Clinical Trial (Clinical Study)

What is a clinical trial?

A clinical trial is medical research that involves people who volunteer to take part in different medical studies. Clinical trials are significant because they’re the only way for researchers to find out if a potential new treatment works and is safe.

There are four types of clinical trials. A trial may focus on new ways to detect, prevent, diagnose or treat diseases. This article is about clinical trials for new treatments. Medical researchers may call these treatment trials. Treatment trials may test new drugs, existing drugs, devices or other treatments.

You may be interested in volunteering because you have the disease that’s under study and participating in a trial is a way to receive a new treatment. But clinical trials also involve people who aren’t sick but want to help people who are sick or support medical research.

What happens before a clinical trial starts?

There’s a lot that goes on before researchers launch a clinical trial. In general, the clinical trial process includes:

• Preclinical tests: All clinical trials start with a theory about a new therapy like a new drug, a combination of drugs or medical devices. Preclinical tests may involve laboratory tests or studies in animals.
• Clinical trial protocol: If preclinical tests show a potential new treatment is effective, researchers develop an action plan or clinical trial protocol. Protocols vary but must include the trial purpose, the kinds of treatments under evaluation and trial endpoints, or what researchers expect they’ll be able to measure when they complete the trial.
• FDA review and approval: Clinical trials in the U.S. require FDA approval before researchers can start the trial.
• Institutional review and approval: This step involves the institutional review board (IRB) or ethics committee for any hospital participating in a clinical trial. The board’s focus is on participant safety and rights.

With approval in hand, researchers can start looking for participants. They may contact people who register to participate. Often, healthcare providers refer people to specific clinical trials.

If you’re considering a clinical trial, healthcare providers who are coordinating the research will explain the trial so you can decide if you want to take part. They may call this the informed consent process.

Informed consent

This conversation is when you learn more about the clinical trial. The research team will share details about what you can expect.

They’ll walk you through an informed consent document. The document is a very detailed description of the trial goals and processes. It also lists any risks and potential benefits. It includes information about any experimental procedures and alternatives to being in a clinical trial. The informed consent document also contains information about how long the trial may last, any follow-up requirements, any costs and compensation. Finally, the document notes that any participation in a clinical trial is voluntary.

The consent document will state if the trial will be randomized, like flipping a coin. That means not everyone in the trial will receive the experimental treatment. That’s important information to know if you’re sick and are joining a trial in hopes of receiving a new treatment.

Clinical trial details may be technical and confusing, so take your time and ask as many questions as you want. An informed consent document isn’t a contract between you and healthcare providers managing the clinical trial. If you decide to enroll in a trial, you’ll need to sign the informed consent document. But signing it doesn’t mean you’re obligated to participate. You can change your mind at any time, including after the trial begins.

What happens during a clinical trial?

Clinical trials often have four phases or testing periods. Each phase has a specific purpose, and researchers seek new volunteers for each phase. The four phases are:

• Phase 1: This phase usually involves a small group of people (20 to 100) who don’t have known medical issues such as healthy volunteers. Researchers do phase 1 trials to establish dosages and see if a treatment causes severe side effects.
• Phase 2: This phase involves 100 to 300 people, all of whom have a specific disease. In Phase 2, researchers focus on whether a treatment works and if it causes new or more severe side effects.
• Phase 3: Phase 3 trials may involve 300 to 3,000 people. This phase is when researchers confirm that an experimental treatment works and is safe. They may compare treatment with current treatments. In the U.S., phase 3 trial results determine whether the FDA approves the treatment for more people to use.
• Phase 4: Researchers monitor the treatment after FDA approval. Some new treatment side effects don’t show up until people use the drug for some time, so researchers also check for side effects or other issues.

What happens after a clinical trial?

Clinical trials end for several different reasons. For example, an experimental treatment may receive FDA approval and be available for more people to use. But a clinical trial may end without going through all four phases because tests show the treatment doesn’t work as expected or doesn’t work better than existing treatments.

What are the benefits of joining a clinical trial?

There are several potential benefits of being part of a clinical trial. If you’re ill, a clinical trial may give you the chance to:

• Get access to new treatments that may be more effective than existing treatments.
• Take a more active role in your healthcare.
• Help others with the same illness.

If you’re healthy, you may benefit by knowing you’re helping researchers find better treatments for others.

What are the risks?

Clinical trials do come with some risks. You learn about risks during the informed consent process, but you may always ask for more information at any time during a trial. You’ll learn about any changes in risks or new risks that may develop during the trial and make a difference in your willingness to participate. Potential risks may include:

• The experimental treatment may cause side effects that require medical care.
• The trial may take more time than the standard treatment for your condition. For example, you may have more appointments, tests or hospital stays than you would with the standard treatment.
• The experimental treatment may not work or may not work better than the standard treatment.
• You may not get the experimental treatment being tested. You may get the standard treatment for your condition or no treatment at all.

What are questions I should ask about a clinical trial?

If you’re like most people, you’ll have many questions about what goes into being a clinical trial volunteer. Here are some suggested questions you may want to ask:

• Why is the study being done?
• How long will my participation in the study last?
• If I receive treatment, how will it be given (pill, IV, implant, etc.)?
• How many visits will I make?
• Where do the visits take place?
• How much time does each visit take?
• Do I have to pay for any part of the study?
• Will my insurance cover any parts of the study?
• What are the risks of the study? How might this affect my health?
• What will happen to my medical care if I choose not to participate or withdraw from the study?
• Who will be in charge of my care?
• Will I receive any results from this study?
• If the treatment receives approval, will I continue to receive it?
• If I have bad side effects from the treatment, can I withdraw from the study?

Who ensures clinical trial safety?

There are safeguards in place throughout the clinical trial process to protect volunteers’ safety:

• Data safety and monitoring boards (DSMBs) include medical experts who review results throughout a clinical trial. These boards can stop trials early if they decide an experimental treatment doesn’t work or side effects are more severe than expected.
• Government agencies like the FDA and the Office for Human Research Protections regulate and monitor all clinical trials in the U.S.
• Institutional review boards (IRBs) at study locations must approve a clinical trial before the trial can take place at the location. The boards protect volunteer safety by making sure trials don’t expose them to unnecessary risks. IRB panels typically include members of the community and providers and researchers who aren’t part of the trial team.

What’s a randomized clinical trial?

In a randomized trial, researchers place volunteers in different groups that receive different treatments. The trial may test a standard treatment against an experimental treatment or test two experimental treatments. The group placements are by chance, or at random, like a flip of a coin. That way, the groups are similar. That helps researchers find out which treatment is best.

What’s a decentralized clinical trial?

A decentralized clinical trial is when trial activities happen in several locations. Clinical trials traditionally require volunteers to travel to a specific site. In a decentralized trial, volunteers can take part without traveling. For example, you may receive treatment in your home, a local healthcare facility or a nearby laboratory.

Who can participate in a clinical trial?

Clinical trials have eligibility guidelines that outline who can and can’t participate. Not everyone who wants to be in a clinical trial can participate. Guidelines may vary but typically include factors like your age, gender, sex, overall health, the type of disease you have and what treatments you’re receiving.

How do I find out about clinical trials?

First, talk to the healthcare provider managing your treatment. They know you and your situation and may be able to recommend trials for you to consider. Another source is the clinical trial database that the U.S. National Center for Biotechnology Information maintains.

How is a clinical trial different from treatment?

The difference is the kind of medical care you receive. If you’re sick and participating in a clinical trial, your regular healthcare team will continue to take care of your ongoing medical issues. The clinical trial team’s focus is on the new drug or device under consideration. That team will monitor your reaction to the experimental treatment and treat any side effects that you may experience.