On this page, you'll find:
You may find this guide helpful if you are living with mitochondrial disease, caring for someone with mito, or exploring clinical trials for another person.
You can take part in mito research through research projects and clinical trials.Research projects may include surveys, interviews or observational research. Clinical trials test medicines or other interventions to understand whether they are safe and effective.
This page explains clinical trials and how they work. To explore opportunities to take part in surveys, interviews, observational research or other research projects, visit our Participate in Clinical Studies page.
A word from the mito community
"Trials are the most effective way for our community to support innovation that can lead to better health. Of course, we’re hopeful for better health for ourselves, but we are also hopeful for better health for others in the mito community, including those that will join our community in the future and those that are affected by similar conditions."
- Kim and Mia are personally impacted by mito.
What you should know about taking part in a clinical trial
Deciding whether taking part in a clinical trial is right for you is a personal decision. It is important to understand the possible benefits, risks, and committments involved 1,2.
There is no guarantee that you will receive any direct benefit from taking part in a trial.3
Taking part in a clinical trial may not directly benefit you, but it can help build the evidence needed to develop better treatments and care for people living with mito, now and in the future.
For more detail on your rights, safety, and what to expect: Learn more about taking part in clinical trials.4
Trials that are currently recruiting in Australia
These are therapies that are being tested in a clinical trial that has sites in Australia. They are actively recruiting participants.
Explore all emerging therapies
View all emerging therapies, including those that have trials recruiting overseas.
Getting prepared to take part in a clinical trial
Even if there is not currently a clinical trial that is right for you, there are steps you can take to be prepared for future opportunities.
Am I eligible for a clinical trial?
Diagnosis
Most trials require a confirmed genetic diagnosis.
Type
Most trials focus on specific types and/or symptoms of mito.
Age
Some trials are limited to children or adults.
Location
You may need to travel or attend regular visits.
If you are unsure whether you have a genetic diagnosis, or what your diagnosis means, our webpage Understanding your diagnosis may help explain the different parts of a mito diagnosis.
You may not be eligible for every trial, but new ones open regularly. Talk to your specialist for guidance. Join the Mito Registry to be contacted about new, relevant trial opportunities.
How clinical trials work
Clinical trials test new treatments for safety and effectiveness. Each new treatment for mito will be tested in multiple clinical trials. Often there is one clinical trial for each phase.
Each phase is a different step in the development of a drug and tests the new treatment in a different way. Sometimes participants in one phase will be invited to also be a participant in the next phase.
When a new treatment is for a very rare disease, clinical trials can work differently5. For example:
- Many treatments have a combined phase 1 and 2 clinical trial, followed by a phase 3 trial
- Cell and gene therapies will usually not go through all of these phases, particularly those that are customised to a very small number of people.
- The number of participants in each phase may be much lower.
- A treatment can be approved for use after only a Phase 2 clinical trial.
Understanding safety and dosage
- This is the first step to test a new treatment in people.
- Checks safe dose, how to give it, and side effects.
Participants involved:
10-100
people, sometimes people without mito
Duration:
Months
Trials advancing to the next phase:
Source reference 6.
How trials are run in Australia
Most clinical trials for mito that are available in Australia are part of larger international studies. They often have funding from pharmaceutical or biotechnology companies. Local mitochondrial disease specialists and clinical research teams run trials in Australia.
Before a clinical trial can begin in Australia, it must be reviewed by an Australian Human Research Ethics Committee. This committee checks that the trial meets Australian ethical standards and that participants are protected.
This includes making sure people are given clear information, understand the possible risks and benefits, take part voluntarily, can withdraw at any time, and their privacy is respected.
What happens after a clinical trial ends?
Clinical trials are an important step in developing new treatments, but they are not usually the final step.
After a trial ends, researchers analyse the results to understand how safe and effective the treatment was. If the results are positive, the treatment may be submitted to health authorities for review.7
In Australia, the Therapeutic Goods Administration (TGA) reviews whether a treatment is safe and fit for purpose. If so, it can be sold in Australia.8
For a medicine to become publicly funded through Pharmaceutical Benefits Scheme (PBS), it goes through a health technology assessment process. PBS medicines are subsidised so they are affordable.9
This process can take time. Not every clinical trial leads to an approved treatment, but every study helps build understanding of mito and guide future research, care and treatment development.
Need help understanding your options?
Contact Mito Foundation support services.
Contact us
Call Mito Foundation's Helpline for more information or assistance on
Please note the Helpline is available Monday to Friday, excluding NSW public holidays.
Immediate support
If you or someone you know needs emergency assistance, please call emergency services on
Mental health support
This page shares information and resources to help you find mental health support that works for you.
Helpful resources
Powering progress: The future of clinical trials for mito
VideoMito Foundation and Research Australia hosted this event to discuss increasing clinical trials for mitochondrial disease (mito) in Australia. Speakers included people with lived experience and leaders in the field. Together, they brought wide-ranging life experiences and professional insights. They discussed:
- the clinical trial process
- why some trials not coming to Australia and what can we do to change this
- how we can work together to shape the future of clinical trials.
The event was recorded on 16 April 2025.
Learn more about our plan to increase clinical trials for mito in Australia.
