Ways to measure clinical effectiveness in the investigation of medicinal products for the treatment of DMD/BMD
Session 1 – What is clinically meaningful and how to measure this?Chaired by Jan Verschuuren |
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| Introduction | Jan Verschuuren | |
| Video showing natural history of the disease demonstrating functions and milestones | ||
| Natural History of DMD/BMD | Craig McDonald | |
| Summary of the decision framework for inclusion of clinical outcome measures in clinical trials and studies across the natural history of DMD | ||
| What patients see as being clinically meaningful | Pat Furlong, Elizabeth Vroom, and Phillippa Farrant | |
| What are the major milestones of the disease and how it is important to measure and quantify these in the context of a clinical study? | ||
| Outcome measures in young children | Eugenio Mercuri | |
| Selection criteria for outcome measures | ||
| Outcome measures in ambulatory patients | Craig McDonald | |
| Outcome measures in non-ambulant patients | Anna Mayhew | |
| Session Discussion | ||
| Natural history and major milestones of the disease Selecting outcome measures for clinical trials | ||
Session 2 – Methods of efficacy measurement – strength versus functionChaired by Francesco Muntoni |
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| Introduction |
Francesco Muntoni |
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| Natural history studies showing relationship in the lower extremities | Craig McDonald | |
| Natural history studies showing relationship in the upper extremities | Thomas Voit | |
| Session Discussion and round-up of morning presentations | ||
| Strength and Function as co-primary endpoints – is this the best choice in trial design? | ||
Session 3 – Animal Models and Biochemical Outcome MeasuresChaired by Annemieke Aartsma-Rus |
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| How reliable are Animal Models? | Annamaria de Luca and Nic Wells | |
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Prosensa and Annemieke Aartsma-Rus | |
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Annemieke Aartsma-Rus | |
Session 4 – Strategy & Design of Clinical Studies
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Accelerated design |
Clinical perspective Thomas Voit and Nathalie Goemans | |
| Video showing quality of life in non-ambulant boys and men | Patient perspective Elizabeth Vroom and Filippo Buccella | |
Final comments and round-up by the Session Chairs |
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| Including on-going work and other issues to be addressed | ||
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