Extension Study of TKT028 Evaluating Safety Outcomes of Replagal
Research type
Research Study
Full title
An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease
IRAS ID
78950
Contact name
Reena Sharma
Sponsor organisation
Shire Human Genetic Therapies Inc
Eudract number
2009-015985-75
ISRCTN Number
N/A
Clinicaltrials.gov Identifier
Research summary
The goal of this research is to assess the long term safety and clinical outcome of treatment with Replagal administered to patients with Fabry disease at a dose of 0.2mg/kg body weight every other week. 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's not working properly. Replagal is an enzyme replacement therapy. Enzyme replacement therapy provides patients with the enzyme they are lacking. Replagal is licensed for marketing in the UK at a dose of 0.2 mg/kg body weight, infused over 40 minutes every 2 weeks. Replagal has been shown to improve heart function at 0.2 mg/kg every 2 weeks in previous clinical trials. In this study a maximum of 43 patients with Fabry disease that completed the Study TKT028 may be enrolled. All patients will receive the approved dose of 0.2 mg/kg body weight by a continuous 40 minute IV infusion every other week. Study consists of 51 weeks treatment and follow up visit at Week 53. In Fabry patients glycolipids such as globotriasylceramide 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 heard failure. There is also significant increase in abnormalities of the heart rhythm.The primary aim of this study is to is to see if there is a change from baseline to Week 53 in the heart structure and function in patients with hypertrophic cardiomyopathy (thickening of the heart muscle). This study is a multinational clinical trial. Week 53 from TKT028 study will serve as the week 1 visit for this study.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
11/NW/0350
Date of REC Opinion
18 Oct 2011
REC opinion
Further Information Favourable Opinion