Twenty-seven years ago, when Defeat Duchenne Canada (formerly Jesse’s Journey) began our quest to find a cure, there was very little research and no clinical trials happening as they related to Duchenne and its specific causes and symptoms.
In recent years, this has changed significantly and there are many companies and organizations investing billions of dollars into research and clinical trials. This is creating a lot of hope for our families but can also feel overwhelming. Defeat Duchenne Canada has developed this section to help you learn more about clinical trials generally as well as those currently underway.
Scientists and doctors use the systematic process of a clinical trial to determine if a potential medical, surgical, or behavioral intervention is safe and effective. If there are already known treatments for a condition, a clinical trial will compare the potential treatment to current treatments. In this case, they are trying to determine if the new treatment is more effective or has fewer side effects than the standard treatment. There are several different stages in a clinical trial and during each step, important data is collected to be analyzed. The entire process must be carried out carefully and the data must be analyzed properly to be able to determine if a treatment will be helpful to the group of patients it is intended to be used for.
What are the phases of a clinical trial?
Once preclinical studies are complete in animal models, and there is sufficient information demonstrating proof of concept, safety, toxicology, and how the drug is made, the sponsoring organization can apply for an investigational new drug (IND) submission with regulatory authorities. Clinical trials follow a specific study plan called a protocol which is developed by the study researcher or manufacturer. It is up to the regulatory authorities to review the protocols and all supporting data to determine if it is safe to proceed to in-human trials. Once approved, the researcher or manufacturer can move to Phase 1 studies.
Who can join a clinical trial?
Every clinical trial has rules called inclusion and exclusion criteria that are used to determine who will be able to participate and who will not be able to.
Inclusion criteria outline the clinical and demographic characteristics a patient must have to be part of the trial. This may include things such as:
It is important all patients enrolled in the study have similar characteristics to help explain the results observed when a treatment or intervention is given.
Exclusion criteria define the characteristics that would prevent a patient from being part of that clinical trial. It may include medications you are taking, the fact you are already participating in a clinical trial study and the willingness or ability to undergo different types of testing that will be asked of you. Exclusion criteria are important as they keep you safe and ensure you can complete the tests.
Why is it important to participate in a clinical trial? Why would individuals and families want to consider participating in a clinical trial?
Being involved in a clinical trial is not a decision that can be made lightly. Often, it involves sacrifices from the family and the patient. Extra visits to the clinic, extra time for travelling and time away from work are just some of the examples of additional burdens a clinical trial can have on a family.
For many parents and patients, one of the primary motivators is the possibility that they may have access to a new therapy that will help improve their condition. Another important reason to participate in a clinical trial is that even if the therapeutic or intervention is not found to be effective, this information is important and helps the scientific community to redirect their resources towards other potential treatments.