Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study) - The INFORM Study

Genzyme, a Sanofi Company0 个研究点目标入组 15 人开始时间: 2015年4月24日最近更新: 2015年7月10日

适应症

试验速览

暂无简介。

•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
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 11
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•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

发起方

Genzyme, a Sanofi Company