FSHD European Trial Network

FSHD Europe establishes European Trial Network

FSHD Europe is proud to announce the establishment of the project “FSHD European Trial Network”. We have initiated and coordinated this project with Nicol Voermans, MD, PhD Neurologist as chair and Pascal Laforet, MD, PhD Neurologist as vice chair. George Padberg, MD, PhD and Baziel van Engelen, MD, PhD are the advisors.

FSHD Europe has taken this initiative to support collaboration to reach trial readiness, building on current research on FSHD in Europe.  Although there have been a number of research workshops held in recent years, these have not yet led to a solid trial network in Europe, which is why FSHD Europe seeks to bring this together.

FSHD is on the threshold of clinical therapeutic trials with interest in FSHD from several pharmaceutical industries and a few trials currently underway in the USA and Europe.  However, the guidelines for clinical trials, pharma regulation, participation and healthcare provisions in the different European countries vary in subtle ways and would benefit from an overall strategy designed for the European situation.

The patient organisations and the clinical and research networks in the USA are well established and collaborate successfully; the organisations in Europe lag behind, especially at the clinical level. In order to offer the FSHD patient community in Europe the best position in discussions with clinical and basic science researchers and with pharma, FSHD Europe is organising this meeting with the following aims:

  • Establish the foundation of a European FSHD Trial Network
  • Harmonise criteria for clinical and genetic diagnosis, for registries and outcome measures
  • Create exchange of clinical experience and genetic reference material
  • Bring Europe on a par with the USA in trial-readiness for FSHD

In addition, if treatments prove to have a positive effect, the European FSHD Trial Network will have to act as a partner to discuss with health authorities. This adds the following aims:

  • Engage Pharma and EMA for a Europe-wide collaboration
  • Harmonise treatment and care for all European FSHD patients

This project is led by a core group of experts from different European countries and the collaboration of new clinicians and researchers in FSHD is welcome. It is our intention that the new network remains united and promotes future European collaborations. It is our goal that the new network becomes well known, expands and works hand in hand with other existing work groups (CTRN) and institutions (TREAT-NMD).

The online workshop will be organised by invitation and has a limited number of participants from the presently active research community, in addition to clinicians and clinical researchers from other groups and patient representation.  

PART 1: 23rd April 2021 GENERAL ASPECTS OF TRIAL READINESS AND IMAGING

  • SESSION 1:  LESSONS FROM OTHER TRIALS AND TRIAL NETWORKS
    Session Leader(s): Alexandre Mejat and Isabelle Desguerre 
  • SESSION 2:  GENETICS, PARTICIPANTS AND REGISTRIES
    Session Leader(s): Teresinha Evangelista and Pascal Laforet
  • SESSION 3:  MUSCLE IMAGING
    Session Leader(s): Shahram Attarian and Giorgio Tasca

PART 2: 7th May 2021 SPECIFIC ASPECTS OF TRIAL READINESS

  • SESSION 4:  CLINICAL OUTCOME MEASURES and BIOMARKERS
    Session Leader(s): Adolfo López de Munaín and Benedikt Schoser
  • SESSION 5:  CURRENT AND EXPECTED TRIALS, AND WHAT DO WE NEED TO CONSIDER?
    Session Leader(s): Baziel van Engelen and John Vissing
  • SESSION 6: FSHD EUROPEAN TRIAL NETWORK
    Session Leader(s): Enrico Bugiardini and George Padberg

The results of the workshop will be a position paper that will be presented at the International Research Conference on FSHD (organised by the FSHD Society) on June 24th and 25th, 2021.

In addition, the network aims to reach out to countries that are not yet (extensively) involved in FSHD research. We would like to connect to doctors and patient representatives from all European countries to be able to disseminate knowledge, to have estimates of patients in the different countries and eventually to make future trials and therapies accessible to all European patients. Please contact us if you are interested.

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