The ins and outs of participating in a clinical study

Lupus Foundation of America

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Have you ever wondered how you can help in the fight for better lupus treatment? You don’t have to have a medical degree to get involved in lupus research—you can advance treatment by participating in a clinical trial. Participating in research is essential for furthering the understanding of lupus and other diseases and, ultimately, leads to more effective treatments.

“The role of volunteers as partners in clinical research is crucial in the quest for knowledge that will improve the health of future generations. The health of millions of people has been improved because of the willingness of thousands of individuals to take part in clinical trials, which in turn have brought advances in medical research,” says Steven I. Katz, M.D., director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases.

Of particular importance to readers of Lupus Now is that participating in clinical research can benefit someone with lupus, says Kenneth Getz, co-founder and board chair of the Center for Information and Study on Clinical Research Participation (CISCRP).

“Clinical trials also give the volunteer a sense of empowerment,” says Getz, who wrote Informed Consent: A Guide to the Risks and Benefits of Volunteering for Clinical Trials and co-founded CenterWatch. CenterWatch provides patients and their advocates with information services and educational materials on clinical research. “Throughout their participation, patients receive the benefit of interacting with very knowledgeable research professionals and experts,” Getz says. “Although their participation may not provide an effective treatment option for them directly, it will provide valuable medical information and insights that ultimately will benefit others facing the disease in the future.”

Evaluating New Drugs

More than 80,000 clinical trials are conducted in the United States every year, according to Getz. Observational studies are designed to find trends in certain aspects of medical interest. Interventional studies test new drugs, devices, or therapies.

One of the most common types of study—and, some would argue, the most important—is a clinical trial to test new drugs.

Precious few drugs are available to treat systemic lupus erythematosus, and no new drugs for lupus have been approved by the Food and Drug Administration (FDA) in decades, according to Joan Merrill, M.D., of the Oklahoma Medical Research Foundation and medical director of the Lupus Foundation of America.

The good news is that there are at least 20 compounds in the pipeline with therapeutic potential for people with lupus, Merrill says.

Federal law requires that all drugs approved by the FDA be proven safe and effective in well-controlled clinical trials. After studies with animals, tissue, or cell cultures show that a substance has a promising therapeutic effect, a drug company may file an investigational new drug application with the FDA and, if approved, may begin clinical trials with human volunteers. 

Phase I trials are usually conducted with small groups of people who may be healthy or have mild forms of the condition for which the drug is being tested. These trials evaluate the safety of a drug, determine a safe dosage range, and identify side effects. Drugs that pass phase I go on to phase II, which involves testing a drug in larger groups of people with the same illness to see if the drug is effective and to further monitor safety.

In phase III clinical trials, a drug is given to very large groups of people with the disease to determine its effectiveness, gauge how it compares with existing therapy, and collect information that will allow it to be used safely.

In each of these phases, there are two groups of participants: a group that receives the drug and a group that receives a placebo instead of the drug. A placebo, sometimes called a “sugar pill,” is a substance that does not have the active ingredient being studied. Depending upon the design of the study, a person will be randomly assigned to the control or placebo group.

But although those in the placebo group are not receiving the potential benefits of the drug, there still are advantages for them to take part in the study, says Ellen M. Ginzler, M.D., M.P.H., professor of medicine and chief of rheumatology at the State University of New York Downstate Medical Center, Brooklyn.

“They get more frequent follow-up, a thorough medical exam, and ready access to the medical professionals running the study,” she explains. 

In some cases, drug companies will then conduct phase IV clinical trials, also known as post-marketing surveillance or open label studies. These studies collect additional information about the long-term risks, benefits, and use of newly approved drugs.

Treatment and Control

Participation in a clinical trial isn’t for everybody. An ideal candidate for a clinical trial is somebody “who fully understands what the trial involves, and the possible risks and benefits to them,” says Merrill.

A clinical trial candidate “should have a level of symptoms or illness that is appropriate for the kinds of treatments and options that the trial protocol will provide,” she adds. “The participant needs to be an active part of the study team, doing their best to stick to the protocol, keep their appointments, and promptly report any changes in their medical condition.”

Because lupus is more common, and can be more severe, in people of color, it is especially important that lupus studies contain ethnically and culturally diverse populations.

Researchers today collaborate with a variety of medical centers and research institutions in their own and other countries. “Multicenter and multinational trials are intended to provide a mix of people,” says Ginzler, whose team is one of 30 centers in the United States and overseas that belong to the Systemic Lupus International Collaborating Clinics group.

Typically, there are specific criteria for inclusion in the trial. Researchers may be looking for people with milder, uncomplicated forms of lupus, for example, or people with family members who are also affected by lupus or other autoimmune diseases.

These details—and all other information essential to a clinical trial—are explained to potential study participants prior to enrollment, during the informed consent period. Informed consent is a process during which the research team discusses key facts of the study and answers any and all questions before a person agrees to participate. It is important to ask any questions you may have about the study and your role in it. The research team should be able to provide answers to your satisfaction. If you aren’t sure of something, ask the team member for clarification. Many people also find it helpful to bring along a family member or caregiver to meet the research team.

Clinical study participants typically receive a thorough medical evaluation and, once accepted, may get access to new therapies that are not widely available. On the other hand, people who volunteer for a clinical trial must understand that they might receive no personal benefit from the experience, says Ginzler.

The best reason for participating in a clinical study is that research demands it. Only through voluntary participation will progress be made in treating lupus and preventing its complications.