Tuesday 3rd July

9.00 – Feedback from TACT review session
Annemieke Aartsma-Rus

Objective: align on strengths and weaknesses and outstanding questions identified by the groups

Session 2 – Clinical trials

9.30 – Introduction to clinical trials
Michela Guglieri

Objective: introduce how and why clinical trials are conducted, the objective of different trial phases, trial design, primary endpoints, secondary endpoints, clinical significance, ethical concerns and informed consent

10.30 – Tea/Coffee

11.00 – Ethical discussion role play
moderated by Silvere van der Maarel, Becca Leary and Teresinha Evangelista

Participants will be provided with a scenario for a clinical trial plan for a drug to be tested in children. Different roles will be given to different participants.
Objective: gain insight in ethical discussions related to clinical trials and the perspectives of different stakeholders

12.30 – Lunch

13.30 – Outcome measures
Anna Mayhew

Objective: overview of the different types of functional outcome measures used in clinical trials for neuromuscular, their opportunities and limitations, need for standardization and training, TREAT-NMD efforts in their area, natural history studies and their usefulness in trial design

14.30 – Showcase on outcome measure development and labelling and marketing
Annemieke Aartsma-Rus

Objective: to illustrate the consequences of using outcome measures limited to certain disease stages with regards to extrapolation and limited indications

15.00 – Coffee/tea

15.30 – Showcase on outcome measure development
Anna Mayhew

Objective: to outline the steps and stakeholders involved in developing and validating functional outcome measures that assess what patient find important using upper limb function outcome measure development for DMD and SMA as showcases

16.30 – TREAT-NMD tools to facilitate clinical trials
Michela Guglieri and Becca Leary

Objective: to gain insight in available tools and services for planning and conducting clinical trials

17.30 – End of day 2

Faculty Dinner