A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Completed · Phase 3
Conditions studied: Fabry Disease
In brief
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.
Key facts
- Study ID
- NCT00074971
- Run by
- Genzyme, a Sanofi Company
- People needed
- 58
- Starts
- 1999-10-01
- Expected to finish
- 2004-12-01
- Last updated by the study team
- 2013-12-04
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Cedars-Sinai Medical Center — Los Angeles, California, United States
- University of California San Fransisco — San Francisco, California, United States
- Northwest Oncology & Hematology Associates — Coral Springs, Florida, United States
- Children's Memorial Hospital — Chicago, Illinois, United States
- University of Iowa Hospital and Clinics — Iowa City, Iowa, United States
- Greater Baltimore Medical Center — Baltimore, Maryland, United States
- Beth Israel Deaconess Medical Center — Boston, Massachusetts, United States
- Fetal Diagnostic and Imaging Center — Billings, Montana, United States
- University of New Mexico — Albuquerque, New Mexico, United States
- Mount Sinai School of Medicine — New York, New York, United States
- University of Rochester School of Medicine — Rochester, New York, United States
- Hart Family Practice — Hickory, North Carolina, United States
- Hematology/Oncology Associates of South Texas — San Antonio, Texas, United States
- University of Washington School of Medicine — Seattle, Washington, United States
- Hospital Edouard Herriot — Lyon, France
- Hospital Europeen Georges Pompidou — Paris, France
- Academisch Medisch Centrum — Amsterdam, Netherlands
- University of Puerto Rico — San Juan, Puerto Rico
- National Hospital for Neurology and Neurosurgery — London, United Kingdom
- Hope Hospital — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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