Unleashing Flexibility in a Rare Disease Gene Therapy Clinical Trial

In the world of biotechnology, where groundbreaking discoveries have the potential to change lives, adaptability and flexibility are paramount. Clinical operations can be a complex web of challenges, especially when dealing with rare diseases and gene therapies. Veristat's recent collaboration with a biotech organization focused on gene therapies for metabolic diseases provides a compelling case study on how flexibility can drive progress even in the most demanding clinical trials.

Solving the Enrollment Puzzle

One of the initial challenges faced by the biotech company was site enrollment. The sponsor had identified a list of potential sites, but none of the sites proved viable. Veristat's Medical Affairs team stepped in and engaged in peer-to-peer conversations with clinicians experienced in treating the disease. Leveraging their extensive network of principal investigators and sites and working collaboratively with the sponsor, nearly 10 sites were recruited globally. A comprehensive site feasibility questionnaire was authored to confirm the suitability of each site, addressing critical aspects such as gene therapy experience, patient scheduling, blood draws, and sample handling.

Timely Administration of Therapy

Another hurdle lay in the time-sensitive administration process required for eligible patients. The therapy's manufacturing dates were fixed, leaving no room for delays. Veristat meticulously planned subject screening and baseline assessments, working backward from the manufacturing date. Detailed schedules were provided to the sites, ensuring all activities adhered to the timeline.

Meeting Evolving Reporting Needs

As if these challenges weren't enough, the sponsor's management team had several new members join during the duration of the study, who introduced new reporting requirements. Veristat swiftly responded by creating a sample specimen tracker, customized ADI and risk logs, and updated monthly metric reports that tracked a range of measures, ensuring transparency and accountability.

The Impact of Flexibility

The results were impressive. Veristat's flexibility and collaborative partnership fulfilled the changing study needs, leading to high sponsor satisfaction. Thorough site evaluation ensured successful site recruitment and enrollment, while the adaptability of Veristat's approach accommodated a study with challenging logistics and evolving reporting needs.

Though the study was halted due to efficacy results, Veristat continues to support the sponsor in managing post-transplantation patient requirements through a long-term follow-up study.

In the dynamic world of biotech, Veristat's case study serves as a testament to the power of flexibility, adaptability, and collaborative partnerships. Clinical trials, especially those involving rare diseases and cutting-edge therapies, demand innovative approaches. Veristat stands as a beacon of innovation, driving progress in the pursuit of better treatments and cures.

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