A natural history study to understand the expected progression of disease in patients with confirmed mutations in the VCP gene over 1 year.
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
100 meter timed test
Timeframe: 12 months
North Star assessment for limb girdle type dystrophies (NSAD)
Timeframe: 12 months
Timed Up and Go
Timeframe: 12 months
4 stair climb
Timeframe: 12 months
Performance of Upper Limb (PUL) 2.0
Timeframe: 12 months
9 hole peg test
Timeframe: 12 months
Forced Vital Capacity
Timeframe: 12 months
PROMIS Global Health scale
Timeframe: 12 months
Neuro-QOL Upper Extremity Function
Timeframe: 12 months
Neuro-QOL Lower Extremity Function (Mobility)
Timeframe: 12 months
Rasch Overall ALS Disability Scale (ROADS)
Timeframe: 12 months
Neuro-QOL Cognition Function
Timeframe: 12 months.
EAT-10
Timeframe: 12 months
Communicative Participation Item Bank (CPIB)
Timeframe: 12 months
Speech Handicap Index
Timeframe: 12 months