Without research, there are no new treatments

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500 – 5,000. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls.

It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

Clinical trials provide early access to treatments, contribute to medical knowledge about a condition, help guide future research, and have the potential to impact how people with the same condition are treated in the future.

Clinical trials can give patients access to the latest medicines and procedures. Studies show that patients who participate in clinical trials have outcomes at least as useful, if not better than the general patient population. In some cases, clinical trials are a last resort — there are no other treatments, or other interventions have not worked or have stopped working. However, many times, they involve addition or adjustment to a standard treatment plan that may provide patients with a better quality of life.

There are clinical trials taking place to find better treatments and improve the quality of life for people affected.

Clinical Trials Simplified (CTS) can help find a clinical trial that matches your condition. > Read more about CTS <


What is a clinical trial?

Clinical trials are research studies that involve testing or studying treatment in people to see if it is safe and effective. Each phase of a clinical trial follows a protocol and has a specific goal. The information gathered in a trial is used to build knowledge about the new treatment and support the subsequent phases of the research process.

When a patient participates in a clinical trial, he is followed closely by the clinical trial doctor and team and has to regularly visit the medical center or hospital where the trial is taking place. It is important to note that not all clinical trials look into testing novel therapies. Some trials aim at testing novel diagnostic methods or supporting patients with specific needs. Other trials aim at testing a drug, previously approved by the health authorities to treat a particular condition, to treat a different condition.

Clinical trial phases

Phase 1

To determine the safety and tolerability of treatment. Usually open-label (both the researchers and participants know what the participant is taking as a medication).

Phase 2

To look more closely at safety and efficacy and how the treatment affects the body. Usually includes control and non-control groups are often double-blind.

Phase 3

The last step before treatment is submitted to Health Canada. Done to confirm treatment’s efficacy, monitor side effects, and compare it commonly used treatments. Can last up to five years or more and are randomized, double-blind and placebo-controlled trials.

Phase 4

Once Health Canada has approved treatment, this study is often done to identify further information about risks, benefits, side effects, and optimal use.

What is a control group?

A control group is a standard against which experimental treatments are evaluated. In Phase II and III clinical trials, one group of patients will be given an experimental treatment, while the control group is given either a standard treatment for the illness or a placebo. A control group is part of the criteria of evidence-based medicine.

What is a placebo?

A placebo is an ‘inert’ pill, liquid, or powder with no active ingredients. In phase II and III trials, experimental treatments are compared with placebos to assess the experimental treatment’s efficacy and safety.

Why participate in a clinical trial?

When you participate in a clinical trial, you contribute to the development of new treatments and the advancement of medical research, and in doing so, you help other patients. Moreover, the new investigational product tested in the clinical trial might turn out to be effective.

Deciding to participate in a clinical trial

The decision to participate in a clinical trial is a personal and important decision. Before making such a decision, you must assess the potential benefits as well as the disadvantages and risks involved.


Some clarifications

  • Clinical trial participants are not human guinea pigs.
  • Investigational medicines are researched extensively in a laboratory before they are ready for clinical trials with human volunteers. Although researchers cannot guarantee outcomes, a patient’s safety is always the top priority.
  • Before participating, you are given in-depth information about the study, a process called informed consent. The process of informed consent continues throughout the study, and the participant is free to withdraw from the clinical trial at any time.
  • Government and international regulations are also in place to make sure that research involving people is done according to strict scientific and ethical guidelines. A panel reviews clinical trial protocols at the hospital, clinic or university before the trial begins. The panel called a Research Ethics Board (REB) or Institutional Review Board (IRB), includes doctors, scientists and members of the general public. Health Canada must approve all clinical trials that assess the safety and efficacy of an investigational molecule (a medicine not approved for use in Canada).
  • Your doctor will not necessarily tell you if a clinical trial could benefit you.
  • While your doctor may be able to direct you to relevant trials, they may not be aware of all of the options available to you. Health Canada authorizes approximately 900 clinical trials every year, so there are numerous trials taking place in a particular disease area at any given time.
  • You should always talk to your doctor or healthcare professional who will help you make the right choice for you.


The approval of new drugs is the responsibility of Health Canada.


For more information about clinical trials in Canada

To join the team ECS: 1 (888) 982-2782  –  info@clinicaltrialssimplified.com  –  Facebook page  –  Website

Or consult the Clinical Trials website: Clinicaltrials.gov

New section on La Force’s website > here <



La Force thanks MS CanadaRoche CanadaCanadian Institutes of Health Research et Clinical Trials Simplified for this website content.






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