Clinical Trials for Mitochondrial Disease


MitoCanada is committed to curating an active list of clinical trials important to patients and mitochondrial disease research. 

MIT-E Study, Canada

Study to evaluate the safety and efficacy of vatiquinone (PTC743), to reduce seizures in patients who have mitochondrial disease with hard-to-treat epilepsy.
Enrolment: Now Recruiting!
Study Site: Virtual and US-based

STRIDE Study, Canada

Study to evaluate the efficacy and safety of 24 weeks treatment with REN001 in patients with primary mitochondrial myopathy.
Enrolment: Now Recruiting!
Study Site: Alberta and British Columbia

Clinical Trials for Kids

Join superheroes Hazel (aka Flare) and Sam (aka Blitz) in this delightful animation from Rich Toons explains the process and potential benefits of participating in pediatric clinical trials.

SURF1 Discovery Platform

Accelerate drug development discovery, research and knowledge about SURF1 Leigh’s in joining AllStripes SURF1 research database. Learn more about participating, gene therapy developments and natural history studies taking place.
Enrolment: Open Now!
Study Sites: Global; Virtual

G.R.I.T. Study 

Join the first Global Rare Disease Insights through Technology study. This is an app-based clinical trial aims to learn better ways to manage virtual care for rare disease metabolic patients.
Enrolment: Open Now!
Study Site: Global

Dietary Patterns & Supplement Use, Survey

Dr. Jane Shearer’s laboratory at the University of Calgary is conducting a survey examining dietary patterns and supplement use in the mitochondrial disease population.
Participation: Survey open now

Dichloroacetate (DCA) PDC Deficiency Study, USA

Phase 3 trial of DCA in young children with PDC
Enrolment: Recruiting now
Study Site: Multi-USA site; Canadian recruits welcome

MOVE-FA Study, Canada

Study to assess the efficacy and safety of Vatiquinone for the treatment of participants with Friedrich Ataxia
Enrolment: Recruiting soon
Study Site: Quebec

FA-COMS Study, Canada

Multi-centre natural history study to provide a framework for facilitating therapeutic interventions
Enrolment: Recruiting soon
Study Site: Quebec and Ontario

International Mito COVID-19 Study

To understand the response to, and improve treatment of, COVID-19 for those with mitochondrial disease is critical. The Highly Specialised Service for Rare Mito. Disorders in London is creating a database of mito patients who contract COVID-19.

ClinicTrial.Gov | Open Trials  

Patients and families can search here to learn more about recruiting studies and learn more about new interventions and treatments being considered. 

Clinical Trials Ontario

The Clinical Trials Ontario website hosts a clinical trials finder, which search all trial databases in Canada. Search for mitochondrial trials taking place here at home.

What is a clinical trial?

Clinical trials are research studies that  look at new ways to prevent, detect, or treat disease. They are the primary means of determining if a new treatment is safe and effective and are designed to evaluate medicines, surgeries or behavioral interventions.

Why would I participate in a clinical trial?

People participate in clinical trials for a variety of reasons. Some participate to receive the newest treatment. Others are looking for additional care from staff of the clinical trial. And, some may simply want to help others. Whatever the motivation, clinical trials offer hope for many people and an opportunity to help discover better treatments for the future.

Is it safe to participate in a clinical trial?

Generally speaking, it is safe to participate in a clinical trial. The structure in a clinical trial aims to protect the safety of the participants through close contact and monitoring. It also ensures results are accurate, well-documented, reliable and trusted-worthy.

It is important to note that all medical tests, treatments and procedures have risks associated with them. Risks may be higher in clinical trials because there are more unknowns, especially in phases I and II before the treatment has been studied in large groups of people. Some new treatments may cause serious side effects or be uncomfortable and may not work or be any better than standard treatments already available.