Patients interested in participating in research

If you are interested in participating in a clinical trial, we are here to help. Below, we will cover some of the basics and provide you with links to current
clinical trials recruiting patients.

What is a clinical trial?
A clinical trial is a study that observes or treats patients in order to develop or discover new treatments or medications.
Almost all medical advances are thanks to the dedicated volunteers who participate in clinical trials. 
These carefully planned-out studies allow scientists to accurately evaluate medications by studying the effects on healthy people
and those with the condition the drug is designed to target.

Why are clinical trials so structured?
The structure in a clinical trial assures the safety of the participants through close contact and monitoring.
As well, it ensures clinical trial results are accurate, well-documented, reliable and can be trusted.

Below, Health Canada defines the four phases of  clinical trials, their purpose and how each phase helps researchers answer specific questions.

  • Phase I – These trials test an experimental drug on a small group of people for the first time. The purpose is to:
    • assess the drug’s safety
    • find out what a safe range would be for dosage
    • identify side effects
  • Phase II – The drug is given to a larger group of people (usually 100 or more) to:
    • obtain preliminary data on the effectiveness of the drug for a particular disease or condition
    • further assess the drug’s safety
    • determine the best dose
  • Phase III – The drug is given to even larger groups of people (usually 1,000 or more) to:
    • confirm its effectiveness
    • monitor side effects
    • compare it to commonly used treatments
    • collect information that will allow the drug to be used safely on the market
  • Phase IV – These trials are done after the drug is approved and is on the market. They gather information on things like the best way to use a drug, and the long-term benefits and risks.

Can anyone participate in a clinical trial?
Every clinical trial has specific protocols or guidelines it must follow — this determines how the trial will be run including who may and may not participate.  
Sometimes trials require patients with a specific condition, or a particular stage of a disease and sometimes, healthy volunteers are needed too. 
The reasons that allow you to volunteer for a trial are called “inclusion criteria” and the reasons that disallow it are called “exclusion criteria”.

What happens when the trial is over?
Just because a trial is over doesn’t mean the end of all communication between you and the research team.
Once treatment is completed and the study is closed, patients should feel free to ask the trial staff what treatment they were getting.
Staff are allowed to share this information as soon as it is available and they can also share the trial results when they become available. 
If you request to see your data from a trial, researchers may have to wait until trial results are made public before sharing the information with you.
Click clinical trials, to learn more.


Trials Currently Open for Recruitment

MMPOWER-3: Current PMM clinical trial available in Canada

MMPOWER-3 is a Phase 3 double-blind, placebo-controlled trial enrolling 202 patients with PMM at sites in North America and Europe. During the first six months of the trial, patients are randomized 1:1 to receive once daily subcutaneous injections of either elamipretide or placebo.

All trial sites in Canada are currently recruiting patients ages 16-80 with genetically confirmed primary mitochondrial disease who have myopathic symptoms of muscle weakness, exercise intolerance and fatigue. For a full listing of study centers including contact information and inclusion and exclusion criteria, please click study centresor visit

A summary report for Stealth Biotherapeutics MMPOWER-3 trial webseminar, hosted on On October 24th,, is available at: PMM Community Webinar

Patients and Families Researching Open Trials 

Patients and families can actively search for recruiting studies that they may be able to participate in or learn about new interventions/treatments that are being considered. To find a study, click: ClinicalTrials

If you are a patient or family member who wishes to learn more about the website, click: HelpforPatients