What is a Phase 1 trial?

A Phase 1 clinical trial is the first step of testing a new drug or treatment in humans. At this stage, researchers are focused on evaluating safety and, for medicines, learning about dosage levels. These trials are small in size and typically seek healthy volunteers for participation. These individuals can help provide a clearer assessment of safety since they do not have serious medical issues that could interfere with how the body processes the therapy. In addition, heathy volunteers are less likely to experience distress from any potential side effects. 

Phase 1 trials lay the foundation for further phases of clinical testing. When a study shows that a therapy met its phase 1 goals, the next stop is a phase 2 trial. Phase 2 trials require larger participant pools (usually hundreds) who have been diagnosed with the condition the therapy is intended for. These trials build upon the dosing knowledge gained during the phase 1 study. Here the primary goal is to assess how well the drug works (its efficacy) along with continuing to look at overall safety.

When phase 1 trials need participants, study organizers commonly use advertisements to the general public – posters, social media posts, and radio ads. Sometimes doctors will have good knowledge of an early phase trial and be able to refer a patient. Other times a research site will have a database of interested people and proactively call or email about a potential study opportunity. 

As part of the recruitment process, interested individuals will answer questions about their health status and history. They may also be asked to undergo a medical exam or testing to make sure they are a fit for the needs of a study. 

Prior to enrolling a volunteer, study researchers will fully explain any risks of participation and reinforce the fact that participation is totally voluntary. This conversation will also detail the length of the trial, the number of visits and any tests, potential side effects, and the safety monitoring in place. Side effects in these early trials may include nausea, headache, or skin redness/swelling (if an injection is given). All of this information will also be provided in a written document which the participant will be asked to sign (this is called Informed Consent).

Phase 1 trials happen in every clinical area for all kinds of medical issues. Infectious diseases like COVID-19 continue to have many studies running that are looking at both vaccines and treatments. Alzheimer’s disease, autoimmune diseases, heart conditions and many forms of cancer are all experiencing high early-stage research activity as well.

People who volunteer in a Phase 1 study often feel quite positive about their contributions to science. And since there is unlikely to be any potential for physical or medical improvement associated with participation, volunteers are often financially compensated to become, and stay, involved with a trial. Payment, when available, will vary based on complexity of the trial.

Interested in helping researchers discover breakthroughs that can change lives? See what is recruiting today by using our search and filtering on “Phase 1 trials.”