Phase III /IV study of Replagal in adult patients with Fabry Disease
Research type
Research Study
Full title
A Multi - center, Open-Label, Randomised Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients with Fabry Disease
IRAS ID
38930
Contact name
Stephen Waldek
Sponsor organisation
Shire Human Genetic Therapies Inc
Eudract number
2007-005543-22
ISRCTN Number
N/A
Clinicaltrials.gov Identifier
Research summary
The goal of this research study is to assess three different doses and dosage scheduling of enzyme replacement therapy with Replagal in patients with Fabry disease. Fabry disease is a rare, inherited disorder characterised by a build up of a fatty substance called globotriaosylceramide in the body due to an enzyme that is not working properly. Under treatment with Replagal this enzyme is replaced. Replagal is licensed for marketing in the UK at a dose of 0.2 mg/kg body weight, infused over 40 minutes every two weeks. Replagal has been shown to improve heart function at 0.2 mg/kg every two weeks in previous clinical trials. In this research study, the approved dose as well as two higher doses (0.2mg per kg weekly and 0.4 mg per kg weekly) will be administered to patients with Fabry disease over a period of 52 weeks. In Fabry patients glycolipids such as globotriaosylceramide build up in different heart cells leading to heart related effects that worsen with age and may lead to an increased risk of heart disease. Accumulation of glycolipids in the heart can lead to increasing thickness of the heart that can eventually lead to heart failure.There is also a significant increase in abnormalities of heart rhythm. The primary aim of the trial is to see if higher doses and different dosing schedules further improve heart structure and function in patients with hypertrophic cardiomyopathy (thickening of the heart muscle). This study is a multinational clinical trial. All patients participating in the study will receive the study drug, a placebo will not be used.It is planned to recruit a minimum of 43 patients in up to 12 sites in 8-9 countries. Study consists of a 14 days screening period, 52 weeks treatment and 4 weeks follow up, making a total of 58 weeks.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
10/H1008/3
Date of REC Opinion
6 May 2010
REC opinion
Further Information Favourable Opinion