Our Phase I and rare tumors team comprises key specialized backgrounds, including pediatric oncologists, clinical pharmacologists, and First in Human specialists, and strategic statisticians, all sharing broad oncology experience and a passion for scientific innovation.
Most early phase studies concern dose escalation/expansion studies with patient populations of either a broad range of solid tumors or pre-specified targeted populations. Many of these studies also include dose escalation/expansion of an investigational medicinal product combined with another agent such as an immune checkpoint inhibitor. Often, a complex innovative trial design (CID) is more practical for ultra-rare disease than randomized controlled trials. CIDs can answer multiple questions about one or more compounds in one or more conditions or patient subgroups. Prespecified, “adaptive” modifications of the trial protocol can allow changes during the study based on interim data analyses. These adaptations can include sample size adjustments, dropping arms because of futility or safety findings, or enriching the target population.
Our team has extensive experience in both the standard rule-based escalation designs as well as the different Bayesian model–based designs, relying on the support of our top-notch biostatistics group. We are leaders in complex adaptive trial designs such as two- or three-stage, basket, umbrella, and platform studies, and have developed best practices in the evolving use of flexible study design in oncology research.