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

Genzyme

Status and phase: Completed; Phase 3

Conditions: Fabry Disease

Treatments: Drug: Fabrazyme (agalsidase beta)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00074971

AGAL-005-99

Details and patient eligibility

About

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.

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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 pregnancy 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)