A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

Genzyme

Status and phase

Completed

Phase 4

Conditions

Fabry Disease

Treatments

Biological: Agalsidase beta

Study type

Interventional

Funder types

Industry

Identifiers

NCT01650779

AGAL19412

Details and patient eligibility

About

This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2 milligram per kilogram (mg/kg) every two weeks (q2w) and who are being switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w.

Enrollment

15 patients

Sex

Male

Volunteers

No Healthy Volunteers

Inclusion criteria

The participant and/or his parent/legal guardian is willing and able to provide signed informed consent, and the participant, if less than (<) 18 years of age, is willing to provide assent if deemed able to do so
Participant is male and has been treated with agalsidase alfa at 0.2 mg/kg q2w for the 12 months prior to switching to agalsidase beta
The participant has a confirmed diagnosis of Fabry disease by alfa-galactosidase A (alfa-GAL) activity and/or genotyping per local standards
The participant when switched to agalsidase beta receives the labeled dose, that is, 0.9 to 1.1 mg/kg (1 mg/kg) q2w, and must be willing to maintain the labeled dose for the duration of the study

Exclusion criteria

The participant is on dialysis or is post renal transplantation
The participant is in end-stage cardiac failure
The participant and/or his parent or legal guardian, in the opinion of the investigator, is unable to adhere to the requirements of the study
The participant has been switched from agalsidase alfa to agalsidase beta and does not have historical blood and urine samples