Extension study of Migalastat HCl in subjects with Fabry disease

• Research type

  Research Study

• Full title

  An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease

• IRAS ID

  90534

• Contact name

  Derralynn Hughes

• Sponsor organisation

  GlaxoSmithKline Research & Development Limited

• Eudract number

  2011-004800-40

• ISRCTN Number

  Unknown

• Research summary

  This is a phase III open-label, long-term extension study funded by GlaxoSmithKline (GSK). The purpose of the study is to investigate the long-term safety and effectiveness of migalastat hydrochloride (HCl) (the experimental drug being tested) in subjects with fabry disease. Subjects enrolled in this study will have completed treatment in a previous study with migalastat HCl and so this study is intended to provide continued treatment with migalastat HCl for these subjects. It is expected that at least 100 subjects will be eligible to enter this study. Approximately 10 participants will be recruited in the UK. The duration of participation could be up to 4 years. The study will consist of a baseline visit which will be performed on the same day as the final visit of the previous study, followed by clinic visits every 6 months for each year of the study. The planned duration of the treatment will vary among subjects and will continue until the date of marketing approval of migalastat HCl or study closure by GSK. All subjects will receive an oral dose of 150mg of migalastat HCl every second day. This study involves procedures including:- Physical examination, vital signs, ECG, heart echo, blood tests, urine tests and subject health questionnaires.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  11/LO/1823

• Date of REC Opinion

  25 Jan 2012

• REC opinion

  Further Information Favourable Opinion