What do FSHD Muscular Dystrophy Patients in Europe want from Clinical Trials?

November 29, 2021 By Bine Haase

FSHD Europe has commissioned this piece of work which will discover, define and describe what matters to more than 80,000 people living with FSHD across Europe in designing and delivering clinical trials.  We want to encourage people with FSHD to participate in and benefit from clinical trials and this will ensure that trials are designed that encourage participation and measure what is important to patients.  This means

  • Designing the trial to make it easy for people to take part – understanding what motivates or discourages people in clinical trials
  • Measuring activities that are meaningful to people with FSHD and which will improve their quality of life
  • Making it relevant for people at different stages of the condition

This work will be carried out by the John Walton Research Centre at the University of Newcastle in the UK and will include

  • a literature survey of any other relevant work
  • an understanding of the requirements of Medicine Regulators across Europe and how trials should be designed for ease of submission to regulators
  • an online survey aimed at FSHD patients across Europe, accessible in six different European languages
  • reports to the research and patient communities showing what matters in clinical trials for people with FSHD.

FSHD Europe acknowledges the generous sponsorship of Fulcrum, Facio-Therapies and Roche who have made this project possible.

“I’m really excited that this project is going ahead, as it will help to design clinical trials that get results that matter to people living with FSHD.”
(Sheila Hawkins, President of FSHD Europe)