The phase 1 study is the gateway between scientific research and clinical outcomes. The primary objectives of a phase 1 study are safety, tolerance, pharmacodynamics and pharmacokinetics.
Oncology studies add an additional layer of complexity because the patients aren’t healthy volunteers. For immuno-oncology studies, there are an entirely different set of complexities.
If you’re a new researcher planning your first phase 1 oncology study, here are some key points that you should be considering.
Patient Population
- How do you overcome the challenges for efficient inclusion of precisely defined patient populations in a FIH study?
- Have you considered how you will design efficient inclusion in a FIH study vs a healthy patient population?
Safety
- There are safety and efficacy challenges with novel, targeted anti-cancer drugs, requiring additional consideration. Have you considered the challenges that may arise with determining the definitions of a biological dose? The collection of fresh tumor tissue for surrogate marker analyses? The management of infusion-related reactions with monoclonal antibodies?
Site Selection
- Training of sites is crucial in conducting a successful FIH study; how will you choose which sites to work with? How will training of medical staff be conducted?
Dose
- The optimal biological dose defines the threshold at which that product is efficacious, but not yet toxic. How will you avoid under-dosing patients, while maintaining reasonable dose escalation steps?
Preparing for Your First Immunotherapy Study
If you’re planning an immunotherapy study, there are numerous additional planning considerations. With the explosion of research in immunotherapy studies and molecule combinations, it’s critical for sponsors, sites and investigators — and everyone else working on your immunotherapy trial – to understand how the immune system responds to cancer treatments and to properly prepare for it in the design stage. This can help alleviate many of the common factors that cause delays in immuno-oncology studies.
Site Selection
- The immunotherapy experience of a site plays a critical role in the study timeline. Many sites are new to immunotherapy studies and require significant training to properly manage one.
SAE and irAE Management
- Since multiple studies have shown that there is an association between irAEs and clinical benefit, it’s important to have a comprehensive approach for evaluating and treating SAEs. Therapies targeting checkpoint receptors have shown to increase toxicities that can create a unique set of adverse events called immune-related adverse events (irAEs).
Site Training, Manufacturing, Drug Delivery and Regulatory
Medelis is an oncology CRO that has been managing immunotherapy studies in oncology since 2008. We work directly with sponsors on the development of clinical strategy (protocol, endpoints, regulatory) and with study execution in this growing field.
If you’d like an expert review of your study timeline and plan, contact us.
