What are Clinical Trials?

Frequently asked questions about clinical trials

Where can I find out more about clinical trials in neuromuscular diseases?
What is a clinical trial?

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.

Who can participate in a clinical trial?

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.

What are the different phases of a clinical trial?

There are several phases of testing before conclusions about safety and effectiveness can be drawn.

  • Phase I: Tests a treatment in a small group of people to assess safety, determine a safe dosage range, and identify side effects.
  • Phase II: Tests the effectiveness and safety of a treatment on a larger number of patients. These trials are often randomized and blinded to establish how well the treatment works compared to either no treatment at all (placebo) or standard treatment.
  • Phase III: Provides a more thorough understanding of effectiveness, benefits, and possible adverse reactions by testing the treatment with a large number of patients. These trials typically involve hundreds (or even thousands) of patients in many different centres and are carried out over several years.
  • Phase IV: Evaluates the long term risks and benefits of the treatment once it is available on the market.
What happens during a clinical trial?

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.

What are the benefits of participating in the clinical trial?

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.

What are the risks and disadvantages of participating in a clinical trial?
  • Many treatments prove, in clinical trials, to be ineffective, or no more effective than existing treatments. Even if a treatment proves generally effective, you may not directly benefit, for example, if you were given a low dose or a placebo.
  • You may have to discontinue your current treatments or medications while the trial lasts.
  • You may experience side effects to the treatment, and, in rare cases, these may be serious or even life‐threatening. Each study has a safety review person or committee as well as a medical monitor to review and respond to any adverse events or reactions that happen while a person is participating in a study. All these adverse events are documented and serious ones are reported directly and immediately to regulatory agencies.
  • Studies often involve multiple and frequent visits to hospital, which may not be easy or practical. Required procedures, such as blood tests or biopsies, could be painful or uncomfortable.

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.

What is informed consent?

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.

What happens after a trial ends?

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.

Should I participate in a clinical trial?

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:

  1. What is the purpose of the study?
  2. Why do researchers think that the approach being tested may be effective?
  3. Has this treatment been tested before?
  4. Who is sponsoring the study? Who will pay for the treatment and any required procedures?
  5. Who has reviewed and approved the study?
  6. What are the medical credentials and experience of the researchers and other study personnel?
  7. What treatments, tests, or procedures will be required? What is their frequency? Where will they take place?
  8. What are possible long and short‐term risks, side effects, and benefits? How do these compare with existing treatment options?
  9. How are the study results and the safety of the participants being monitored?
  10. Will I be reimbursed for expenses incurred, such as travel?
  11. How long will the study last?
  12. When will the results of the trial be available?
  13. Will the treatment be available after the trial is over?
  14. Will there be follow‐up after the study is over?

For more tips and information about participating in clinical trials, visit the Center for Information and Study on Clinical Research Participation.


Dr. Craig Campbell

Clinical Trials Webinar
Video Presentation

Presenter: Dr. Craig Campbell, MD, MSc, FRCPC
Description: Dr. Craig Campbell provides information about the research process and progress of clinical trials for people with neuromuscular disorders.
Length: 1:02:14

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.

Debra Chiabai

A Parent’s Perspective
Video Presentation

Presenter: Deborah Chiabai
Description: Debra discusses her son, who is affected by Duchenne muscular dystrophy, and her perspective on having him participate in clinical trials.
Length: 10:42



Useful Links

“Being a Co-Adventurer: Clinical Trials involve risks, rights, responsibilities”

An article from Quest magazine

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