Case Study

Site Selection, Manufacturing & Long-Term Follow-Up: Looming Challenges For A Rescue Phase 1/2 Gene Therapy Trial

By Naomi Kautz

DNA Gene Strand


In the following article we introduce a case study as a framework for exploring critical study design considerations of gene therapy trials and offering strategies for addressing those hurdles.


This case study is based on Premier Research’s work operationalizing a rescue Phase 1/2 gene therapy trial involving localized administration of gene therapy using specialized equipment. The study comprised two parts, the second of which includes a sham procedure (see Figure 1):

  • 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

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