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Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

NCT00075244 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2008-03-04

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Replagal

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Study Design

Purpose
TREATMENT

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-01-31
Completion
2005-11-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00075244 on ClinicalTrials.gov