ALS Platform Trial Case Study

How did this first-of-its-kind platform trial tackle the challenges of ALS’s debilitating disease progression?

When a leading ALS research hospital and a nationwide network of clinical research centers wanted to speed the development of an effective treatment for people living with Lou Gehrig’s disease, they designed a platform trial that would test multiple investigative products at the same time–and included in-home visits as a critical element of the study.

During the 24-week double-blind portion of the study, 80 patients with amyotrophic lateral sclerosis (ALS) would be seen for a total of 320 visits. Participants would also have the option of additional visits for up to a year and could participate in an indefinite open-label extension depending on how they were responding to the treatment protocol.

The study team faced multiple challenges including:

  • Limited mobility
  • Progressive neurodegeneration
  • Highly distributed patient pool
  • Rural participants
  • Sporadic, indefinite visits (open-label extension)
  • COVID nursing shortage