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
  Purpose

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).


Condition Intervention Phase
Fabry Disease
Biological: Fabrazyme (agalsidase beta)
Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of Plasma Lyso GL-3 for Patients on Replagal Switching to Fabrazyme

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • 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 ]

Enrollment: 17
Study Start Date: August 2012
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fabrazyme 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)

  Eligibility

Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01650779

Locations
United States, Florida
Coral Springs, Florida, United States
United States, Georgia
Decatur, Georgia, United States
United States, Maryland
Baltimore, Maryland, United States
United States, Michigan
Grand Rapids, Michigan, United States
United States, Pennsylvania
Hellertown, Pennsylvania, United States
United States, Virginia
Fairfax, Virginia, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01650779     History of Changes
Other Study ID Numbers: AGAL19412
Study First Received: July 24, 2012
Last Updated: January 22, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Sanofi:
alpha-galactosidase A
a-GAL
Fabry
GL-3
Fabrazyme

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on April 23, 2014