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

Genzyme

Status and phase: Completed; Phase 4

Conditions: Fabry Disease

Treatments: Biological: agalsidase beta

Study type: Interventional

Funder types: Industry

Identifiers

NCT00081497

AGAL02503

Details and patient eligibility

About

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.

Enrollment: 67 patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

67 participants in 1 patient group

Fabrazyme 1.0 mg/kg every 2 weeks

Experimental group

Description:

This is an open-label extension study to AGAL-008-00 (NCT00074984) and all patients received Fabrazyme treatment.

Treatment:

Biological: agalsidase beta