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A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00196716
First received: September 12, 2005
Last updated: August 11, 2009
Last verified: April 2007

September 12, 2005
August 11, 2009
June 2003
April 2006   (final data collection date for primary outcome measure)
Globotriaosylceramide (GL-3) Clearance in Kidney Interstitial Capillary Endothelium [ Time Frame: Throughout study; 96 weeks ] [ Designated as safety issue: No ]
Evaluate 1.0 mg/kg of Fabrazyme followed by a maintenance dose of 0.3 mg/kg of Fabrazyme to clear and maintain clearance of globotriaosylceramide from the vascular endothelium of the kidney in patients with Fabry.
Complete list of historical versions of study NCT00196716 on ClinicalTrials.gov Archive Site
  • Skin Globotriaosylceramide (GL-3) Clearance From Superficial Skin Capillary Endothelium [ Time Frame: Throughout study ; 96 weeks ] [ Designated as safety issue: No ]
  • Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Throughout study; 96 weeks ] [ Designated as safety issue: No ]
  • Plasma Globotriaosylceramide (GL-3) [ Time Frame: Throughout study; 96 weeks ] [ Designated as safety issue: No ]
  • Urine Globotriaosylceramide (GL-3) [ Time Frame: Throughout study, 96 weeks ] [ Designated as safety issue: No ]
Evaluate the efficacy of Fabrazyme to clear and maintain clearance of GL-3 from the skin and from other cell types in the kidney, to decrease and maintain plasma clearance of GL 3, and to evaluate clinical stabilization of the disease and pain.
Not Provided
Not Provided
 
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
A Multicenter, Open-label Study of Low Dose Maintenance Treatment of Fabrazyme (Recombinant Human Alpha-Galactosidase A (R-h Alpha-GAL)) Replacement Therapy in Patients With Fabry Disease

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 fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because alpha-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This trial is designed to evaluate the efficacy of a lower dose of Fabrazyme in patients who initially received 1.0 mg/kg every 2 weeks of Fabrazyme by investigating if the achieved clearance of glycosphingolipid deposits in the vascular endothelium of the kidney can be maintained at a lower dose.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Biological: Fabrazyme (agalsidase beta)
1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months
Other Name: r-hαGAL
Experimental: Fabrazyme
Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months.
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
21
March 2007
April 2006   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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 Criteria:

  • 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
Male
16 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Czech Republic,   Estonia,   Poland,   Slovakia
 
NCT00196716
AGAL-017-01
Not Provided
Medical Monitor, Genzyme Corporation
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
April 2007

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