Treatment Studies

What You Should Know

What You Should Know

… before you volunteer for a treatment study

What You Should KnowPeople who volunteer for treatment studies (often referred to as clinical trials) make it possible for new or existing medications, and novel nondrug therapies to gain approval for the treatment of fibromyalgia. It is the only way that people with this life-impacting condition will eventually receive effective treatment. Yet, participating in a clinical trial is obviously not for everyone. Before giving serious thought to any trial or study, you should weight the benefits versus the disadvantages of being a clinical trial participant.

The following is a brief explanation of a few key factors that you need to know about being a study participant. You may find postings about various clinical trials on the United States government’s website www.clinicaltrials.gov, but unfortunately this is not a user-friendly site. In fact many of the explanations below are to help you understand what is found on this site and the terminology used in the postings for fibromyalgia clinical trials. A free comprehensive guide on deciding whether you should enter a clinical trial is available in the ECRI Institute’s Patient Reference Guide.

  1. What is a clinical trial?
  2. What is typical trial protocol?
  3. Understanding the criteria for participating
  4. Is getting involved in a clinical trial the right action for you?
  5. Safety Issues
  6. Do I need a physician referral?
  7. Does it cost money to participate in a trial?
  8. What questions should I ask the research coordinator?
  9. Can I drop out after the study has begun?
  10. Can I continue to take the medication after the trial is over?

1. What is a clinical trial?

Prior to testing any drug or therapy in humans, its safety and effectiveness for treating a symptom or medical condition are evaluated in lab animals (usually rats or mice). If the Food and Drug Administration (FDA) passes the test results in lab animals for safety, the manufacturer of the drug or therapy may proceed to the next step, which is small-scale testing in humans (see Study Phases for more details). Studies carried out in humans, rather than in lab animals or a test tube, are called clinical studies. When the project is designed to test the treatment effectiveness of a medication or form of therapy in people, it is referred to as a clinical trial.

Drug Trials
With the exception of a few treatment approaches in the first Phase of testing, a portion of the patients in a clinical trial will be given a placebo. A placebo is simply a sugar pill that looks exactly like the medication being tested. Patients who receive the placebo during a trial are commonly referred to as the control group. The response of the patient group (those who receive the experimental drug) is compared to the control group (patients who receive the placebo) so that the effectiveness of the study medication can be determined. If the most effective dose for a drug is not yet known, more than one patient group at varying doses may be compared to a placebo group. Also, if the experimental drug produces some drowsiness or a bitter after-taste, then the research team may put something in the placebo to mimic these side effects. These measures help to prevent patients from figuring out if they are taking the placebo or experimental drug (i.e., patients and the evaluating physicians must be blinded, which means neither knows who is taking the active treatment and who is taking the placebo).

Nondrug Therapy Trials
Not all clinical trials involve drugs; many are designed to test nondrug therapies or medical devices that may lead to symptom relief. Take for example, transcranial magnetic stimulation (TMS), a device that emits a tiny electrical current into the brain with the use of a magnetic field that enables it to pass through the skull to alter neurochemical function in the brain. TMS is painless. Based on its use in other medical conditions, theories have been developed for how it might work to help fibromyalgia patients. However, the machine must be placed on the proper frequency, power and other settings for it to work. (A study evaluating TMS in fibromyalgia will soon be recruiting patients, and we will post it at that time.)

How do researchers do clinical trials with nondrug therapies, such as TMS? It is very similar to a trial involving a study medication. One group of patients will receive TMS treatments, in which the machine is place on the settings believed to be most effective for relieving pain. A control group of patients will also receive TMS treatments, but the machine will not be placed on the settings believed to produce any physiological effects. This control group does not actually receive a placebo (such as a sugar pill), but rather they receive sham treatments. Regardless of the nondrug therapy being tested, the sham treatment is designed to be as similar as possible to the active treatment (the therapy being tested).

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2. What is typical trial protocol?

A protocol is a set of procedures that explains every step of the clinical trial and answers all of the “what ifs” before the study begins. A common clinical trial design involves the following steps:

  • initial evaluation of all participants
  • two week period in which everyone receives a placebo (referred to as the washout part of the trial because all drugs are essentially washed out of the system)
  • patients are randomly divided into the control group (receives the placebo or sham treatment) or the patient group (receives the active drug or active treatment) but neither the patients or the person evaluating them knows which group they have been assigned to (this represents a randomized, placebo-controlled and blinded trial)
  • patients receive the study drug/therapy or placebo/sham for a set number of weeks or months, during which time their symptoms and other test measures are recorded
  • patients may go through a second wash out period of two weeks so that the researchers can determine if abruptly stopping the drug causes problems for patients
  • during the study important information about symptoms, function and overall impression of well-being is collected so that the data from the patient group can be compared with the control group

Throughout the study period, the protocol must be followed. If a participant deviates significantly from the protocol, they cannot remain in the study because any information collected from their continued participation will be shrouded in uncertainty. So, if you chose to become involved in a clinical trial, you should anticipate that there will be many rules and procedures to follow, and you should never hesitate to ask questions about them. Detailed protocols are required to enhance patient safety and produce quality research and make certain that no one’s time is wasted on a study that does not meet the standards for being published in a medical journal.

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3. Understanding the criteria for participating

Age, gender, lifestyle, medical conditions other than fibromyalgia, and a number of other factors may influence the response to the experimental drug or therapy. This means researchers must be selective when recruiting patients so that all of these variables are similar for the “patient” and “control” groups. In fact, this is just one of many reasons why a clinical trial’s entrance criteria are very lengthy; the research team must reduce the variability in the data that is not caused by the test drug itself.

All clinical trials have inclusion and exclusion criteria to clearly define who is a good candidate for a given study. The inclusion criteria for fibromyalgia will specify that you must meet the American College of Rheumatology (ACR) criteria, and a few other factors may be required as well. The list for exclusion criteria is often lengthy and it would be a good idea to review it with your physician to determine if you qualify. For the most part, patients with other painful medical conditions are excluded from fibromyalgia treatment trials because it would be impossible to determine which condition the drug or therapy might be treating. This means that patients with autoimmune diseases such as lupus, rheumatoid arthritis, and others are excluded.

Patients receiving any compensation for their fibromyalgia, such as long-term disability or Social Security disability, are often excluded. Why? If you are disabled by your fibromyalgia, chances are you have tried every type of medication and form of therapy, and nothing has worked. This means that your odds of responding to the new investigational treatment are reduced. Including you (and other similarly disabled patients) would make a drug look less effective than it truly is for the general fibromyalgia patient population. If the drug or therapy does get FDA approval for fibromyalgia, you will then have an opportunity to try it and hopefully you will respond positively to it.

On the other hand, some studies are actually looking for people who have tried many therapies with little improvement and there is a strong chance that they are work-disabled. If a treatment intervention is a bit more invasive than taking a pill, such as the vagus nerve stimulator trial that involves minor surgery, the study investigators may want to recruit patients in which commonly prescribed medications have failed (i.e., refractory to drug therapies). After all, most treating physicians will first try to help patients with medications before they consider surgery, even if it is minor. However, if such a therapy shows promise in patients who are refractory to the medications, then further studies on this therapy would likely be opened up to the general population of fibromyalgia patients.

Impending lawsuits (including workman’s comp and long-term disability cases), divorce, or any other ongoing “battle” in a person’s life will automatically exclude them from participating in a clinical trial. Legal disputes are terribly stressful and their outcome could be devastating to a person. This could lead to significant alterations in the person’s study data that would have absolutely nothing to do with the treatment being tested, which is why people involved in legal proceedings are automatically excluded from clinical trials.

If you look through the clinical trial criteria for the studies posted on this site and discover that you do not qualify, keep in mind that this has nothing to do with you as a person. The lengthy criteria are set in place to increase the safety of the participants and to optimize the quality of the trial. And if you meet the criteria for a study, you need to ask yourself if this is the right thing for you at this particular time in your life.

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4. Is getting involved in a clinical trial the right action for you?

There are pros and cons of becoming involved in a clinical trial, and you should take a close look at each. You also may want to get the opinion of your family, close friends, and your doctor prior to making a final decision. Before you even inquire about a specific trial in your area, you might consider the following benefits and risks of becoming a participant:

Benefits

  • be able to try a therapy before it becomes available
  • interact with knowledgeable healthcare providers in the field
  • receive free medical tests, exams, and evaluations, but patients are usually required to stay with their primary care provider during the trial period
  • gain information about your health that you might not otherwise receive, especially if any novel diagnostic tests are being employed (however this information may not be provided to you until after the study is complete)
  • feel good about your role in advancing the science of fibromyalgia and helping other patients eventually receive better treatments
  • the educational experience obtained from being a part of a research project

Risks

  • you may get a placebo or sham treatment for the study period
  • even if you do get the active treatment or drug, there is no guarantee that it will relieve your symptoms
  • you may experience adverse events that could cause your health to seriously decline instead of get better
  • it could involve more of your time than you initially anticipated (it’s important to know how much time the study will take and to be honest with yourself about the amount of time you have available for the study)

In addition to weighing the benefits versus risks of participating in a clinical trial, you should also consider factors pertaining to your own personal situation. For example, will your family support your decision to participate or will they begrudge you for the time you are spending on the trial? If you work outside the home, do you have a supervisor or co-workers who understand your fibro flares (in case you have a bad reaction)? Can you cope with the uncertainty of being placed on a placebo? Or, are your current medications not that big of a factor in your life?

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5. Safety Issues

Ideas for clinical trials are first conceived in the lab and tested on lab animals before they are tried in humans. Still, if the drug is newly developed (not already used in other countries), there might be risks, but they are minimized by the mandate that all clinical trials be approved by an Institutional Review Board (IRB). The National Institutes of Health (NIH) must also approve research it funds, and the FDA must approve any trial for an investigational new drug or device. So there may be multiple layers for review of a project to help ensure patient safety.

The IRB is made up of scientists, professionals in other fields, as well as at least one patient representative. An IRB assesses the ethics and validity of the trial design and the risks it poses to patients. If the risks are deemed to great, the IRB will reject the proposal or it might require changes in the protocol to improve patient safety. Many IRBs have an independent patient advisor as one of its board members, so that patients in a given trial can contact them for advice if the patient perceives a problem. During the “informed consent” interview, ask whether a patient advisor is a member of the IRB.

When an FDA approval is being sought, an agency representative also oversees each Phase of each clinical trial. The FDA’s job is to protect the safety of people while it also set standards for showing that a drug is effective for a specific condition, such as fibromyalgia.

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6. Do I need a physician referral?

No, but it would be good idea to ask your physician to review the information on Fibromyalgia Network’s website and referral links. Your physician can advise you about whether you are likely to meet the study criteria, and you can discuss the pros and cons of volunteering. Also, most treatment trials require that you continue to see your primary care physician for regular check-ups during the trial period. So, even if you visit the research physician every month, you still need to keep tabs with your personal physician.

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7. Does it cost money to participate in a trial?

Usually, the answer is NO. All trials have a budget, and within that budget is money to compensate patients for their time and expenses incurred (e.g., parking, gasoline, etc.). However, if you chose to enter a study that is a significant distance away (necessitating that you stay overnight in a hotel), then your hotel expenses are probably not covered.

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8. What questions should I ask the research coordinator?

  • What is the purpose of this study and what evidence is there that this treatment might benefit people with fibromyalgia?
  • What Phase is the study? Who is funding it?
  • Will some patients be given a placebo, and if so, what are my chances of getting a placebo or sham treatment? Will there be more than one group receiving the active drug at different doses? How many treatment groups altogether will there be and will they all consist of fibromyalgia patients?
  • Has this treatment already been FDA-approved? If not, how many fibromyalgia patients has it been tested on in the United States and has it been approved in other countries? If yes, what medical conditions has the FDA-approved this treatment for and when was it first approved?
  • How long will the trial last and how many visits to your center will it require? What other time commitments will this involve?
  • What are the possible side effects?
  • How might this therapy affect my ability to work, clean house, or tend to my family?
  • How does this therapy differ from what I am currently using to manage my fibromyalgia?
  • What role does my primary care doctor (or the doctor who is currently tending to your fibromyalgia) play while I am involved in this trial?
  • Will I be provided a contact person to call if I have problems, and how does this work (i.e., is this a message machine or someone’s cell phone number)?
  • If I have an adverse reaction to the therapy and need additional medical help, who will take care of me and who will pay for this assistance?
  • What tests will be done? After the trial is over, will I be able to find out about the results of my tests?
  • Will I receive the results of the trial once it is over? Will I ever find out if I was in the treatment group or the placebo? If I received the placebo or sham, will I be given an opportunity to try the active treatment after the study is over?
  • If I don’t get the placebo and I respond to the therapy, will you work with my doctor to teach him/her how to prescribe this therapy for me (assuming it is already FDA approved for another condition)?
  • Will I be reimbursed for my expenses, and if so, when?

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9. Can I drop out after the study has begun?

Yes. You are under no obligation to remain in the trial for the full duration. Although you signed many forms during the informed consent interview, this process is for the purposes of protecting you (i.e., making you aware of all of the potential risks of the study and what to do should anything happen, such as the development of side effects). If you decide to withdraw from the trial, notify the study coordinator and please specify your reasons for discontinuing. If it is a personal matter that is unrelated to the study treatment, please say so. This information is collected as part of the study’s data, and the most common reasons for leaving are included in the clinical trial results.

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10. Can I continue to take the medication after the trial is over?

If the test medication is already FDA-approved for another condition, it is possible that your primary care physician could prescribe this medication for you in an off-label manner. You may ask if the trial center might assist your physician with determining dose to prescribe and what, if anything, needs to be monitored for safety reasons. If the drug is not yet FDA approved, your physician will not be able to prescribe it for you. Sometimes, patients who volunteer for testing a study drug or therapy may be “invited” to take this treatment on a longer-termed basis to determine if the drug remains effective over 6-12 months. Otherwise, if you had a favorable response to the study medication and it is not yet on the market, hopefully the drug will receive prompt FDA-approval.

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