Case Study: Duchenne Muscular Dystrophy

A sponsor conducting a Phase III study in India needed to evaluate the effectiveness of their drug on the ambulation of pediatric patients with Duchenne Muscular Dystrophy (DMD). At the time, the study was already 72 weeks in and was slated to carry out an additional 72-week OLE. However, for it to be successful, the sponsor would need to randomize the participants and ensure that they would be able to complete all visits over the extended duration of the study.

One of the more challenging aspects of this task was the remote locations of the patients in relation to the site facilities. In addition to these logistical challenges, the advanced disease state of the patients and the related need for two caregivers to accompany the patient at each visit also proved difficult.

Access this case study to discover how the sponsor was able to provide logistics services and expense management services to 45 patients and families located in remote areas of India participating in a Phase III study and its Open-Label Extension.