Status:
COMPLETED
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Fabry Disease
Eligibility:
All Genders
16+ years
Phase:
PHASE4
Brief Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to breakdown an...
Eligibility Criteria
Inclusion
- Patients must provide written informed consent
- Patients must be at least 16 years old
- Patients must have a current diagnosis of Fabry disease and have a clinical presentation consistent of Fabry disease (decreased sweating, Fabry pain, angiokeratoma, etc.)
- Patients may not have received enzyme replacement therapy as a treatment for Fabry disease
- Patients must have a documented plasma a-galactosidase A (aGAL) activity of \< 1.5 nmol/hr/mL or a documented leukocyte aGAL activity of \< 4 nmol/hr/mg
- Patients must have one or more of the following: a serum creatinine measurement of 1.2 to 3 mg/dL (106.1 to 265 umol/L) OR estimated creatinine clearance \< 80 mL/min only if the patient's serum creatinine measurement is \< 1.2 mg/dL
- Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception
Exclusion
- Patient has undergone or is currently scheduled for kidney transplantation or is currently on dialysis
- Patient has acute renal failure
- Patient has participated in a study employing an investigational drug within 30 days of study entry
- Patient has diabetes mellitus or presence of confounding renal disease
- Patient has a history of transient ischemic attack (TIA) or ischemic stroke within 3 months of study entry documented by mild-to-moderate neurological deficit
- Patient has critical coronary disease
- Patient has congestive heart failure
- Patient has severe residual neurological deficit that will confound the detection of new events as determined by an attending neurologist and/or Principal Investigator
- Patient is unwilling to comply with the requirements of the protocol or the patient has a medical condition, serious intercurrent illness, or extenuating circumstances that would significantly decrease study compliance, including prescribed follow-up
Key Trial Info
Start Date :
February 1 2001
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2004
Estimated Enrollment :
82 Patients enrolled
Trial Details
Trial ID
NCT00074984
Start Date
February 1 2001
End Date
January 1 2004
Last Update
December 27 2013
Active Locations (26)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
2
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
3
University of San Francisco
San Francisco, California, United States, 94143
4
University of Connecticut Health Partners
Farmington, Connecticut, United States, 06119