Hypersomnia Foundation

Participating in a Clinical Trial: Frequently Asked Questions

 

So, you are thinking about taking part in a clinical trial or research study, but you would like to know more about what this means. We have put together a list of frequently asked questions on the topic. In addition to reading this material, be certain to speak with the person responsible for the study, known as the primary investigator or PI, or another researcher or study coordinator about questions that you might have that are specific to her or his trial.

 

What is a clinical trial?

A clinical trial is a type of research study in which researchers test new ways to prevent, screen for, diagnose, or treat a disease. In this article, we will discuss clinical trials that are investigating treatments for a disease or condition. In a clinical trial, new treatments, such as drugs, devices, or procedures, are tested or existing treatments are tested in new combinations or for new uses. The purpose of a clinical trial is to see if a treatment is safe, effective, or both safe and effective. Some trials also look at other outcomes, such as change in quality of life.

 

What is the difference between a clinical trial and a research study?

All clinical trials are research studies, but not all research studies are clinical trials. Research studies may be observational – they might simply watch a group of people who have been
exposed to something to see if it causes disease in the future, or follow patients with a specific disorder over time. Clinical trials involve human beings. Other research studies may involve animals, basic research at the cellular level, or other types of research that don’t involve the testing of a treatment in people. New treatments are first tested in animals or in other ways before being tried in humans.

 

What is a study protocol?

The protocol is the plan of action for a clinical trial, set up to answer a series of questions that the researchers developed. Study protocols typically outline

 

What is randomization or a randomized controlled trial?

Some trials test a single treatment in all participants. Others separate the participants into one or more groups and test the treatment against another drug or against a placebo (a substance that looks like the treatment being studied but that has no active ingredient). When participants are assigned to different treatments, it is important that this occurs without bias, which is why treatment assignments are done randomly. Randomization is essentially a coin toss to determine who will receive the treatment being studied and who will receive the placebo (sham treatment) or other treatment (active comparator). These groups are often known as arms of the study. The people who receive the treatment being studied are in the active arm, and the others are in the control arm. When there is no approved or available treatment for a condition, the active arm receives the treatment being studied, and the other group receives a placebo. When a treatment is available for a condition that is considered to be the standard of care, the active arm again receives the treatment being studied, but, in this case, the control group receives the standard treatment.  There are also variations in study design that allow each participant to receive both placebo and active drug for a defined period of time.  These studies are called “crossover” studies. In these types of studies, the order in which people receive the different treatments is determined randomly, and the responses participants have to each of the different treatments are compared.

 

What is meant by the term blinding?

In a blinded study (actually masking or masked is a better but infrequently used term), the participants, the researchers, or both do not know to which arm a given participant is assigned. When neither the participants nor the researchers know the assigned group, the study is said to be double-blind.  This helps minimize the chance that bias will affect the results of the study.

 

What is an Institutional Review Board?

An Institutional Review Board or IRB is a group of people (scientists, statisticians, and members of the general public) who review study protocols to ensure that participants are kept safe and treated ethically and that the benefits of participating in the study are proportionally higher than the risks. Academic medical centers (doctors or hospitals associated with universities or medical schools) typically have their own IRBs, but there are also commercial IRBs that charge a fee to review study protocols, which are subsequently and typically conducted outside of academic medical centers. Institutional Review Boards also review and approve consent documents used in each clinical trial.

 

What is meant by informed consent?

Informed consent is the process by which you learn about a given research study in which you might choose to participate. The study coordinator, the doctors or scientists involved in the study, or nurses will explain all aspects of a clinical trial to you in terms that you can understand. After talking with you, they will give you a printed version of this information, which you should carefully review. Once you are comfortable that you understand all of the information presented to you and have made the decision to take part in the study, you sign a consent form. This form is not binding, however. At any time before or throughout the study, you can stop taking part in the trial (that is, you can withdraw consent) without penalty to you.

 

Are there any other safeguards besides informed consent that protect research participants?

The first round of safeguards involves the Institutional Review Board. In addition, the federal government regulates most clinical trials that take place in the United States through the Office of Human Subjects Research Protection and the Food and Drug Administration.  The research institution in which the study takes place – whether a university medical center, private hospital or doctor’s office, or some other setting, should also oversee the safety of the participants. Finally, each protocol should include ongoing plans for monitoring the data that are gathered in the trial and participants’ safety.

 

Why should I participate in a clinical trial?

People decide to take part in a clinical trial for any of a number of reasons. Most studies need healthy people to participate in trials early in the process to test the safety but not the effectiveness of a treatment. For others, perhaps there is currently no treatment for their medical condition or the available treatments are ineffective and they want to have a chance at being in the arm of the study that receives the new treatment. Some people participate in clinical trials for altruistic reasons—they simply want to help others with their condition. For example, a person with a disease might currently have their symptoms under good control with their current drug regimen, but a new treatment is being studied in a clinical trial. They might have to stop their current treatment to take part in the new study, but they do so with the intention of helping to make a new treatment available for others.

 

Who pays for clinical trials?

Pharmaceutical companies, academic medical centers, nonprofit organizations and foundations, the National Institutes of Health, the U.S. Department of Defense, the U.S. Department of Veterans Affairs, physicians, other health care providers, and other individuals can all sponsor clinical research.

 

How do I find out about what clinical trials are available?

Many clinical trials in the United States are registered at clinicaltrials.gov. The Food and Drug Administration requires registration for all interventional studies (with one or more arms) of drugs, biological products, or devices that are subject to FDA regulation, meaning that the trial has one or more sites in the U.S; involves a drug, biologic, or device that is manufactured in the US (or its territories); or is conducted under an investigational new drug application or investigational device exemption. In addition, most medical journals now require, as a term of publication, that the clinical trial had been registered at a publicly available site such as clinicaltrials.gov.  Be sure to explore research studies currently looking for participants with idiopathic hypersomnia, Kleine-Levin syndrome, and narcolepsy.