Preventive trial design in genetic prion disease is hampered by insufficient at-risk population

Takeaway

  • There is not a large enough population at risk for the development of genetic prion disease to accurately power efficacy in randomized clinical trials at current time.

Why this matters ?

  • Current clinical trials for genetic prion disease randomize symptomatic patients; however, once randomized, patients are already in advanced dementia, and drugs may not show any clinical benefit.

  • Redesigning the approach to clinical trials for genetic prion disease may be essential to providing access to medications.