Managing a Pediatric Trial in an Orphan Indication

Due to our extensive pediatric experience, a biopharmaceutical company focused on the discovery and development of novel products to address unmet medical needs contracted with Premier Research to manage their 30-site, 212-patient double-blind placebo-controlled trial in pediatric patients with Eosinophilic Esophagitis (EE), an often undiagnosed illness. We provided full-service management of the study including clinical trial management, data management utilizing Oracle Clinical RDC, IVRS, medical monitoring and safety management, and quality assurance services.

Study challenges included obtaining IRB approval from local IRBs, training naïve investigators and an independent qualified staff member required to mix the drug, difficult patient enrollment due to limited population, invasive protocol procedures including esophageal biopsies and monthly IV infusions, and protocol amendments based on lessons learned in this new indication.

Our experienced pediatric team conducted a thorough investigator meeting, assisted sites with their IRB submissions, performed intensive site initiation visits, and provided continual training and support while the sponsor reached out to the EE community for enrollment assistance. By working very closely with both the sponsor and the sites in a combined effort through all phases of the study, we were able to successfully mitigate the study challenges and exceed the sponsor’s expectations in this difficult indication.

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