What is a Phase 1 Clinical Trial?

Once preclinical research has shown that a clinical method can potentially work and appears safe, it is tested on a small group of volunteers for a few hours or days, a few months, or even up to a year or two. If the clinical trials are studying cancer or rare diseases, the phase 1 clinical trials may enroll patients rather than healthy volunteers.

In the case of potential medical drugs or treatments, phase 1 trials determine safety for humans in a few different ways, including adverse effects, how the human body absorbs, distributes, and eliminates the drug, and whether it is safe to use with other medications.

If the treatment and method of administration appear to be safe in healthy people at the end of phase 1, it will be tested on more people with the targeted disease in the next phase of development (Phase 2). According to the Food and Drug Administration (FDA), 70% of drugs move from phase 1 to phase 2.

Understanding Clinical Trial Phases

Clinical trials follow a specific timeline, from early, small-scale, phase 1 clinical studies to late-stage, large-scale, phase 3 studies. There are many clinical trial phases involved in developing new treatments; each phase answers important questions that help determine the safety and efficacy of a treatment.

Purpose of Phase 1 Clinical Trials: Safety and Dosage

The primary purpose of a Phase 1 study is to evaluate the safety of a new drug before it proceeds to further clinical studies. In addition to safety, the research team can answer other questions in the Phase 1 clinical trial about how much drug is measured in the blood after administration, how the drug works in the body, and the side effects of increased dosage.

Key Points of Phase 1 Clinical Trials

• The first few people in the clinical study receive a very low dose and are monitored very closely. If there are only minor adverse effects, the next participants get a higher dose. This process will continue until doctors find a dose most likely to work while having an acceptable level of side effects.
• Phase I trials also examine the effects of the drug on the body and the body's response to the drug.
• Safety is the primary concern. The research team closely monitors participants and watches for any severe side effects. Due to the small number of people in phase I studies, rare adverse effects may not be seen until later phases of clinical trials when more participants receive the treatment.
• Disease response is not the main concern of a phase I trial.
• Phase I trials usually involve fewer people.
• Phase I trials often include people with various types of disorders/illnesses.

Number of Participants

A drug is given to a small number of volunteers who are closely monitored: 20 to 100 healthy volunteers or people with the disease/condition.

Determination of Drug Dose and Schedule

A treatment plan usually consists of 1) the amount of medication to be given to a patient, 2) how the medication will be given, and 3) the times when it will be given. The former is known as the dose, and the latter is known as the schedule of the treatment.

In a dose-schedule-finding trial, the investigators aim to identify an optimal combination of dose and schedule of an investigational drug that is both tolerable and sufficiently active to warrant further testing of the medication.

Uses of Placebo

In this group, patients receive inactive medicine. Placebos are often called "sugar pills" but are not limited to oral medications. They are only approved to be used if receiving a placebo would not be harmful to any study participant.

It's important to note that not all studies involve placebos or control groups, and the trial design is decided on before a clinical trial starts. Placebos are not allowed to be used in a study if it would mean putting people at risk by not letting them have effective therapy. For example, in cancer clinical trials, placebos are rarely used. There are also types of trials where placebos are rarely used. For example, phase 1 trials and studies that involve new treatments rarely use placebos.

In several cases, patients will not have to stop their current therapy if they're placed in the placebo group. Furthermore, in trials with a control group, it's common that patients who initially don't receive the new treatment will be switched to receiving the study treatment later during the study. This usually occurs after 8-12 weeks when sufficient information has been collected on how they perform without the new treatment.

Safety and Toxicity Evaluation

Before a clinical trial starts, researchers perform extensive preclinical studies in the lab to ensure that their methods (e.g., drug, procedure, preventative method, or diagnostic) are not harmful to people. The level of harm is measured in terms of toxicity.

The research in preclinical trials is not conducted on people. Instead, potential drugs and therapies, and the methods to administer them, are first tested in cells or animals, or both, long before they make it to human trials.

Usually, preclinical studies are not very large. However, these studies provide detailed information on dosing levels and are required before clinical trials can begin.

After preclinical testing, researchers review their findings and decide whether the method should be tested on people. If the treatment appears safe in cells or animals, the process continues and proceeds to phase 1, where the potential medical treatment is first tested in humans.

Length of Study

Phase 1 clinical trials in healthy participants can last anywhere from several days to months.

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At Santos Research Center, Corp., we have completed over 400 clinical trials. We make use of efficient and reliable systems to plan and manage quality medical research studies.

For more information about Phase 1 clinical trials, you may visit us at www.santosresearch.com.

If you are interested in participating in a clinical trial, call us at (813) 249-9100.