WHAT ARE CLINICAL TRIALS?

What is clinical research?

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Clinical research is medical research involving human participants. There are two types of clinical research: clinical trials and observational studies.

What are clinical trials?

Clinical trials are research studies that aim to evaluate a medical, surgical, or behavioral intervention in people. They are the primary way that researchers can investigate whether a new treatment is safe, effective, or safer/more effective than standard treatments.

How do clinical trials work?

Before the US Food and Drug Administration (FDA) approves a clinical trial to begin, scientists perform laboratory tests and studies in animals to test a potential therapy’s safety and efficacy. If these studies show favorable results, the FDA gives approval for the intervention to be tested in humans.
Once approved, clinical trials proceed in 4 phases:

 

• Phase I trials test an experimental treatment on a small group of often healthy people (20 to 80) to evaluate safety, side effects, and the correct drug dosage.

• Phase II trials use more people (100 to 300). While continuing to study safety, including short-term side effects, these trials emphasize efficacy and gather preliminary data on whether the drug works in people with the target disease or condition.

• Phase III trials gather more data on safety and efficacy by studying different populations, dosages, and drug combinations. Study populations range from several hundred to several thousand. If Phase III results are positive, the FDA will approve the experimental drug or device.

• Phase IV trials take place after FDA approval. A drug or device’s safety and efficacy are monitored in large, diverse populations to further investigate possible side-effects, especially those that may only become apparent in large populations or after a long period of time.

In order to conduct clinical trials, especially Phase II and Phase III trials, researchers need to ensure they study sufficiently large and diverse patient populations to get high-quality data. As a result, pharmaceutical companies recruit clinical centers around the country/world to help recruit participants, facilitate their involvement in a trial, and gather research data.

We are one of these sites. Most of our studies are Phase II and III clinical trials that evaluate pharmaceutical drugs. Some of our studies involve novel drugs, while others investigate the use of an already FDA-approved drug for a different indication. For example, some drugs that are commonly used to treat psoriasis have also been shown to improve conditions like hidradenitis suppurativa. Each new indication for a drug needs another round of clinical trials and FDA approval because safety, efficacy, and dosage measures may be different.

What are observational studies?

Observational studies observe people in normal conditions, i.e. receiving either no treatment or standard care, without any investigational intervention. Researchers gather data, group volunteers according to broad characteristics, and compare changes over time. Data collection may involve medical exams, tests, and questionnaires administered at various points in time throughout the duration of the study. These studies help elucidate disease characteristics and identify new possibilities for clinical trials.

We currently run several observational studies in addition to our clinical trials. These studies are also sponsored by pharmaceutical companies.

Why participate in clinical research?

There are many reasons people choose to join a clinical trial. Oftentimes, patients may not see improvement using typical treatments or experience adverse reactions to them. In other cases, no treatments for the condition may currently be available. Many people enjoy playing a more active role in their own health care and helping researchers learn more about certain health problems. Our clinical studies are always entirely sponsor-funded; participants will not pay any costs of treatment or medication and may be provided a small stipend.

 
Whatever the reason, participation in clinical research is vital to medical discovery and advancement, and many treatments available today and in the future depend on the generosity of clinical trial participants. We have a deep gratitude and appreciation for anybody interested in participating in clinical research!

What happens during a trial?

Here’s what happens in a typical trial:

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1. After initial contact has been established and you are deemed possibly eligible for a study, a study coordinator will explain the trial in detail and gather more information about you. For most studies, this will involve an in-person "pre-assessment" visit to determine eligibility.

2. We will schedule an official screening visit with you. At the screening visit, you will read an informed consent form (ICF), and we will answer any of your questions. The ICF will explain important information about the study, safety and privacy measures, and your rights as a participant. It is crucial that you read the entire ICF and have all of your questions answered before consenting to the study.

3. If you agree to participate, you will sign the ICF and be screened to make sure you qualify for the trial. Each study has different inclusion/exclusion criteria involving demographic, disease, and medical history factors. Screening visits can involve other assessments, such as questionnaires, physical exams, vital signs, blood draws for labs, and EKGs.

4. If you are accepted into the trial after the screening visit, we’ll schedule a baseline visit. You will be randomly assigned to a treatment or control group and be given the corresponding treatment. More safety assessments will also take place.

5. You will follow the trial procedures and report any issues or concerns to researchers. For example, a study may or may not involve a washout period of specific medications prohibited during the study.

6. You will visit the research site at regularly scheduled times for new cognitive, physical, or other evaluations and discussions with staff. At these visits, the research team collects information about effects of the intervention and your safety and well-being. The frequency of visits, as well as the study duration, is dependent on the specific study. You may ask for this information at your screening visit.

7. You may continue to see your regular physician for usual health care throughout the study. It is very important that you discuss participation in any clinical study with your physician.

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Adapted from "What are Clinical Trials and Studies?" the National Institute on Aging (NIA), part of the National Institutes of Health, 2020. https://www.nia.nih.gov/health/what-are-clinical-trials-and-studies