Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
Status:
Completed
Trial end date:
2005-11-01
Target enrollment:
Participant gender:
Summary
The main goal of this study is to assess the pharmacodynamic effects of different or more
frequent doses of Replagal compared to the standard dosing regimen. Replagal is a genetically
engineered form of alpha-Galactosidase A, an enzyme that normally breaks down a fatty
substance called globotriaosylceramide (Gb(3)). In patients with Fabry disease, GB(3) does
not function properly and therefore builds up causing problems with the kidneys, heart,
nerves, and blood vessels.
Male patients 18 years of age or older with Fabry disease who are not on dialysis and have
not received a kidney transplant may be eligible for this study.
Participants are randomly assigned to receive one of the following five regimens of Replagal
infusions, given through a vein over 20 to 80 minutes:
0.1 mg/kg body weight every week
0.2 mg/kg body weight every week
0.2 mg/kg body weight every other week
0.4 mg/kg body weight every week
0.4 mg/kg body wieght every other week
In the US, the infusions are given at the NIH Clinical Center. Vital signs are measured
before, immediately after, and 1 hour after each infusion.
Baseline evaluations are done on an inpatient or outpatient basis. Baseline tests include a
check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure);
physical examination; laboratory tests; and review of treatment side effects. Evaluations are
also done at every infusion visit, and 1 week and 1 month after the last infusion.
Safety evaluations are done periodically and include vital sign measurements, physical
examination, blood and urine tests, review of drug side effects, electrocardiogram (ECG),
Holder monitor (2 hour ECG), and QSART (NIH only). The QSART (quantitative sudomotor axon
reflex test) measures the amount of sweat in a particular area of skin, mostly the forearm.
For this test, a cup partly filled with a liquid is strapped on the arm. A weak electric
current is turned on, stimulating the sweat glands, and the amount of sweat produced is
measured. There is a tingling sensation when the current is turned on.
Patients who complete the study will be offered the opportunity of receiving Replagal for 6
months in an extension study.
Phase:
Phase 2
Details
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)