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Sarcoidosis

Clinical Trials FAQs


1. Do all drug studies have to have a placebo group?
Not all drug studies have to have a placebo group. Sometimes an active drug is compared to standard of care therapy, which is usually corticosteroids, such as prednisone in the case of sarcoidosis. Sometimes a study drug is given alone without placebo or any no comparison medication. These are called, “open label drug trials.” Such studies still have an important place in studying potentially useful medications. These open label drug trials are often done as “pilot studies” with a small number of patients just to determine if there is any response to therapy. Large meaningful drug studies are usually very expensive (costing millions of dollars). These small pilot studies are not adequate to prove that the study medications will work. However, they can give a clue to whether or not they could work and therefore allow for a relatively inexpensive way to get an idea if a large drug trial is worthwhile.

2. Does all clinical research involve drugs?
No. Clinical research can involve studying the results of blood tests, breathing tests, X-rays, and other clinical information collected on patients, surveying patients to determine what is important to them, and obtain physician and other health care provider opinions on how patients with a disease should be cared for.

3. Isn’t it unethical to do drug studies with a placebo medication because it should not help my medical problem?
When a treatment for a disease has been clearly established, then it is often unethical to study a group of patients who are not receiving therapy. Often this problem can be overcome by adding the study drug or a placebo to an existing drug regimen. For example, one could study a drug plus prednisone for sarcoidosis in one group to a placebo plus prednisone in the comparison group. In this way, both groups would be receiving prednisone, the standard therapy for sarcoidosis.

In the case of sarcoidosis, it must be kept in mind that NO drug, including corticosteroids, is FDA-approved for treatment. In addition, corticosteroids such as prednisone have many potential side effects. Researchers are in general not satisfied with steroid therapy as the standard therapy for sarcoidosis for this reason. Therefore, in certain situations, it may be ethical to have a sarcoidosis placebo group when studying certain forms of sarcoidosis. It is recommended that you ask this to a researcher who wants you to participate in a sarcoidosis study where the only medications that you could possible receive is a placebo. It is not that this could not be done in certain situations, but the researcher should clearly explain why it is ethical in this case and you need to understand and accept this explanation before you agree to participate.

4. Is it right for my doctor to conduct research with me? Isn’t my doctor experimenting on me in this case?
Yes, the doctor is experimenting on you. However, there are a lot of important points to make here. First, as mentioned above, no medication concerning corticosteroids is FDA-approved for the treatment of sarcoidosis, and corticosteroids are not ideal treatment because of their potential for side effects. Therefore, as long as the risks of the study drug are reasonable and the potential for benefit to you is reasonable, it is ethical to try such therapy as long as you understand the potential risks and benefits and agree to participate. Second, since there is no FDA-approved treatment of sarcoidosis, ANY therapy that your doctor has prescribed for you, including corticosteroids, is technically experimental. Third, most patients selected for clinical drug studies have been considered to have failed standard therapy such as corticosteroids for their sarcoidosis. Therefore, the doctor is searching for a therapy that will be effective. Fourth, although I cannot speak for all doctors or researchers, almost all of them sincerely want their sarcoidosis patients to live better lives and wipe out this disease. They are not satisfied with the current therapies available. That is the motivation for most of them wanting to conduct research.

5. Why can’t sarcoidosis drug research be focused on animals?
Presently, there is no animal model for sarcoidosis. That means we have been unable to cause sarcoidosis to occur in animals, so we cannot study the disease in them. Also, even if sarcoidosis could be studied in animals, the results of animal drug studies have to be confirmed in humans because a) some drugs work for diseases in animals but not in humans; and b) some drugs have serious side effects in humans that are not present in animals.

6. How can I go about participating in clinical research concerning sarcoidosis?
Please feel free to contact Dr. Marc Judson at judsonm@mail.amc.edu for more information on clinical trials. Or, to find out about sarcoidosis studies being conducted in the United States is by searching a clinical study database. One of the largest ones is www.clinicaltrials.gov. This website lists a large percentage of all sarcoidosis trials currently underway in the U.S.