AGAL00800: A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients with Advanced Fabry Disease

This study has been completed.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00074984

Purpose

People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrayzme 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 ("globtriaosylceramide" 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.

Condition	Intervention	Phase
Fabry Disease	Drug: Fabrazyme (agalsidase beta)	Phase IV

Study Type: Interventional

Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Official Title: Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients with Fabry Disease

Further Study Details:

Expected Total Enrollment: 80

Study start: February 2001; Study completion: January 2004

Eligibility

Ages Eligible for Study: 16 Years and above, Genders Eligible for Study: Both

Criteria

Inclusion Criteria:

Patients must provide written informed consent

Patients must be at least 16 years old

Patients must have a current diagnosis of Fabry disease

Patients may not have received enzyme replacement therapy as a treatment for Fabry disease

Patients must have a documented plasma a-galactosidase A (aGAL) activity of < 1.5 nmol/hr/mL or a documented leukocyte aGAL activity of < 4 nmol/hr/mg

Patients must have one or more of the following: a serum creatinine measurement of 1.2 to 3 mg/dL (106.1 to 265 umol/L) OR estimated creatinine clearance < 80 mL/min only if the patient's serum creatinine measurement is < 1.2 mg/dL

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

Exclusion Criteria:

Patient has undergone or is currently scheduled for kidney transplantation or is currently on dialysis

Patient has acute renal failure

Patient has participated in a study employing an investigational drug within 30 days of study entry

Patient has diabetes mellitus or presence of confounding renal disease

Patient has a history of TIA or ischemic stroke within 3 months of study entry documented by mild-to-moderate neurological deficit

Patient has critical coronary disease

Patient has congestive heart failure

Patient has severe residual neurological deficit that will confound the detection of new events as determined by an attending neurologist and/or Principal Investigator

Patient is unwilling to comply with the requirements of the protocol or the patient has a medical condition, serious intercurrent illness, or extenuating circumstances that would significantly decrease study compliance, including prescribed follow-up

Location Information

Alabama
Alabama at Birmingham, Birmingham, Alabama, 35294, United States

California
Cedars-Sinai Medical Center, Los Angeles, California, 90048, United States
University of San Francisco, San Francisco, California, 94143, United States

Connecticut
University of Connecticut Health Partners, West Hartford, Connecticut, 06119, United States

Florida
Oncology Hematology Association, Coral Springs, Florida, 33065, United States

Georgia
Emory University School of Medicine, Atlanta, Georgia, 30322, United States

Illinois
Children's Memorial Hospital, Chicago, Illinois, 60614, United States

Kansas
University of Kansas Medical Center, Kansas City, Kansas, 66160, United States

Minnesota
Gene Therapy Center - Dept. of Pediatrics and Institute of Human Genetics, Minneapolis, Minnesota, 55455, United States

New York
Children's Hospital, Buffalo, New York, 14209, United States
Mount Sinai School of Medicine, New York, New York, 10029, United States
University of Rochester School of Medicine, Rochester, New York, 14642, United States

North Carolina
Duke University Medical Center, Durham, North Carolina, 27710, United States

Ohio
Children's Hospital Medical Center, Cincinnati, Ohio, 45229, United States

Pennsylvania
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh, Pittsburgh, Pennsylvania, 15261, United States

Texas
Baylor College of Medicine, Houston, Texas, 77030, United States

Washington
University of Washington School of Medicine, Seattle, Washington, 98195, United States

Canada, Nova Scotia
Queen Elizabeth II Health Center, Halifax, Nova Scotia, B3H 1V8, Canada

Canada, Ontario
North York General Hospital, Toronto, Ontario, M2K 1E1, Canada

Canada, Quebec
Hopital du Sacre-Coeur de Montreal, Montreal, Quebec, H4J 1C5, Canada

Czech Republic
University Hospital, Prague, 128 08, Czech Republic

Hungary
Sopron Megyei Jogu Varos Erzsebet Korhaz, Sopron, 9400, Hungary

Poland
Klinika Chorob Metabolicznych Instytut, Warsaw, 04-730, Poland

United Kingdom
Hope Hospital, Manchester, M6 8HD, United Kingdom

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL00800
Record first received: December 24, 2003
ClinicalTrials.gov Identifier: NCT00074984
Health Authority: United States: Food and Drug Administration