Performing a Rare disease-Oriented Master Observational Trial to decipher complexity and optimize trial readiness

PROMOT will develop an innovative precision medicine approach integrating the ELSI considerations to answer the complex challenges regarding prognosis and clinical trial readiness in rare diseases: A Learning and knowledge mobilization Platform (LEAP FORWARD) based on the adapted Master Observational Trial (MOT).

This will be done through 1) developing a MOT approach using oculopharyngeal muscular dystrophy as a flagship disease; 2) conducting a pilot MOT among four neuromuscular disorders with strong commonalities;3) documenting the feasibility of conducting a MOT approach in these vulnerable populations and across different legislations, ethical and social interfaces; 4) developing the LEAP FORWARD platform and 5) providing research-based “engagement, ethical, legal and social issues” (EELSI) guidance to support PROMOT and to propose EELSI frameworks needed to inform and buttress future research in “next-generation deep medicine”.Phase 1 f on the signs and symptoms specific and common to the four targeted neuromuscular diseases and inclusion in the MOT protocol and LEAP FORWARD platform.

Phase 2 tests MOT to document the feasibility for rare diseases. All PROMOT data will be used to develop the LEAP FORWARD platform.

PROMOT will be conducted in collaboration with patient organizations, patient partners, clinicians, researchers, and ethical and legal experts. It will respond to several unmet needs found in most rare diseases regarding prognosis and clinical trial readiness.