Clinical trial NCT01650779: A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
Official title: Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Fabry Disease
Keywords: alpha-galactosidase A
a-GAL
Fabry
GL-3
Fabrazyme
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2012-07-26
Enrollment: 15
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6
timeFrame: Baseline, Month 2, 4, 6
Secondary outcomes:
measure: Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6
timeFrame: Baseline, Month 2, 4, 6
measure: Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6
timeFrame: Baseline, Month 2, 4, 6
measure: Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6
timeFrame: Baseline, Month 2, 4, 6
Other outcomes:
Locations:
city: Coral Springs
country: United States
state: Florida
city: Decatur
country: United States
state: Georgia
city: Baltimore
country: United States
state: Maryland
city: Grand Rapids
country: United States
state: Michigan
city: Hellertown
country: United States
state: Pennsylvania
city: Fairfax
country: United States
state: Virginia
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: