For early-phase clinical trials, especially first-in-human trials, the primary objective should be an evaluation of safety. Safety evaluation includes an assessment of the nature and frequency of potential adverse reactions and dose estimation. For gene therapy products, these early-phase trials often assess not only safety of specific dose regimens and routes of administration but may also include secondary objectives regarding feasibility of administration and pharmacologic activity. You should consider the design of early-phase studies in the context of the objectives of the overall development program – you can, therefore, include design elements to foster further product development. Some Phase 1 studies include selected features of Phase 2 study design to gather preliminary evidence of effectiveness.
Secondary trial objectives for early phase studies include:
- Safety Assessment
- Pharmacokinetics and Pharmacodynamics
- Dose Finding and Optimization
- Preliminary Efficacy Assessment
- Feasibility and Proof of Concept
- Biomarker Identification
- Evaluation of Formulation or Delivery Methods
- Assessing Treatment Combination or Sequencing