Status:
COMPLETED
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
Lead Sponsor:
Shire
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
Detailed Description
Fabry disease is an inherited, metabolic disease caused by mutations in the GALA gene. Patients with Fabry disease accumulate a complex glycosphingolipid named globotriaosylceramide (Gb3) in various t...
Eligibility Criteria
Inclusion
- \>18 years-old;
- Male:Fabry disease confirmed by deficiency of alfa galactosidase A activity OR Female:Fabry disease confirmed by a mutation of the alfa galactosidase A gene;
- ERT-naïve;
- LVM/h \> 50g/m2.7 for males and \>47 g/m2.7 for females;
- Negative pregnancy test at enrollment and contraception use required throughout study for female patients;
- Signed informed consent;
Exclusion
- Class IV heart failure;
- Clinically significant hypertension;
- Hemodynamically significant valvular stenosis or regurgitation;
- Morbid obesity;
- Known autosomal dominant sarcoplasmic contractile protein gene mutation;
- Treatment with any investigational drug or device within the 30 days;
- Unable to comply with the protocol as determined by the Investigator;
- Positive for hepatitis B, hepatitis C or HIV
Key Trial Info
Start Date :
December 29 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 5 2012
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT00864851
Start Date
December 29 2008
End Date
July 5 2012
Last Update
June 9 2021
Active Locations (12)
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1
AKDHC Tucson Access Center
Tucson, Arizona, United States, 85719
2
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
3
New York Unversity School of Medicine
New York, New York, United States, 10016
4
O & O Alpan, LLC
Springfield, Virginia, United States, 22152