Premier Research coordinated a global pediatric gene therapy trial for a rare genetic disorder, highlighting the complexity and high-risk nature of such studies. With only 16.7% of WHO-registered trials being pediatric, this study required specific strategies to meet new regulatory requirements and manage intricate processes. Challenges included enhancing participant experience, tracking patient data across sites, and navigating GMO use as an investigational product. Premier Research developed informed consent materials, high-touch travel support, and a robust data management plan. The trial successfully enrolled 10 patients, performed surgeries, and maintained patient well-being.

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