At a glance
ClinicalIndex Comparison Record- ✓Clinical manifestations of Fabry disease documented in patient's history
- ✓Plasma α-galactosidase A activity <1.5 nmol/hr/mL OR leukocyte α-galactosidase A activity <4 nmol/hr/mg
- ✓Male gender and age ≥16 years
- ✓Written informed consent from patient or parent/guardian prior to study procedures
- ✕Renal insufficiency defined as serum creatinine ≥2.2 mg/dL (194.7 μmol/L) AND/OR estimated GFR <80 mL/min using MDRD equation
- ✕History of kidney transplantation or current dialysis treatment
- ✕Clinically significant organic disease or unstable condition (excluding Fabry-related symptoms) that would preclude trial participation in Investigator's opinion
- ✕Prior enzyme replacement therapy for Fabry disease
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 2 clinical trial evaluating Fabrazyme (agalsidase beta) for Fabry Disease. Completed, enrolled 21 participants across 4 sites in 4 countries.
Detailed 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 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.
Study Details
Timeline
Interventions
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