First clinical trials best practice workshop

As part of its mission to bring effective therapies to patients in a timely & sustainable way, we have started to expand Europe’s current capacity to run clinical trials for potential SMA therapies.

One of the activities undertaken to achieve this goal was the organisation of a workshop to inform neurologists, physiotherapists and research nurses interested in running SMA clinical trials in their countries about clinical trials best practice.


Left, top to bottom: Charlotte Lilien, Hinal Patel and Mariacristina Scoto
Right, top to bottom:
Giovanni Baranello, Eduardo Tizzano, Allyson Gray and Laurent Servais

Renowned European experts in SMA clinical trials were recruited to organise and run this workshop:







The workshop

This first workshop took place on Tuesday 4th February in Evry, France. Eleven Neurologists and physiotherapists from hospitals in Portugal, Russia, Serbia, Switzerland and Ukraine participated.

In order to take part in the workshop, participants were asked to review a clinical trial protocol on a fictitious gene therapy product and answer some questions pertaining to:

  1. Any practical or ethical concerns observed in the protocol
  2. The timeline, manpower & equipment needed and budget to recruit patients
  3. The adequacy of safety and efficacy assessments.

All preparatory work was reviewed by our experts ahead of the workshop.

The workshop programme consisted of four sessions, each including brief presentations and interactive exercises:

  1. Setting up a clinical trial, including feedback on the preparatory work
  2. Patient recruitment
  3. Endpoints, outcome measures and safety;
  4. Audit, monitoring, protocol deviation and patient retention.


The day ended with patient advocates feeding in the patient’s perspective and with members of the pharmaceutical companies with programmes for SMA (AveXis, Biogen, Cytokinetics, Roche and Scholar Rock), who came to find out about the European arm of this initiative.

This workshop was extremely well reviewed by all stakeholders. We are now working on our next endeavours.

Participants from Portugal, Russia, Serbia, Switzerland and the Ukraine, together with the trainers and SMA Europe’s Clinical Trials Project Team.