Duchenne Muscular Dystrophy Trial Case Study

Duchenne Muscular Dystrophy (DMD) arises from a genetic mutation, impacting around 1 in every 3,500 - 5,000 male births globally. This condition progressively weakens and damages the body's muscles, resulting in fatality.

In a two-phase pediatric study, patients aged 7 to 13, as well as young adults, were first given an initial treatment involving double-blind IMP administration before moving into an open-label phase. Once that was completed, the patients were able to move into a long-term extension study where they remained on treatment for up to five years.

The overall aim of this study was to evaluate the impact of a new IMP to treat the symptoms of DMD. While recruiting trial patients had been smoother due to site teams recognizing MRN's Home Trial Support advantages for this vulnerable patient group across trials, on-site, various tests assess muscle function in patients. Weekly home visits by MRN's specialists included IMP infusion, vital signs checks, and medication review. COVID-19 led to extra visits in the schedule. Some safety blood draws, typically done on-site, were added as Home Trial Support Visits to protect patients from travel-related virus exposure.

Access the full case study below to learn of the success MRN had in establishing sites and enrolling new patients for the initial study phases across Belgium, France, Israel, Italy, Spain, Sweden, and the United Kingdom.