Phase 1 Trial

In Detail

A Phase 1 trial is the earliest stage of clinical testing in humans. Its primary goal is to evaluate the safety profile of a new drug or treatment, determine the appropriate dosage range, and identify side effects. Phase 1 trials typically enroll between 20 and 100 healthy volunteers or patients, though cancer Phase 1 trials often enroll patients with advanced disease who have exhausted standard treatments. Researchers start with a very low dose and gradually increase it, a process called dose escalation, while closely monitoring participants for adverse reactions. These studies usually last several months and are conducted at specialized research centers with intensive medical oversight. Phase 1 trials also collect pharmacokinetic data, which describes how the body absorbs, distributes, metabolizes, and excretes the drug. Approximately 70% of Phase 1 drugs move on to Phase 2 testing. While Phase 1 trials are not designed to test whether a treatment works, researchers may observe preliminary signs of efficacy. Participants in Phase 1 trials receive extensive informed consent about the experimental nature of the study and the potential for unknown risks. These trials are a critical safety gateway, no treatment can advance without first demonstrating acceptable tolerability in humans.