Clinical Trials for FAP
Finding new treatments for rare diseases such as familial amyloid polyneuropathy (FAP) is challenging. One way that patients can help with developing and evaluating new treatments is by participating in clinical trials.
What are clinical trials?
Clinical trials allow researchers and physicians to test new devices, medications, and therapies in healthy volunteers or patients with a particular disease. They test whether the new treatment is better than available ones, or better than no treatment at all. Clinical trials also allow researchers to determine the side effects a new treatment may cause.
Clinical trials are an important part of the process of approval of potential new treatments. Regulatory agencies, such as the U.S. Food and Drug Administration (FDA) in the U.S., and the European Medicines Agency in the E.U., must approve the format of the clinical trial before it can recruit participants. Before applying for approval, researchers usually have to show that a treatment is safe and effective in an animal model of the disease in question. If successful, regulatory agencies may approve the clinical trial design.
What are the advantages of participating in clinical trials?
There are some potential benefits to participating in a clinical trial. The trial usually pays for participants’ medical treatment. This usually includes the tests, scans, and medications that patients may require. Often, the trial also reimburses patients for travel costs to the trial site and time lost at work.
Participating in a clinical trial also gives you the opportunity to receive a new medication that isn’t commercially available yet, and may not be available outside of a clinical trial for years.
What are the disadvantages of participation?
In some clinical trials, not all participants receive the medication that the trial is testing. Participants may not know whether they are receiving the new medication until the trial has concluded. In “double-blind” trials, participants are assigned randomly to receive the new medication or a placebo. Neither the participants nor the physicians administering the medication are aware of which group a patient is in until the end of the trial. This is done to avoid biasing the results of the trial. However, in open-label trials, all participants know which treatment they are receiving.
By participating in a clinical trial, you may be taking a medication for which the side effects are not known. Although researchers do as much studying in the laboratory as they can prior to a clinical trial, it is not possible to predict all side effects that may occur as a result of a new treatment. In rare cases, serious side effects can occur that might require you to drop out of the trial.
Am I eligible for a clinical trial?
If you want to find out whether you are eligible to participate in a clinical trial, talk to your doctor who is managing your FAP treatment. He or she can help you find out where there may be clinical trials near you that will be recruiting patients soon, whether you are eligible to participate, and whether a particular treatment is likely to be effective for you. Your doctor also can discuss the pros and cons of participating in a clinical trial in more detail.
Sometimes, you may need to undergo laboratory tests to confirm your eligibility to participate in a clinical trial. Your doctor may be able to perform these tests or arrange for the tests at your local hospital or clinic.
Here is a current list of clinical trials recruiting, or preparing to recruit, people with FAP.
Last updated: June 11, 2020
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