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

Completed · Phase 4

Conditions studied: Fabry Disease

In brief

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes 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 analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.

Key facts

Study ID

NCT00081497

Run by

Genzyme, a Sanofi Company

People needed

67

Starts

2004-01-01

Expected to finish

2005-09-01

Last updated by the study team

2015-04-02

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-008-00 (NCT00074984)
• Patients must provide written informed consent prior to study participation
• Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception throughout the study

You may not qualify if…

• The patient was unable to complete AGAL-008-00 (NCT00074984)
• The patient has undergone kidney transplantation or is currently on dialysis
• The patient has diabetes mellitus or presence of confounding renal disease
• The patient has a clinically significant organic disease or an unstable condition that precludes participation
• The patient is unwilling to comply with the protocol requirements

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Cedars-Sinai Medical Center — Los Angeles, California, United States
• University of San Francisco — San Francisco, California, United States
• University of Connecticut Health Partners — West Hartford, Connecticut, United States
• Oncology Hematology Association — Coral Springs, Florida, United States
• Emory University School of Medicine — Atlanta, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics — Minneapolis, Minnesota, United States
• Children's Hospital — Buffalo, New York, United States
• Mount Sinai School of Medicine — New York, New York, United States
• University of Rochester School of Medicine — Rochester, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Washington School of Medicine — Seattle, Washington, United States
• Queen Elizabeth II Health Center — Halifax, Nova Scotia, Canada
• North York General Hospital — Toronto, Ontario, Canada
• Hopital du Sacre-Coeur de Montreal — Montreal, Quebec, Canada
• University Hospital — Prague, Czechia
• Sopron Megyei Jogu Varos Erzsebet Korhaz — Sopron, Hungary
• Klinika Chorob Metabolicznych Instytut — Warsaw, Poland
• Hope Hospital — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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