Status:
COMPLETED
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Fabry Disease
Eligibility:
MALE
16+ years
Phase:
PHASE2
Brief Summary
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. This enzyme helps to break down and remove certain types of ...
Eligibility Criteria
Inclusion
- Have clinical manifestations of Fabry disease
- All patients have to have a plasma αGAL activity of \< 1.5 nmol/hr/mL or a documented leukocyte αGAL activity of \< 4 nmol/hr/mg
- Patient or patient's parent/guardian had to provide written informed consent prior to any study-related procedures being performed
- Patients had to be male and ≥ 16 years of age
Exclusion
- There is evidence of renal insufficiency, as defined by serum creatinine greater than or equal to 2.2 mg/dL (194.7 μmol/L) AND/OR has an estimated glomerular filtration rate (GFR) of \<80 mL/min (using the equation derived from the Modification of Diet in Renal Disease Study (MDRD))
- Has undergone kidney transplantation or is currently on dialysis
- Has a clinically significant organic disease or an unstable condition (with the exception of symptoms relating to Fabry disease) that in the opinion of the Investigator would preclude participation in the trial
- Has participated in a study employing an investigational drug within 30 days of the start of this trial
- Patients who received prior treatment with enzyme replacement therapy for Fabry disease
- Patient was unable to comply with the requirements of the protocol
Key Trial Info
Start Date :
June 1 2003
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2007
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT00196716
Start Date
June 1 2003
End Date
March 1 2007
Last Update
April 3 2015
Active Locations (4)
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1
II. interní klinika 1. LF UK
Prague, Czechia, 128 02
2
Tartu University Clinics, Department of Internal Medicine
Tartu, Estonia, 51014
3
Klinika Chorob Metabolicznych, Instytut "Pomnik-Centrum Zdrowia Dziecka"
Warsaw, Poland, 04-736
4
Detská fakultná nemocnica Kramáre I. Interná klinika
Bratislava, Slovakia, 833 40