Prior to the COVID-19 pandemic, much research was devoted to the study of gene therapy and, in particular, to adeno-associated virus vector gene therapy. The research was carried out in oncology, hemophilia, and other serious and/or orphan diseases, however, approved FDA did not take place. Gene therapy appears to eventually become a reality.
Here you can find publications with attachments:
- 🔥Clinical Study Protocol;
- 🔥🔥 Statistical Analysis Plan;
- 🔥🔥🔥 TLF layouts for SAP;
- 🔥🔥🔥🔥 Full package of documentation for DMC.
Studying a design based on a published example, that eventually was done by the FDA with received a Priority Review, Orphan and Breakthrough Therapy designations is really helpful in practice.