Alpha-Galactosidase A Replacement Therapy for Fabry Disease
Status:
Completed
Trial end date:
2003-11-01
Target enrollment:
Participant gender:
Summary
This study will determine the safety of the drug Replagal or treating patients with Fabry
disease, an inherited metabolic disorder. In this disease, an enzyme called
Alpha-galactosidase A, which normally breaks down a fatty substance called
globotriaosylceramide (Gb3), is missing or does not function properly. The resulting
accumulation of Gb3 causes problems with the kidneys, heart, nerves, and blood vessels.
Replagal is a genetically engineered form of Alpha-galactosidase A. Previous studies have
shown that patients with Fabry disease who had not progressed to end-stage kidney failure
tolerated Replagal replacement therapy well. This study will examine the effects of the drug
in patients with kidney problems associated with Fabry disease.
Patients with Fabry disease who are on kidney dialysis, or have had a kidney transplant, may
be eligible for this study.
During this 6 to 12-month study, participants will receive a 40-minute intravenous (IV)
infusion of Replagal every other week, with close monitoring during and after the infusions.
Before the first infusion, patients will be evaluated with a medical history, physical and
neurological examinations, electrocardiogram (ECG), routine blood and urine tests, kidney
test, and measurements of height, weight, and vital signs (blood pressure, pulse, breathing
rate, temperature). In addition, they will have pharmacokinetic studies immediately before
and following the first infusion of Replagal. For these studies, blood samples of less than a
teaspoon each will be drawn to measure the level of Replagal enzyme activity. The samples
will be collected at the following time points: immediately before the infusion; 20 minutes
into the infusion; at the end of the infusion; after the infusion at 50, 60, and 90 minutes,
and 2, 3, 4, and 8 hours.
Safety evaluations will be done once a week for the first month and then once a month for the
rest of the study period. These evaluations include a physical examination, measurement of
vital signs, electrocardiogram, routine blood and urine tests, and kidney testing.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)