When an emerging U.S.-based pharmaceutical sponsor initiated its Phase I clinical trial in 2007 for a novel gene therapy for subjects with a rare inherited form of vision loss, it faced many obstacles. Gene therapy was less than 20 years old, and several years earlier the unfortunate death of a clinical trial subject had spiked concerns about the treatment. Nevertheless, the sponsor pushed forward on its ground-breaking research.

While preparing an interim data report for the U.S. Food and Drug Administration (FDA) in 2010, the original CRO fell short, and the sponsor critically needed a replacement able to hit the ground running. The sponsor knew Westat’s work having previously contracted with us for statistical support and asked us to step in. We took on the project management, site monitoring, pharmacovigilance, data management, and SAS programming support for the Phase I study already in progress, as well as the planned Phase II mobility validation and natural history studies, and the Phase III pivotal trial. 

Stepping in With an Immediate and Assured Start

Our agile and experienced team promptly took action by meeting with the site staff to understand the complex processes for ophthalmologic testing and data reporting. We uncovered numerous data discrepancies in the original interim data report and implemented processes to standardize the testing procedures. We also trained the site staff on best practices for source documentation. This resulted in a solid foundation for the integrity of the data, ensuring our sponsor could count on an optimally designed and statistically sound study.  

Providing Trusted, Ongoing Project Support

One of the sponsor’s requirements was a dedicated project director who would oversee all of the studies and serve as an integral part of the team. Westat CLS delivered with an experienced project manager and support team to oversee the projects for the life of the program. In fact, our project management was so trusted that when members of the sponsor organization founded their own company, they continued to rely on us to support their number-one priority program.  

Securing FDA Approval

In 2017, the FDA advisory panel unanimously recommended the approval of the sponsor’s gene therapy product. Westat was instrumental in preparing for the advisory panel meeting; we developed tables, graphs, slides and proposed answers to a multitude of questions that could be posed by the panel. Later that year, the FDA approved the novel gene therapy to treat children and adult patients with the inherited condition.

Westat CLS was an assured, experienced, and reliable partner to our sponsor from the early and pivotal phases through the Biologics License Application and Advisory Committee Meeting with the FDA.