam, Netherlands
National Hospital for Neurology and Neurosurgery
🇬🇧London, United Kingdom
University of Washington School of Medicine
🇺🇸Seattle, Washington, United States
Fetal Diagnostic and Imaging Center
🇺🇸Billings, Montana, United States
University of New Mexico
🇺🇸Albuquerque, New Mexico, United States