November 2007, Volume 4 Issue 5

Frequently Asked Questions About Clinical Trials

What is a clinical trial?
Clinical trials are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention and their outcomes are measured. Observational studies are those in which individuals are observed and their outcomes are measured by the investigators.

Why participate in a clinical trial?
The reasons people choose to participate in clinical trials are compelling: (1) To gain access to new therapies; (2) To advance science and help others who are trying to cope with illnesses; (3) To earn extra money; and (4) To receive free medical care. Only about 2% of the American population gets involved in clinical research each year.

"I didn’t enter the studies for relief, because there were other things I could take. Even if I found something better, it wouldn’t be approved for public consumption right away. The main reason I did this was because I had several friends who died from prostate involvement in their case, cancer. Any trial with 300 people is just a small step and being one part of 300 is an even smaller step, but it’s necessary. It’s the way the system works in this country, and it protects people. I saw it as an opportunity to do something for science."

- Robert, a subject in three enlarged prostate clinical trials

Who can participate in a clinical trial?
All clinical trials have guidelines about who can participate. 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.” Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. These criteria are based on such factors as age, gender, the type and stage of a disease, 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 clinical trial process depends on the kind of trial being conducted The clinical trial team includes doctors and nurses as well as social workers and other health care 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 protocol is carefully followed and there is frequent contact with the research staff.

Barbara Holtz says having cancer was terrifying but participating in a clinical trial made her feel she was “doing something about it.” “I had complete faith that this was the right step,” she says. The intensive monitoring she received throughout her treatment also proved comforting as she battled various side effects. “I’m glad I did it. The whole thing was a positive experience for me,” she says. “My message to others would be, have an open mind to being a clinical trial subject. Learn all you can about the trial’s purpose and requirements and go for it!”

- From Standing on the Shoulders of Those Who Came Before, The Participant, July 2007

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.

"I help (patients) understand that the clinical trial is being heavily monitored by regulatory individuals so they know that somebody’s looking over my shoulder. I also want them to understand what the potential outcome could be for the community because that’s often what they want to know. How is this going to help us? What am I getting out of this? You have to help them understand that. I think honesty is extremely important when you’re describing a clinical trial."

University of Pennsylvania
Associate Professor of Nursing
Christopher L. Coleman, PhD, MS,
MPH, APRN-BC, ACRN

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

Clinical trials that are well-designed and well executed are the best approach for eligible participants to:

• Play an active role in their own health care.
• Gain access to new research treatments before they are widely available.
• Obtain expert medical care at leading health care 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 to experimental treatment.
• The experimental treatment may not be effective for the participant.
• The protocol may require more of their time and attention than would a nonprotocol treatment, including trips to the study site, more treatments, hospital stays or complex dosage requirements.

What are side effects and adverse reactions?
Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

"I started chemotherapy on Oct. 7, 1974. I got the drug five days in a row. Then on October 21, I was back in the hospital from the side effects. It was my fourth protocol and I’d already been beaten pretty badly, and it was more of the same in a lot of ways. Vomiting, stomatitis (sores in the mouth and throat), high fever, muscle soreness. I was dehydrated, malnourished because of all the vomiting. It hurt so much to swallow, I’d go days without eating. So they put me in the hospital. Upon my admission to the hospital, they took a routine chest x-ray. The radiologist was reading my chest x-ray and he thought there was a mistake because two weeks after my first injection of the experimental drug cisplatin, my chest x-ray was clear."

- Former clinical trial participant John Cleland, a 37-year testicular cancer survivor

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, 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.

What should participants consider before enrolling in a clinical trial?
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the post of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the 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?
• What kind of preparation should a potential participant 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.

Who is looking out for me?
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.

"We need to educate the public so they ask the right questions and think a little harder about that they’re doing when they enroll in a clinical trial. Ensuring that clinical trial participants, understand the protocols, ask questions, and make educated, knowledgeable choices, will benefit all."

Patient Education Advocate and Vical Inc.
Executive Director of Oncology Clinical
Operations Linda G. Strause, Ph.D.,
The Participant, September 2007

Does a participant continue to work with a primary health care provider while in a trial?
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 having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.

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.

When the tumors came back two and a half years later, he tried a drug that was thought to break down the metabolism of tumors. It didn’t work. Neither did the next two study drugs he tried. One of them caused him to hallucinate. It was horrible, said Frederick. I had a lot of mental anguish. So he went back on the Gleevac, which at least killed some of the biggest tumors. But after six months, the drug started to lose its effectiveness and the tumors slowly began to reappear. During the extremely grueling eight months that followed, he was put on yet another experimental drug regimen. I finally dropped out. I was getting no results and feeling sad.

- Frederick, a gastrointestinal survivor and participant in multiple clinical trials

Where do the ideas for trials come from?
Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

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.

What are the different types of clinical trials?
Treatment trials test experimental treatments, new combinations of drugs, or new approaches to
surgery or radiation therapy. Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes. Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition. Screening trials test the best way to detect certain diseases or health conditions. Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.

What are the phases of clinical trials?
Clinical trials are conducted in four phases. The trials at each phase have a different purpose and help scientists answer different questions. Phase I trials test new drugs or treatments in a small group of people; Phase II trials expand the study to a larger group of people; Phase III trials expand the study to an even larger group of people; and Phase IV trials take place after the drug or treatment has been licensed and marketed.

"I was very happy, both with the benefit I received from the medication and the way the study was conducted. The study involved a placebo and a low dose and high dose of the study drug. I think I got the higher dose. It didn’t totally eliminate my symptoms, but it decreased the frequency of the hot flashes."

- Rose, a patient who participated in a six-month trial of an experimental drug for the treatment for hot flashes

To view other articles in this issue, click here.