A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
Status: | Completed |
---|---|
Conditions: | Hematology, Metabolic |
Therapuetic Areas: | Hematology, Pharmacology / Toxicology |
Healthy: | No |
Age Range: | Any |
Updated: | 11/18/2012 |
Start Date: | August 2012 |
End Date: | February 2013 |
Contact: | Medical Information |
Email: | medinfo@genzyme.com |
Phone: | 800-745-4447 |
Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The Inform Study)
This is an exploratory study to evaluate changes in glycosphingolipid levels and other
(exploratory) Fabry disease parameters in male Fabry disease patients who were previously
treated with agalsidase alfa (Replagal®) 0.2 mg/kg q2w (every two weeks) and who are being
switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w (every two weeks).
Inclusion Criteria:
- The patient and/or his parent/legal guardian is willing and able to provide signed
informed consent, and the patient, if <18 years of age, is willing to provide assent
if deemed able to do so.
- Patient is male and has been treated with Replagal® at 0.2 mg/kg every 2 weeks
("q2w") for the 12 months prior to switching to Fabrazyme®.
- The Patient has a confirmed diagnosis of Fabry disease by αGal (α-galactosidase A)
activity and/or genotyping per local standards
- The patient when switched to Fabrazyme® receives the labeled dose, i.e., 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 patient is on dialysis or is post renal transplantation
- The patient is in end-stage cardiac failure.
- The patient and/or his parent or legal guardian, in the opinion of the Investigator,
is unable to adhere to the requirements of the study.
- The patient has been switched from Replagal® to Fabrazyme® and does not have
"historical" blood and urine samples
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