Setting up an effective phase one trial for a novel investigational oncology product is rife with challenges. Every aspect from site identification to estimation and coordination of enrollment and safety review is interconnected. Nuance is important.
What factors can affect, or even derail, the timeline? Who are the key influencers and experienced sites? Does a potential site have the requisite expertise in multicenter dose escalation studies and the ability to collect the appropriate patient pharmacokinetic and pharmacodynamic biomarker samples? What options are available for a suitable dose-escalation design strategy: Rules-based design (e.g. 3+3 or Rolling-6) or Model-based design (e.g. CRM, etc.)?
Topics in this presentation include:
- Medical informatics: Benchmarking similar trials via proprietary and public domain sources to project accurate timelines, anticipate enrollment rates, and identify potential sites
- Site selection: Vetting identified sites based on their expertise in conducting multicenter phase 1 oncology dose-finding studies, specific relevant capabilities, and local regulations
- Study design: Considerations for dose-escalation design
Peter Larson, MD, Vice President, Medical Affairs, Premier Research
Abie Ekangaki, PhD, Vice President, Statistical Consulting, Premier Research
Meghan Shea, Manager and Solutions Lead, Premier Research
Federica Vinciati, PhD, Senior Regulatory Start-Up Manager, Premier Research