Help fund our innovative and time sensitive functional measures study

This innovative study will help with future VCP disease clinical trials

Help fund our innovative and time sensitive functional measures study image

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This innovative study will help with future VCP disease clinical trials

COVID-19 has presented an opportunity, with the increased acceptance of telehealth processes, to conduct the physical measurement of patients from the comfort of their homes. Cure VCP Disease has partnered with the Lowes Lab at Nationwide Children's Hospital (NCH) in Columbus, Ohio to pilot and conduct a remote functional measures study. Cure VCP Disease desires to self-fund the study in order to expedite its start and completion.

In September 2020, we piloted conducting a remote functional measures study with a small group of five VCP disease patients. In 2021, we will add fifteen more participants. The cost of the study is $120,000 and funds the NCH researcher's time as well as helping to offset patient's travel costs to Columbus, OH. Some of the study will have to be conducted at NCH to validate that there is no statistical difference in patient performance between the clinic and home.

Quantitatively understanding the progression of VCP disease is vital towards developing future therapeutics and conducting clinical trials. Capturing patient medical records, measuring patients' physical performance and archiving outcomes over time is the preferred method and a prerequisite for pharmaceutical organizations. These activities and measurements are considered a "natural history study." Some members of the VCP disease research community have conducted smaller natural history study activities, but not at the scale that will be required by pharmaceutical organizations and the FDA for clinical acceptance.

Many times these studies are funded by pharmaceutical companies or by the National Institutes of Health (NIH). Because VCP disease is an "ultra" rare disease, finding a well funded and interested pharmaceutical or biotech company could take years. Applying for NIH grants could also take years. Time is not a VCP disease patient's friend and we desire to lead this study.

In parallel, Cure VCP Disease has been selected by a company called AllStripes to facilitate the collection and analysis of patients' medical records. VCP disease patients can consent to allow AllStripes to collect their medical records, which will then be curated into reports and information that can be utilized for advancing treatments.

All of these efforts will drive significant momentum towards the completion of the most comprehensive natural history study of VCP disease ever conducted!! Be a part of history!