AGAL02503: A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease

This study is complete.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00081497

Purpose

People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown 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 (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 efficiacy 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, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease That Previously Participated in the AGAL-008-00 Study

Further Study Details:

Primary Outcomes: stabliization of renal function by estimating the difference within placebo patients' inverse serum creatinine slope in AGAL-008-00 versus the inverse serum creatinine slope while in study, AGAL02503

Secondary Outcomes:Serum creatinine,; estimated glomerular filtration rate, GFR [as estimated by the Modification of Diet in Renal Disease (MDRD) Study Group equation incorporating: serum creatinine, age, gender, race],; plasma GL-3; proteinuria

Total Enrollment: 70
Study Start: 2004-01; Study Completion: 2005-09

Eligibility

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

Criteria

Inclusion Criteria:

Patients must have successfully completed the previous double-blind study (AGAL-008-00)

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

Exclusion Criteria:

The patient was unable to complete AGAL-008-00

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

Location Information

Alabama
University of Alabama at Birmingham, Birmingham, Alabama, 35294-0006, 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, 66160-7233, United States

Minnesota
Gene Therapy Center - Department 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

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: AGAL02503
ClinicalTrials.gov Identifier: NCT00081497
Health Authority: United States: Food and Drug Administration