Phase 1 Rare Disease 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:
- Your first symptoms of Friedreich's Ataxia (FA) started when you were 25 years old or younger
- You have been diagnosed with FA through both a clinical assessment and genetic testing
- Your level of disability from FA falls within a specific range on a standard FA rating scale (a score of 1 or higher but less than 6 on the FARS Functional Disability Staging Score)
- If you are currently taking omaveloxolone (Skyclarys), you have been on the same dose for at least 12 weeks, plan to stay on that dose throughout the study, and are willing to stop taking it if your doctor sees signs of liver problems during the study
- If you are not currently taking omaveloxolone, at least 12 weeks have passed since your last dose, and you agree not to take it again for 18 months after receiving the study treatment
Who may not be able to join:
- You have certain antibodies in your blood that react to a virus called AAV9 (used to deliver the gene therapy)
- Your FA symptom score on a specific assessment (called mFARS) is below 20
- Your body weight is 25 kg (about 55 lbs) or less, or your body mass index (BMI) is 33 or higher
- You have a medical reason why you cannot safely have a heart tissue sample taken (endomyocardial biopsy) or a heart catheter procedure
- You are unable to have an MRI scan of your heart or brain with contrast dye — for example, due to an allergy to the dye used, having a non-MRI-compatible pacemaker or defibrillator, or physical limitations such as joint contractures
- You have poorly controlled diabetes, shown by a blood sugar marker (HbA1c) above 9%
- You have taken part in another clinical trial or received an experimental treatment within the past 3 months (or longer, depending on the treatment)
- You have ever received gene therapy of any kind at any time in the past
- You have a medical reason why you cannot take steroid medications (corticosteroids)
- You have a medical reason why you cannot safely undergo the surgical procedure needed to receive the study treatment
- You have a known heart condition that is not caused by FA, such as blocked heart arteries (coronary artery disease)
- There may be additional criteria not listed here (confirm with trial site)
Important: Always verify eligibility with the trial site directly before applying.
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
Phone: +1617-337-4680
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
Primary endpoints
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
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.