Since my journey began as an Irritable Bowel Syndrome and Crohn’s Disease patient I have watched clinical trials related to my illnesses closely because I knew that additional treatment options would be available that were newer and different than current treatment ones. I’ve always encouraged patients in my community to enroll in clinical trials because it might allow them to gain access to new treatment options now versus waiting years for the trial to end, then be approved and then marketed.
What is a Clinical Trial?
A clinical trial refers to studies in which people participate as patients or volunteers. There are different terms used to describe clinical trials such as: clinical studies, clinical research, studies, research, trials, protocols.
The idea for a clinical trial study often originates in the laboratory. After researchers test new therapies or procedures in the laboratory and in animal studies, the most promising experimental treatments are moved into clinical trials, which are conducted in phases. During a trial, more information is gained about an experimental treatment, its risks, and its effectiveness.
Strict rules for clinical studies have been put in place by the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Clinical Trial Volunteers have a Bill of Rights that are adhered to. Some clinical studies involve promising new treatments that may directly benefit participants. Others do not directly benefit participants, but may help scientists learn better ways to help people.
Some people participate in clinical trials because none of the standard (approved) treatment options have worked, or they are unable to tolerate certain side effects of current treatments. Clinical trials provide another option when standard therapy has failed. Others participate in trials because they want to contribute to the advancement of medical knowledge.
The four phases for a clinical trial are:
- Phase 1: Screening for safety involving animal studies and testing on a few patients
- Phase 2: Establishing the test protocol with a small group of patients, ie: quantity of medication, how often, inclusion guidelines
- Phase 3: Final testing with a much larger, perhaps multi-centered study with many researchers nationwide
- Phase 4: Postapproval studies are conducted after a treatment is approved and marketed to learn how the treatment is affecting the general population
For more information about understanding Clinical Trials, please refer to Clinical Trials: What Parents Need to Know (FDA).
My Mother-in-Law’s experience in a Clinical Trial
My Mother-in-law is 79 years old and suffers from a rare neuromuscular illness. The illness slow progresses with only about 20,000 people who have it in the US. Since it is so rare there are no clinical trials specifically for the illness; however, a recent Phase 3 clinical trial, for another illness and a medication already approved for marketing, had a clinical trial which looked at whether the same medication could slow the progression of the neuromuscular illness. My Mother-in-law decided to participate in the clinical trial. The treatments consisted of an infusion of unknown medication every 6-weeks for 12 months. She did not know whether she was receiving the actual medication (double-blinded clinical study) or a placebo. The 12 weeks were a rather onerous process for everyone due to travel time and the side effects. Following the treatment, my Mother-in-law was given access to the actual medication on a 6-week cycle for another 12-months for free.
So why did she participate in the clinical trial?
The primary reason was that my Mother-in-law wanted to advance research for the illness. She also wanted access to the medication once her clinical trial was completed because she hoped that it would actually slow down the progression of her illness. She didn’t feel any different after the clinical trial even when she knew she was getting access to the real medication. It gave her, and our family, some hope.
My friend’s daughter and their experiences in a Clinical Trial
My friend’s daughter is 7 years old and suffers from rare non-cancerous tumours in her lungs. Without medication or surgery the tumours begin to grow and restrict breathing and usually result in serious pneumonia. Treatment is difficult and adhoc. The Children’s hospital where her daughter is seen regularly made her aware of a Phase 2 clinical trial for a cancer drug that might shrink the tumours and prevent their relapse. Making a decision for yourself to enroll in a clinical trial is hard enough. Making it for your child is even more difficult and my friend took months to decide. In the end, she decided to enroll her daughter. Her daughter cooperated much of the time, though at times it was difficult to explain it all to her. My friend shared the time commitment at the hospital with her husband when it was treatment time. They took turns going for the follow-up MRI scans that were required after each treatment. This went on for 12-months. There was no offer of access to the medication after the completion of the clinical trial.
So why did my friend enroll her daughter in the clinical trial?
My friend felt that the difficult and adhoc treatment for the tumours were not a long term solution for her daughter. She knew that the cancer medication would definitely shrink the tumours so her daughter would be immediately better off in the clinical trial. The risk of the clinical trial was the side effects and whether there would be long term relief after the clinical trial. It was not a double-blinded clinical trial so they knew they were getting the actual medication in each treatment.
Clinical Trials for Irritable Bowel Syndrome or Crohn’s Disease
Deciding on whether to enroll in a clinical trial for yourself will likely have the same thoughts as to why my Mother-in-law or friend’s daughter’s decided to enroll — is it right for you? For me, gaining access to a new treatment option would be the primary reason for myself to enroll in one.
We are often contacted by researchers and Clinical research centers to help recruit patients for Clinical Trials. We list them on our Clinical Trials page.
One particular Clinical Trial that we are very interested in is an oral investigational drug called filgotinib for people with moderate to severe Crohn’s Disease. Researchers want to see if it reduces the signs and symptoms of Crohn’s by lowering the immune response that causes inflammation in the digestive system. You can learn more about the trial at www.myibdstudy.com.
We believe that the more patients who are willing to participate in Clinical Trials, the better the research and treatment options are for patients.
FDA: Clinical Research Versus Medical Treatment
Informed Consent: A Guide to the Risks and Benefits of Volunteering for Clinical Trials
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