Friedreich Ataxia
Plain English clinical trial information, updated daily from ClinicalTrials.gov and public government registries. Not medical advice.
How these figures are counted
- Trials on record is every trial tagged to this condition at any stage, except those withdrawn before starting or terminated early. Most are completed rather than ongoing, so this is a measure of research history, not of current activity. It is also smaller than the sitewide "trials tracked" figure, which does include withdrawn and terminated trials.
- Recruiting now counts only trials whose registry status is "Recruiting". Trials listed as not yet recruiting or enrolling by invitation are excluded, so this understates how many may open to new participants soon.
- Australian sites counts trials counted under "Recruiting now" that list at least one Australian site. A site being listed does not mean it is currently enrolling, and Australian trials registered only on ANZCTR are not counted yet, so this understates local availability.
- Phase 3 trials counts trials counted under "Recruiting now" that are at Phase 3.
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Understanding Friedreich Ataxia
Friedreich Ataxia (sometimes written as Friedreich's Ataxia, or FA) is a genetic condition that affects the nervous system and the heart. It causes the nerve cells that control movement and coordination to gradually break down over time, which leads to difficulties with balance, walking, and coordinating movements. It is caused by a change in a gene called FXN, and a person needs to inherit a changed copy of this gene from both parents to develop the condition. FA is not contagious and is not caused by anything a person or their family did.
In Australia, FA is considered a rare condition, affecting roughly 1 in 50,000 people. It most commonly begins to show symptoms in childhood or adolescence, typically between the ages of 5 and 15, though it can appear in adulthood for some people. Both males and females can be affected. Because it is rare, many people with FA are supported through specialist neurology centres, and organisations such as Ataxia Australia can connect patients and families with others in similar situations and with relevant support services.
At present, there is no cure for FA, but there are a range of supports and therapies that aim to help manage symptoms and maintain quality of life. These generally involve a team of health professionals and may include physiotherapy to support movement and strength, occupational therapy to assist with daily tasks, and monitoring and management of heart-related symptoms by a cardiologist. A GP or specialist will guide decisions about care based on each person's individual circumstances. Research into FA is ongoing, and it is worth discussing with your treating team what options may be available or appropriate for you.
This overview is an AI generated summary written from public medical sources and checked on a sample basis. It is general information, not medical advice or a diagnosis. Always speak with your doctor about your situation. How we use AI
Can you access treatment for Friedreich Ataxia in Australia right now? Here's what's publicly known.
- No PBS-subsidised medicines are currently mapped for Friedreich Ataxia on Voxsanity. Your doctor or pharmacist can advise on what may be available.
- 2 clinical trials currently recruiting at Australian sites for Friedreich Ataxia.
Information on this site does not constitute medical advice. Data is sourced from publicly available government registries. Always consult your doctor before making any health decisions. Trial status should be verified directly with the trial site.
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Become a founding memberResearch pipeline
Where trials for Friedreich Ataxia currently sit in the development pipeline. The funnel shape shows how few drugs reach later phases in general; the counts are this condition's actual trial numbers.
Most active research sponsors
Organisations with the most recruiting trials for Friedreich Ataxia.
Where trials are running
Clinical trial sites for Friedreich Ataxia by country. Australia is highlighted.
Clinical trials for Friedreich Ataxia
Showing 18 of 18 trials
The plain English eligibility text on each trial below is an AI generated summary of the official criteria, checked on a sample basis. How we use AI
🇦🇺 NCT02255435 · Biogen Phase 2 · 5 countries
**Who might be able to join this trial:** - You have been diagnosed with Friedreich's ataxia, confirmed through genetic testing - Your disease severity falls within a specific range as measured by a scoring system called the modified FARS (confirm with trial site for your exact score) - You are between 16 and 40 years...
🇦🇺 NCT04577352 · PTC Therapeutics Phase 3 · 9 countries
**Who might be able to join this trial:** - You have a confirmed diagnosis of Friedreich's ataxia caused by a specific type of genetic change (a GAA repeat expansion in both copies of the frataxin gene), proven by genetic testing. - Your disease severity score (called mFARS) falls between 20 and 70 at the start of the...
🇦🇺 NCT06772870 · Design Therapeutics, Inc. Phase 1 · 1 country
Who might be able to join this trial: - You must be between 18 and 45 years old at the time you sign the consent form. - You must be generally healthy, based on a medical check-up that includes your health history, a physical exam, blood/lab tests, and a heart tracing (ECG). - You must agree to avoid intense or strenuo...
🇦🇺 NCT06874010 · Design Therapeutics, Inc. Phase 2 · 1 country
**Who might be able to join this trial:** - You must be between 18 and 65 years old at the time of signing the consent form - You must have a diagnosis of Friedreich's Ataxia (FA) confirmed by genetic testing, specifically with a particular type of genetic change (called homozygous GAA repeat expansions) in the frataxi...
🇦🇺 NCT06953583 · Biogen Phase 3 · 16 countries
**Who might be able to join this trial:** - You have been diagnosed with Friedreich's Ataxia (FA) confirmed by genetic testing, showing the specific gene changes associated with this condition. - You are showing symptoms of Friedreich's Ataxia, as confirmed by a doctor's assessment. - Children aged 7 to under 16 must a...
NCT02316314 · Weill Medical College of Cornell University Not Applicable · 1 country
**For people with Friedreich's Ataxia (FRDA):** Who might be able to join this trial: - You are male or female, between 12 and 50 years old - You are willing and able to give your consent to participate (teenagers will need to give their own agreement, and a parent must also give consent) - You have a confirmed diagno...
NCT02497534 · University of Florida Not Applicable · 1 country
**Who might be able to join this trial:** - You have been diagnosed with Friedreich's ataxia through genetic testing or a related lab test, OR you are a healthy person with no known nerve or muscle conditions - You are between 6 and 70 years old (including those ages) - You are able to take part in an exercise test tha...
NCT05302271 · Weill Medical College of Cornell University Phase 1 · 1 country
**Who might be able to join this trial:** - People aged 12 to 50 years, both male and female. - People who are willing and able to give informed consent (for those under 18, a parent or guardian would typically be involved — confirm with trial site). - People who have a confirmed diagnosis of Friedreich's ataxia (FA),...
NCT05943002 · German Center for Neurodegenerative Diseases (DZNE) Not Applicable · 3 countries
Who might be able to join this trial: - You have been diagnosed with Friedreich's Ataxia (FA) confirmed through genetic testing - Your ataxia (loss of coordination) is rated at 30 points or below on a standard medical scale used to measure ataxia severity (called the SARA scale) - You have access to a smartphone or tab...
NCT06447025 · Larimar Therapeutics, Inc. Phase 2 · 1 country
**Who might be able to join this trial:** - You have Friedreich's ataxia (FRDA) and either have never been in a previous CTI-1601 study, or you completed one without experiencing serious side effects, severe reactions, or withdrawing early. - Your HbA1c (a blood sugar level test) is 7.0% or below. - You are able to gi...
NCT06623890 · Biogen Not Applicable · 7 countries
**Who might be able to join this trial:** - You have been diagnosed with Friedreich's Ataxia (FA), confirmed through genetic testing. - You are 16 years old or older when you start taking the medication omaveloxolone. - *(If you have never taken omaveloxolone before)* You are starting omaveloxolone as an approved trea...
NCT06628687 · Biogen Not Applicable · 1 country
**Who might be able to join this trial:** - You took the medication omaveloxolone (for Friedreich's ataxia) at any point during pregnancy — including up to 12 days before becoming pregnant — or at any point while breastfeeding (until your baby is 1 year old or you stop breastfeeding, whichever happens first) **Who may...
NCT06692296 · IRCCS Eugenio Medea Not Applicable · 1 country
Who might be able to join this trial: - You are 14 years old or older - You weigh less than 150 kg (about 330 lbs) - You have been diagnosed with Friedreich's Ataxia through genetic testing - Your walking ability falls within a specific middle range on a standard ataxia assessment scale called SARA (not too mildly or s...
NCT06865482 · Lexeo Therapeutics Not Applicable · 8 countries
**Who might be able to join this trial:** - You are male or female and are 6 years old or older at the time of signing the consent forms - You have been diagnosed with Friedreich's Ataxia (FA), confirmed by both your symptoms and a genetic test showing the specific gene changes linked to FA, and your symptoms started w...
NCT07013292 · Centre Hospitalier Universitaire de Nice Not Applicable · 1 country
Who might be able to join this trial: - You are aged 16 years or older - You have been diagnosed with Friedreich's ataxia, confirmed through genetic testing - You have been receiving omaveloxolone treatment between February 2024 and May 2025, and have been on this treatment for at least 6 months Who may not be able to...
NCT07095062 · IRCCS Eugenio Medea Not Applicable · 1 country
**Who might be able to join this trial:** - You are between 8 and 60 years old - You have been diagnosed with Friedreich's ataxia through genetic testing, confirmed by having a specific type of gene change (called a GAA expansion) in both copies of the frataxin gene - Your ataxia symptoms are below a certain level of s...
NCT07508631 · Centre Hospitalier Universitaire de Nice Not Applicable · 1 country
Who might be able to join this trial: - You are between 18 and 70 years old. - You have been diagnosed with Friedreich's Ataxia confirmed through genetic testing. - You are currently being seen and followed at the Neurogenetics Competence Centre at CHU Nice (a specialist centre in Nice, France). - You have had a periph...
NCT07721025 · Lexeo Therapeutics Phase 2 · 1 country
Who might be able to join this trial: - People who are male or female and are at least 6 years old at the time of signing the consent forms - People who have been diagnosed with Friedreich's ataxia (FA), confirmed by both their symptoms and a genetic test showing a specific change in the frataxin gene - People whose FA...
Advocacy and support in Australia
Organisations that can offer information, support, or connection for Friedreich Ataxia. Where more than one is listed, they serve different needs or regions, so choose whichever fits your situation.
fara Australia (the Friedreich Ataxia Research Association) is the dedicated national body for Friedreich ataxia, active since 2003, funding research and running patient clinics and support services in Melbourne and Brisbane.
This is a starting point, not medical advice or an endorsement. Contact details change; search the organisation name to find its current website.