Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad of challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs.
Premier Research recently faced these challenges firsthand while operationalizing a transition Phase 1/2 trial involving localized administration of gene therapy using specialized equipment. The study comprised two parts, the second of which included a sham procedure:
- Part A: A dose-escalation study consisting of three cohorts and dose levels, with safety reviews in between each dose
- Part B: Once an optimal dose is found, randomize study participants to either gene therapy or sham procedure with subsequent follow-up