Key Words: Duchenne Muscular Dystrophy (DMD), Safety Profiles, Rare Disease Patients
Objective:
Describe and discuss Medical Directors (MDs) support and collaboration, clinical trial related considerations, challenges, successes and follow up in MDs day-to-day activities with sites and project teams.
Method:
A Phase III, randomised, multinational trial in ambulatory DMD patients, enrolled approximately 190 patients, conducted in over 40 sites in 9 countries, in USA, Canada, and Europe, with CRO performing services including Medical Management full service.
Results:
Medical Directors input to the site selection, and Principal Investigators follow up was crucial for the trial.
The MDs involvement with the Clinical project team had tremendously supported the recruitment. First Patient In was achieved on schedule. Trial was run during the COVID pandemic, and Home Health nursing was implemented to assure patient safety during the pandemic. Significant positive results were delivered, enrolment was swiftly resumed, and the Last Patient In achieved on time.
Monthly Safety medical meetings were ongoing for medical and safety data listings review. The Serious Adverse Event rate, haematology and chemistry/lipids profiles reported were continuously reviewed by MDs to assure patient safety and to identify potential safety signals. MDs escalated and discussed important safety signals in haematology and chemistry/lipid labs. These signals were reviewed by the Data Monitoring Committee and were reflected in Protocol amendment that added criteria for selected chemistry lab parameters cut offs values. Sites were in close contacts and the follow ups by MDs made an impact.
Conclusion:
Challenges in medical management of safety parameters in a global DMD trial were shared and indicated that Medical Directors input was crucial. Effective relationships between MDs, Clinical project team and the sites are noted as one of the key success factors.