Global Pediatric Full-Service Phase I/II Clinical Trial Begins On Heels Of Successful Natural History Study

In the early stages of clinical development, a mid-sized biotechnology company leading a worldwide clinical trial focused on rare diseases realized that it would need an extraordinarily high degree of coordination to be successful. Given the additional challenge of patients having to relocate for their participation in this study, the sponsor is tasked with a greater set of responsibilities than what is typically encountered in a standard clinical program.

As a result, the biotech engaged Veristat to assist with a natural history trial aimed at addressing the exceptionally rare neurological disease affecting pediatric patients with no existing treatment options. Following the execution of the natural history study, the sponsor proceeded with the groundbreaking gene therapy program, which has already administered treatment to its initial patients.

Veristat remains actively involved in overseeing the ongoing natural history study and has initiated the Phase I/II clinical trial, offering comprehensive support encompassing strategic consulting, statistical and programming assistance, site management, data management, program management, and medical affairs services.

Dive into the details of the successful case study and how teams can ensure a seamless trial while optimizing the patient experience.