Casmir - Remote Video Assessment, a Pilot Study

Casimir was founded in 2016 by Christine McSherry and Mindy Leffler, both campaigners for and champions of patients with Duchenne Muscular Dystrophy. Casimir develops novel outcome measures for decentralized and hybrid trials by engaging patient communities as partners in research. Every project starts from the patient perspective, taking new approaches to understanding and documenting disease progression and treatment benefit, including video assessment and other innovative tools.

By developing relationships with patients, our Study Coordinators can better support implementation, thus enhancing the participant experience and increasing enrolment and retention. Casimir engages stakeholders including KOLs and regulators to develop new methods of capturing and quantifying patient outcome changes.

The Primary Mitochondrial Myopathy Video Assessment (PMMVA) is intended to measure compensatory movements exhibited by PMM patients that occur as a result of muscle weakness and fatigue. The measure focuses on movements used for everyday activities and can be administered at home, making it a highly versatile and inclusive measure.

Our purpose is to fully develop the PMMVA as a rigorous outcome measure that is sufficiently sensitive and clinically meaningful to detect changes during treatment trials. Casimir has initiated an outcome measure development program in which the PMMVA is developed in collaboration with PMM experts, patients, and caregivers, then undergoes validation and reliability testing using well-established scientific methods.

For more information, please feel free to contact research@polgfoundation.org