AGAL00599: A Study of the Safety and Efficacy of Fabrazyme in Patients with Fabry Disease

This study has been completed.

Sponsored By:	Genzyme
Information Provided By:	Genzyme
ClinicalTrials.gov Identifier:	NCT00074971

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 III

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Official Title: A Multi-Center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients with Fabry Disease

Further Study Details:

Primary Outcomes: Safety and efficacy; Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney

Secondary Outcomes: 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

Expected Total Enrollment: 58

Study Start: 1999-10; Study Completion: 2004-12

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-1-002-98)

Patients must provide written informed consent prior to study participation

Female patients must have a negative pregancy 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

Location Information

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

Florida
Northwest Oncology & Hematology Associates, Coral Springs, Florida, 33065, United States

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

Iowa
University of Iowa Hospital and Clinics, Iowa City, Iowa, 52242, United States

Maryland
Greater Baltimore Medical Center, Baltimore, Maryland, 21204, United States

Massachusetts
Beth Israel Deaconess Medical Center, Boston, Massachusetts, 02215, United States

Montana
Fetal Diagnostic and Imaging Center, Billings, Montana, 59101, United States

New Mexico
University of New Mexico, Albuquerque, New Mexico, 87131, United States

New York
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
Hart Family Practice, Hickory, North Carolina, 28601, United States

Texas
Hematology/Oncology Associates of South Texas, San Antonio, Texas, 78229, United States

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

France
Hospital Edouard Herriot, Lyon, Cedex 03, France
Hospital Europeen Georges Pompidou, Paris, Cedex 15, France

Netherlands
Academisch Medisch Centrum, Amsterdam, 1105 AZ, Netherlands

Puerto Rico
University of Puerto Rico, San Juan, 00935, Puerto Rico

United Kingdom
Hope Hospital, Manchester, M6 8HD, United Kingdom
National Hospital for Neurology and Neurosurgery, London, WC1N 3BG, United Kingdom

More Information

Fabrazyme® FDA-approved labeling information

Study ID Numbers: AGAL-005-99
ClinicalTrials.gov Identifier: NCT00074971
Health Authority: United States: Food and Drug Administration