A clinical trial is a way to study in humans experimental treatments that have shown promise in the laboratory. Clinical trials follow a strict protocol that is carefully designed to safeguard the health of participants as well as answer specific research questions. The purpose of treatment trials is to measure the safety and effectiveness of drugs, devices, or other types of therapies.
Before joining a clinical trial, a participant must quality for the study. Every trial has specific guidelines about who can or cannot participate. These guidelines are called inclusion and exclusion criteria. These might include factors such as age, gender, the type and stage of a disease, and treatment history. These criteria are not used to reject people personally – they are established to ensure that the results of the trial are reliable and to protect the safety of participants.
There are several phases of testing before conclusions about safety and effectiveness can be drawn.
What is required of a participant will vary, based on a specific study’s protocol. Generally speaking, participants are given a treatment that is administered over a specified length of time. It may be the treatment being tested, another existing treatment, or simply a placebo (an inactive pill, liquid or powder with no treatment value.) Participants will have regular appointments with the doctors, nurses and other members of the trial team, at which time they will perform specific tests relevant to what they are measuring, and to monitor the overall health of the participants.
The most obvious benefit is that you may receive access to a new treatment before it is widely available. It is important to remember that clinical trials are experiments ‐ there is no guarantee that the treatment being studied will be effective. This is particularly important to try to help children participating in trials understand. Telling them from the outset that the study drug will be helpful can be difficult to reconcile if the study drug turns out not to be effective or if they develop a concerning side effect.
Participants will also benefit from the frequent monitoring of their health. This close attention could result in better disease management.
Many individuals derive great satisfaction knowing that they are helping others by contributing to the accumulation of knowledge and advancing medical research.
These, and any other risks particular to the study, should be explained by the trial team as part of the informed consent process, which takes place before you are enrolled in the trial.
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. Doctors and nurses involved in the study will explain the purpose, duration, required procedures, and the risks and benefits of participating. All participants will be expected to sign an informed consent document to formally acknowledge that they understand the features of the study. For children, an assent form is used and is written at a level that children can understand and give a degree of agreement based on their level of understanding. This would accompany a consent form for the adult caregiver.
Informed consent is not a contract, and a participant can decide at any point to leave the trial. When withdrawing from the trial, the participant should let the research team know about it, and the reason for leaving the study.
Once a trial ends, researchers take time to analyze the results of the trial. If the results are positive, the manufacturer of the treatment will apply to Health Canada for permission to market the treatment in Canada. This all takes some time. As a trial participant, most often you will be able to continue receiving a treatment while it is undergoing the approval process through an open label extension study.
Participating in a clinical trial can be a rewarding experience. The decision whether or not to participate in a trial is a personal one, and requires careful consideration. It is important that you gather all the facts about the trial, and determine if it is right for you to get involved. Be sure that you understand and are comfortable with the answers to the following questions:
For more tips and information about participating in clinical trials, visit the Center for Information and Study on Clinical Research Participation.
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Dr. Craig Campbell, MD, MSc, FRCPC is a Pediatric Neurologist at Children’s Hospital London Health Sciences Centre, the medical director of the neuromuscular clinic at the Thames Valley Children’s Centre, and Associate Professor at University of Western Ontario. He is the principal investigator for several clinical trials studying new therapies for muscular dystrophy.