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.
We recommend great publications on ClinicalTrial.gov that hide nothing: https://lnkd.in/ghcBne-e
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.
Source: https://lnkd.in/e66UAMuD.