Clinical trials and patient registries

Researchers around the world are working to find new therapies that will slow the progression of Duchenne muscular dystrophy, reduce the severity of the disease and improve the lives of those affected. A number of promising new therapeutic approaches have been developed and are going through clinical trials to explore the safety and effectiveness of the new treatments in humans.

Clinical Trials

What is a clinical trial?

A clinical trial is a way to study new treatments, therapies and devices in humans that have shown promise in the laboratory. Clinical trials are carefully designed to answer specific research questions and follow strict protocols to ensure the safety of the participants. The purpose of the trial is to understand the effectiveness of the therapy and to ensure its safety.

Who conducts clinical trials?

Every clinical trial is led by a principal investigator who is often a medical physician. Often, the studies are co-led by a team of investigators comprised of doctors, nurses, social workers and other healthcare professionals.

Who can participate?

All clinical studies have pre-determined criteria outlining who can participate. These standards are called eligibility criteria and are listed in the protocol. Some research studies seek participants who have the illnesses or conditions that will be studied, other studies are looking for healthy participants, and some studies are limited to a predetermined group of people who are asked by researchers to enroll.

For more information about participating in clinical trials, visit Muscular Dystrophy Canada.

How do I find a clinical trial?

Now more than ever, researchers are discovering new therapies that have the potential to improve the lives of children with Duchenne muscular dystrophy. Canadian clinical investigators are taking an active role in this research. To find out what trials are taking place in Duchenne, visit ClinicalTrials.gov, a searchable registry of publicly and privately supported clinical studies.

Patient Registries

What is a patient registry?

A patient registry is a database containing important medical information about individuals affected by a specific disease, condition or disorder. Registries collect this information to improve the understanding of the impact of the disease, clinical care and disease management, and to accelerate the development of new therapies through clinical trials. Respected registries take every precaution in safeguarding data. Before joining a registry, confirm their security and data-sharing policies.

For more information about patient registries, visit Muscular Dystrophy Canada.

There are two patient registries that are available to Canadians:

Canadian Neuromuscular Disease Registry – The Canadian Neuromuscular Disease Registry (CNDR) is a Canada-wide registry of people diagnosed with a neuromuscular disease. It collects important medical information from patients across the country to improve the understanding of neuromuscular disease and accelerates the development of new therapies.

Duchenne Connect – Duchenne Connect was founded by a group of thought leaders in the Duchenne community that recognized the need to connect and serve the needs of the Duchenne community. The site hosts a registry that collects patient data helping to connect registrants to relevant clinical trials.

To find out more about global patient registries, visit TREAT-NMD.

 

To see a video of Dr. Jean Mah, Associate Professor of Pediatric Neurology at Alberta Children’s Hospital speak on emerging therapies for DMD, click here.