Creating Solutions to Ensure Endpoint Protection in Challenging Rare Disease Trial

The collection of high-quality endpoint data was paramount to the success of this Phase II program. Because this was the first clinical trial ever done for this disease, it was necessary to develop non- disease-specific clinical assessments, in collaboration with Key Opinion Leaders (KOLs), for use as endpoints. The newness of these tools presented challenges to site selection and training, as well as risks to uniformity in patient recruitment and consistency in data collection.