As part of clinical research, there are four phases in the clinical trial process. The first is known as Phase 1 or early phase clinical trials. A Phase 1 clinical trial tests the safety, side effects, best dose and timing of a new treatment.

Norton Cancer Institute Early Phase Trials Program

What Is Phase 1 in Clinical Trials?

An early phase clinical trial, also known as a Phase 1 or sometimes Phase 1/2 trial, is the first step in testing a new drug or treatment in humans after it has shown promise in preclinical studies. It examines the safety, side effects, best dose and timing of a new treatment. It also may test the best way to give a new treatment (for example, by oral intake, infusion into a vein, or injection) and how the treatment affects the body. Phase 1 trials can occur only when there has been demonstrated preclinical safety and efficacy data available from animal studies, modeling or other predictive studies. Later clinical trial phases include giving the treatment to a larger and larger group of patients, compared with other treatments. If proven safe and effective, the treatment can be approved for use by the public or other researchers.

Why Are Phase 1 Clinical Trials Important?

Phase 1 clinical trials play a critical role in the development of new treatments by evaluating safety, dosage levels and effectiveness in human participants. These trials are pivotal, as they provide the first opportunity to assess how a potential treatment interacts with the human body and identify any adverse effects or risks it may pose.

Researchers carefully monitor participants to determine the optimal dosage and assess the treatment’s safety profile. The data obtained from Phase 1 trials informs subsequent phases of clinical testing and guides decisions regarding further development. Additionally, regulatory agencies rely on the findings of Phase 1 trials to assess the treatment’s potential benefits and risks, ultimately influencing its approval process. In summary, Phase 1 clinical trials are essential to advancing new therapies through the clinical development pipeline.

Who Can Participate in a Phase 1 Clinical Trial?

Many types of people can and should be part of human clinical trials. However, this phase of trials typically has a small number of participants who have a very specific set of characteristics. Clinical trials follow a specific set of criteria for participants and may include healthy patients and those with specific conditions or diseases. People experience diseases differently, so it is important that Phase 1 trials include people of all socioeconomic backgrounds, ethnicities, sex assigned at birth and other characteristics so that all people can benefit from the results of early phase clinical trials.

What Are the Kinds of Phase 1 Clinical Trials?

There are several different types of clinical trial. All human trial protocols have different requirements for participants.

• Screening: Tests new ways to detect health conditions and diseases via blood tests, machine testing (such as X-ray) or other tool
• Behavioral: Evaluates changes you can make to your life that may improve your health, prevent disease from developing or recurring
• Diagnostic:  Compares tests or techniques for diagnosing a specific disease
• Quality of life: Explores ways to improve the life of people with certain conditions, such as reducing pain
• Treatment: May test a new medical device, an experimental drug or a new surgical technique

Why Should You Be Part of a Phase 1 Clinical Trial?

By participating in a Phase 1 clinical trials program, you are at the forefront of groundbreaking research. These tests are aimed at developing new treatments for some of the world’s most debilitating conditions and deadly diseases. Human clinical trials are the initial step in assessing the safety and effectiveness of promising therapies. As a participant, you’ll receive careful monitoring and support from our team of dedicated medical professionals, prioritizing your safety and well-being.

What Are the Benefits of Joining a Clinical Trial?

Participation in an early phase clinical trial can provide you with access to new therapies or drugs that may not be available otherwise. Many participants appreciate the close collaboration with their health care team and the availability of additional education about their condition.   

What Should I Know About Being Part of an Early Phase Clinical Trial?

Clinical trials may involve risk. Your clinical trial care team will discuss with you:

• The possible risk of harm and the chance of harm occurring
• Any long-term effects of the clinical trial

There may be some discomfort associated with a clinical research trial. It may short-lived and minor. Some early phase trials involve more serious complications or long-term effects. All risks are discussed in the informed consent documents that you will receive and sign before the trial begins. You should carefully consider the risks as they are presented to you and ask your care team any questions you have.