Phase I study to assess safety and tolerability of investigative medication in pediatric patients with Sanfilippo syndrome (MPS IIIA)

A specialty healthcare company developing treatments for rare diseases sought our support for an early-stage pediatric trial program testing an investigational medicine in Sanfilippo syndrome. We were commissioned to run a nine-month online campaign to raise the profile of the program and to find and refer up to four of the nine patients needed for the Phase I study in the United States; to a study site that initially had no patients of their own.

We found, prescreened and referred three eligible children and their parents within three weeks of study start-up.