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

Conditions: Fabry Disease
MedDRA version: 18.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2015-000697-35-Outside-EU/EEA

Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: Male

Target Recruitment: 15

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

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

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: 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.<br><br><br>;Secondary Objective: -;Primary end point(s): Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3);Timepoint(s) of evaluation of this end point: At Month 2, 4 and 6

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3)<br>Percent Change From Baseline in Urine GL-3<br>Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities)<br>;Timepoint(s) of evaluation of this end point: Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6<br>Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6<br>Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6