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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01650779
First received: July 24, 2012
Last updated: January 22, 2014
Last verified: January 2014

July 24, 2012
January 22, 2014
August 2012
March 2013   (final data collection date for primary outcome measure)
  • Percent change from baseline at Months 2, 4, and 6 in plasma lyso-GL-3 (globotriaosylceramide) [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in plasma GL-3 [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in urine GL-3 [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in gastrointestinal (GI) symptoms (abdominal pain, abdominal distention, and bowel irregularities) [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01650779 on ClinicalTrials.gov Archive Site
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A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
Evaluation of Plasma Lyso GL-3 for Patients on Replagal Switching to Fabrazyme

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).

Not Provided
Interventional
Phase 4
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Biological: Fabrazyme (agalsidase beta)
Patients will receive commercial Fabrazyme® infusions in compliance with the label (0.9 to 1.1 mg/kg; i.e., "1 mg/kg" q2w) 1 mg/kg q2w (every two weeks)
Experimental: Fabrazyme
Intervention: Biological: Fabrazyme (agalsidase beta)
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
17
March 2013
March 2013   (final data collection date for primary outcome measure)

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
Male
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01650779
AGAL19412
No
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP