Delivering The Global Expertise Required To Coordinate A Pediatric Gene Therapy Trial

A European biotech company approached Premier Research with a pediatric trial involving a rare, incurable genetic disorder. With only 16.7 percent of World Health Organization-registered trials classified as pediatric, children are significantly underrepresented in clinical studies. Recent regulations such as the Research to Accelerate Cures and Equity (RACE) Act are designed to promote research and development of therapies for children.

In addition to new regulatory considerations, this trial also called for an extremely challenging intracerebral administration. To succeed, Premier needed to develop a clear, compliant strategy for obtaining informed consent and implementing an innovative data management plan to track patients between surgical and clinical sites.