Phase 3 Friedreich Ataxia Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
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 also score between 10 and 34 on a specific balance and stability test at the start of the study.
- For later parts of the study, you must have already completed the first part without stopping early, and the study doctor must feel it is safe for you to continue.
Who may not be able to join:
- Your long-term blood sugar level (a test called HbA1c) is too high — above 11% — which may suggest poorly controlled diabetes.
- A specific heart-related protein in your blood (called BNP) is above a certain level at your initial screening, which could indicate heart stress (confirm with trial site).
- Your heart's pumping strength (called ejection fraction) is below 40%, based on a heart scan done at your first visit.
- You have a significant heart condition, except for mild to moderate heart muscle disease related to Friedreich's Ataxia.
Important: Always verify eligibility with the trial site directly before applying.
Please note: Other eligibility requirements not listed here may also apply, so it is essential to speak with the trial team for full details.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Director, Biogen
Phone: 1-877-223-3576
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Part 1: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 52; Part 2A: Change From Baseline in USS Subscale E of mFARS at Week 52; Part 2B: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE); Part 2B: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) at Weeks 52 and Week 104; Part 2B: Change From Baseline in Height at Weeks 52 and Week 104; Part 2B: Change From Baseline in Weight at Weeks 52 and We...
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.