Powering progress - Mito Foundation

There’s real hope on the horizon for people living with mitochondrial disease (mito). Unprecedented progress is being made in developing new treatments for mito. More than 60 potential treatments are in development, and over 20 clinical trials have already started around the world.

This is an exciting time for people with mito, their families, health professionals, and researchers around the world. But right now, Australians are at risk of missing out.

We need to act now to make sure Australians can take part in clinical trials that could lead to life-changing treatments. Our 10-year action plan sets out the steps to make this happen.

Mito Foundation and Research Australia hosted an event to discuss increasing clinical trials for mito in Australia. Watch, listen to, or read about the event.

Read our 10-year action plan

Year 1 progress report

This work was only possible because of the generosity of our supporters. Help us power progress and increase clinical trials by donating today.

The vision

Australia is a global leader in clinical trials for mito, with an efficient, collaborative, and sustainable ecosystem that meets the needs of the mito community, clinicians, researchers, industry and other funders.

A trial-ready mito community

Accessible participation: Individuals with mito have equitable access to clinical trials through effective recruitment, transparent communication, as well as practical and psychosocial support. Clinical trials are considered part of a range of management options for mito.

Empowered involvement: Mito community members are knowledgeable about clinical trials. The design, implementation, and evaluation of clinical trials involves the mito community. Because of this involvement, there is low attrition of patients in Australian clinical trials.

Improved outcomes: Clinical trials address
real-world patient needs, leading to therapies that improve quality of life and long-term care.

Capable and resourced clinical research teams

Infrastructure and support: Clinician researchers have access to administrative support, clinical infrastructure, and can access pooled resources to enable them to take part in and lead clinical trials.

Collaborative networks: Robust mito clinical trial networks to share expertise, referrals, resources, and innovative methodologies.

Research capacity: Positions that include dedicated time for clinical research and career development opportunities for clinician researchers to ensure long-term sustainability.

Engaged industry and other funders

Streamlined processes: Simplified and harmonised regulatory and ethical requirements to accelerate trial setup and safe implementation.

Strategic partnerships: Strong collaborations with patient organisations and local institutions to enhance recruitment and trial success.

Sustainable investment: A commercially favourable environment with robust infrastructure, comprehensive data, and a highly skilled workforce to attract and retain industry sponsors. Growing investments by philanthropic and Government funders to complement research by industry.

I have mito. What can I do?

Get clear on your diagnosis
Most clinical trials require people to have a confirmed genetic diagnosis before they can participate in them. Learn more about genetic testing and mito.
Join the Mito Registry
Stay informed about opportunities to take part in clinical studies relevant to you. By joining the registry you can contribute towards possible treatments for mito.
Start the conversation with your health care team
You can tell your mito specialist that you are interested in clinical trials.

Priority areas and actions

Building clinical research capacity

Actions to ensure that clinician researchers are motivated, empowered, and resourced to lead clinical trials

Supporting the mito community to participate in
clinical trials

Actions to create a clinical trial-ready mito community, and to ensure participants are well supported during and after trials

Icon of 3 people with a lightbulb overhead

Strengthening collaboration

Actions to enhance connections and partnerships between the mito community, professional organisations, industry and regulators

Contributing work

Enhancing data infrastructure, including patient registries to support participant recruitment
Continuing work beyond industry-led trials, such as investigator-led trials, outcome measures, and translational research
Preparing for the future through proactive work towards registration and reimbursement in Australia
Advocating for long-term improvements through policy change

What is Mito Foundation doing to implement the action plan?

We've already started powering progress. Here are some of the actions already in the works.

In May 2025, we'll open applications for our Clinical Fellowship funding. We particularly encourage applications relating to clinical research, including clinical trials.
We'll be dedicating funds to building clinical research capacity in Australia, balancing this with continued funding for other types of research. We'll share information about this funding in late 2025.
We've set a goal of increasing the numbers in the Mito Registry from 500 to 1000 in the next 5 years. We look forward to working with mito specialists and the mito community to make this happen.

This is a 10-year action plan that relies on collaboration. We can't deliver this plan alone.

We welcome discussions regarding specific opportunities for collaboration with clinician researchers and industry partners. Get in touch to work with us to make clinical trials a real option for the mito community in Australia.

Contact us

Phone: 1300 977 180 (option 2)

Email: info@mito.org.au

Read our 10-year action plan

Year 1 progress report

Mito Foundation and Research Australia hosted this event to discuss increasing clinical trials for mito in Australia. Access the audio file and event summary.

Frequently asked questions

How can I find out about clinical trials for mito?

You can explore current clinical trials and emerging therapies for mito using our Therapy Finder.

Use the Therapy Finder to:

Explore emerging therapies.
See which types of mito they may target.
Find clinical trials currently recruiting in Australia or overseas.

Visit our guide to taking part in clinical trials resource to find information on:

the difference between clinical trials and other research projects
how researchers assess whether someone may be eligible
what screening and participation may involve
questions you may want to ask.

The guide cannot tell you whether a clinical trial is right for you. It can help you understand the process, prepare questions and have an informed conversation with your healthcare team.

More to read:

ClinicalTrials.gov and the WHO International Clinical Trials Registry Platform (ICTRP) list trials by condition, location, and status.
The United Mitochondrial Disease Foundation (UMDF) website lists trials in the USA. Some of these may also have sites in Australia.
Your mito specialist may also be aware of ongoing trials that might be suitable for you, particularly for trials they are involved in.

How can I find out if I’m eligible for a specific clinical trial?

Each clinical trial has its own eligibility criteria. These may include age, diagnosis, symptoms, medical history and location.

Our taking part in clinical trials guide explains common eligiblity considerations and questions to ask. Therapy pages include contact details for the relevant trial coordinator, where available.

You can contact the research team or clinical trial coordinator for guidance on whether you may be eligible for a specific trial. Your treating specialist can also help determine if a trial is suitable for you.

Is funding support available for participants who need to travel for clinical trials?

I have mito. How can I prepare to take part in a clinical trial?

Even when there is no suitable trial currently available, you can prepare for future opportunities by:

Get clear on your diagnosis. Most clinical trials require people to have a confirmed genetic diagnosis before they can participate in them. Learn more about genetic testing and mito.
Join the Mito Registry. Stay informed about opportunities to take part in clinical studies relevant to you. By joining the registry, you can contribute towards possible treatments for mito.
Start the conversation with your health care team. You can tell your mito specialist that you are interested in clinical trials.

What can be done to improve government support for increasing clinical trials for mito in Australia?

Governments play an important role in clinical trials. To help increase clinical trials for mito in Australia, governments particularly need to:

Increase targeted funding for mito-specific trials, including low-cost therapeutics like repurposed drugs and dietary supplements.
Encourage state government investment in clinical trial units and precision therapy manufacturing facilities.
Streamline trial approvals to make the process more efficient and accessible.

Mito Foundation is actively advocating for more research funding and greater government support for mito trials. We often do this in collaboration with others. Learn more about how we work to drive change.

Mito community members can play a role by sharing their experiences, participating in government consultations, supporting advocacy campaigns, and raising awareness with decision-makers. Adding your voice helps demonstrate the real need for trials and better support. We regularly offer opportunities for the community to get involved in advocacy and policy work. You can sign up for our newsletter to receive updates about these. Community members can also share their story through the Mito Stories Project to help drive change across multiple areas.

What are the rules in Australia for compassionate use of treatments after clinical trials?

In Australia, the Therapeutic Goods Administration (TGA) regulates compassionate use through programs like the Special Access Scheme and the Authorised Prescriber Scheme. These programs allow doctors to prescribe unapproved treatments under specific conditions, ensuring safety and ethical standards are met. Open-label extension studies may also allow participants continued access to treatments after a trial ends. Read more about these schemes in the Australian clinical trial handbook.

I am a health professional or researcher interested in being part of a mito clinical trial. What can I do?

If you are a researcher interested in planned clinical trials, the best first step is to connect with the clinical trial leads or networks coordinating mito trials. You can also talk to Mito Foundation, as we work to build a stronger network to run clinical trials for mito in Australia. We encourage interested researchers to reach out to us for more information and connection to relevant networks.

There's a lot of work to do.

Help power progress and support our work by donating today.

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Work that has informed the development of the action plan

Consultations with stakeholders, including:

Mito community members
Clinicians and researchers
Industry representatives

Read the Stakeholder consultation summary

Thank you to everyone who contributed to this work.

A literature review to understand the barriers and enablers to conducting clinical trials for mito in Australia.

Read the Literature review

An analysis of Mito Foundation’s Horizon Scan of recent mito clinical trials.

Powering progress:

A plan to increase clinical trials for mitochondrial disease in Australia