What are Clinical Trials and What to Consider about Participating

There are many types of clinical trials, but two that may be of particular interest are:

1. Biomedical studies that involve the application of principles of the natural sciences, especially biology and physiology, to clinical medicine
2. Health-related studies in people that follow pre-defined sets of rules

Observational studies involve gathering information about a subject by gathering behavioral or biological data from individuals who are more or less in their natural state, while interventional studies are trials in which research subjects are assigned by an investigator to a treatment (a medicine, physical intervention, or behavioral therapy); outcomes of that treatment are then measured.

Some studies in fibromyalgia involve the use of pharmaceutical medications to evaluate their usefulness in treating FM. This particular type of research is only done in human participants after years of studies involving tissue cultures from both animals and human cells. The resulting compounds are then tested in animals to evaluate their safety in humans.

Once this stage of testing is successfully completed the resultant data is submitted to the US Food and Drug Administration (FDA) for approval to conduct human testing. This is called an Investigational New Drug Application or an IND. If the FDA approves the animal safety data, the next step is a Phase I trial in humans, in which the effect of the medication on the liver, kidneys, and other organs is evaluated in a small group of healthy volunteers. Once the safety of a medication is shown in healthy human volunteers, it may then progress to a Phase II study (sometimes referred to as a “proof of concept” study) to determine whether it has a beneficial effect in patients. If so, large scale trials (Phase III) are generally undertaken in order to gain approval from the FDA for that medication’s use in a particular disorder.

Why volunteer to take part in a clinical trial? How are patients’ rights protected?

 People who participate in clinical trials play a more active role in their own healthcare. They may gain access to new forms of treatment before they are available to the general public. They also help others by contributing to medical research and expanding insight on their disorder.

 A research participant’s rights are protected in two important ways. First, an Institutional Review Board, made up of physicians and laypeople, is charged with examining the study’s design to ensure the patients’ rights are protected and the study does not present undue or unnecessary risk. Second, an individual taking part in a clinical trial in the United States is required to sign an informed consent form, which details the type of study, risks involved, and what may happen to the volunteer during the study. It also informs participants that they have the right to leave the study at any time.

 Are there different types of clinical trials?

 Yes, there are.

• Treatment trials test experimental treatments to determine their effectiveness in treating a medical condition. These trials may involve new types or combinations of medications, new approaches to surgery, or novel forms of radiation therapy. They may also involve physical modalities such as massage, physical therapy, exercise, or electrotherapy.
• Prevention trials look for better ways to prevent disease in people who have never had the disease, or to prevent a disease from recurring. A prevention trial may involve medication, dietary supplements (e.g. vitamins, minerals or other nutriceuticals), vaccines, physical interventions (such as exercise), or lifestyle changes.
• Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition. They may include analysis of bodily fluids (blood tests, urine studies, spinal taps), imaging studies (x-ray, MRI, etc.), or genetic testing.
• Screening trials evaluate the best way to detect certain diseases or health conditions in their earliest stages in order to improve treatment outcomes.

What happens during a clinical trial?

 The scientific process used depends on the kind of study being conducted. (See above.)

 Regardless of the type of clinical trial, participants work with a research team that typically includes doctors, nurses, and sometimes social workers and other healthcare professionals. They explain what is involved in the study in great detail so the participant can make an informed decision whether to participate; they also check the health of the volunteer 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 in order to ensure the long-term safety of the treatment. Some research studies involve more tests and doctor visits than the volunteer would normally have for an illness or condition.

What are the benefits and the risks of volunteering for a research study?

Volunteers benefit from participating in scientific investigations by:

• Playing an active role in their health care.
• Gaining access to new research treatments before they are widely available.
• Obtaining expert medical care at leading health care facilities during the study.
• Helping others by contributing to medical research.

Risks to volunteers participating in clinical trials include:

• Unpleasant, serious, or even life-threatening side effects from experimental treatment.
• Experimental treatment may not be effective for the participants.
• Protocols may require more time and attention than a normal treatment regimen, including trips to the study site, more treatments, hospital stays, or complex dosage requirements.

What should a volunteer consider before participating in a research study?

 The participant should know as much as possible about the clinical trial and feel comfortable asking members of the healthcare team about the care expected while in the study, the cost of the trial, and any other pertinent questions. Following are questions that might be helpful for the volunteer to discuss with the healthcare team.

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

ClinicalTrials.gov and Centerwatch.com are two websites that give information pertinent to this kind of scientific investigation, including specific fibromyalgia trials that are seeking volunteers.