What are Clinical Trials?

What is a clinical study or "clinical trial"?

Clinial trials are medical research studies carried out under very strict guidelines and they are the building blocks of scientific progress. Researchers, physicians and other healthcare professionals rely on these studies for scientifically sound information on whether or not treatments are safe and effective.

How does a clinical trial work?

To start, the research team must first determine the objective of the study - for example, whether an experimental treatment is safe or whether a treatment known to work for hyperhidrosis on one part of the body will work for hyperhidrosis on another part of the body. The research team then designs the study to answer that question, including developing a protocol for the trial.

Clinical trials with human subjects follow only after the completion of tests using laboratory animals. These trials fall into four categories called phases. In Phase I a trial tests a potential new treatment with a small number of volunteers (20-80 individuals) to determine the treatment’s safety and potential side effects. Phase II studies test a treatment with known dose and side effects with a larger number of volunteers to learn more about side effects, how the body responds to the treatment, and whether the treatment is effective in treating the condition. The new treatment is compared with commonly used treatments and monitored for long-term side effects in Phase III and IV trials. Phase IV trials are done after the treatment is marketed.

Clinical researchers call the standard scientific approach for trying out treatments a “double-blind, randomized, controlled clinical trial.” Such trials are "grade A" research because the researcher has taken precautions to get the most unbiased results possible. Ideally, such research can also be replicated; that is, another researcher using the same protocol would get similar results.

The "control" aspect refers to the fact that researchers are usually looking at how one treatment stacks up against another or even against no treatment at all. To make the comparison useful, the investigator must try both methods on similar groups of subjects. Researchers call the treatment with the predictable, or known effect, the control. The control may be a standard, commonly used treatment, or it may be a placebo. A placebo is an inactive pill, liquid, powder, or other modality with no treatment value (commonly referred to as a "sugar pill"). Some studies use both a standard treatment and a placebo as controls. The control helps an investigator find out if any changes in the experimental group are, in fact, due to the new treatment.

Trials are randomized because researchers assign patients by chance either to the group getting the new treatment (called the treatment group) or to the group receiving a standard treatment or placebo (called the control group). This randomization keeps the study's results from being affected by human choices or other factors not related to the treatments being tested.

In some studies, the patient does not know whether he or she is in the treatment or control group (a single-blind study); in others, neither the patient nor the researcher know (a double-blind study). This, again, is a way of avoiding bias in the results. Sometimes when people know what treatment they are getting, it changes the way they react; similarly, the researchers' own expectations about the treatment could influence how they perceive patients' reactions, side effects, and progress during the study.

Participants in clinical trials are randomly assigned once they've been accepted into the study and have agreed to participate by signing an informed consent.

Who can participate in a clinical trial?

All clinical trials have guidelines about who can participate. These guidelines are an important part of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called "inclusion criteria" and those that disallow someone from participating are called "exclusion criteria". The criteria are based on such factors as age, gender, the type and stage of a disease or condition, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

What happens during a clinical trial?

The exact clinical trial process depends on the kind of trial being conducted. In general, the clinical trial team includes doctors and nurses as well as social workers and other healthcare professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the trial’s specific instructions (or protocol) are carefully followed and there is frequent contact with the research staff.

What is informed consent?

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided. Then the research team provides an “informed consent document” that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

What are the benefits and risks of participating in a clinical trial?

Benefits
Clinical trials that are well-designed and well-executed can help eligible participants to:

• Play an active role in their own healthcare.
• Gain access to new treatments before they are widely available.
• Obtain expert medical care at leading healthcare facilities during the trial.
• Help others by contributing to medical research.

Risks
There are risks to clinical trials.

• There may be unpleasant, serious or even life-threatening side effects related to experimental treatment.
• The experimental treatment may not be effective for the participant.
• The trial's instructions or protocol may require more time and attention than would a non-protocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

How is the safety of the participant protected?

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built in safeguards to protect the participants. The trial follows a carefully controlled protocol which is a set of instructions or a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.

Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.

What should I consider before participating in a trial?

If you are considering participating in a clinical trial, talk to a healthcare professional who knows your medical history and is familiar with hyperhidrosis (preferably a dermatologist). For help finding a physician, physician assistant or nurse practitioner, visit our Clinician Finder. Also discuss clinical trial options with your loved ones. Before making any decisions, you should know as much as possible about the clinical trial and feel comfortable asking the members of the healthcare team questions about it - such as the care you may expect while in a trial, and the cost of the trial. The following additional questions might be helpful to you if you decide to discuss participation in a trial with its healthcare team. Some of the answers to these questions will also be found on the trial's informed consent document.

• What is the purpose of the study?
• Who is going to be in the study?
• Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
• What kinds of tests and experimental treatments are involved?
• How do the possible risks, side effects, and benefits in the study compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the experimental treatment?
• Will I be reimbursed for other expenses?
• What type of long-term follow up care is part of this study?
• How will I know that the experimental treatment is working? Will results of the trials be provided to me?
• Who will be in charge of my care?

If I decide I want to pursue participation in a clinical trial, what kind of preparation should I make for the meeting with the research coordinator or doctor?

• Plan ahead and write down possible questions to ask.
• Ask a friend or relative to come along for support and to hear the responses to the questions.
• Bring a tape recorder to record the discussion to replay later (be sure to ask for and receive permission before recording).

If I'm enrolled in a clinical trial, will I continue to work with my primary healthcare provider?

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by including your healthcare provider in your clinical trial experience and getting him/her involved with the research team can help to ensure that any other medications or treatments you may be using will not conflict with the study or pose any additional risks.

Can a participant leave a clinical trial after it has begun?

Yes. A participant can leave a clinical trial at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.

Who sponsors clinical trials?

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.