SGT-212 gene therapy for Friedreich's ataxia
Official title: A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)
Run by Solid Biosciences Inc. · NCT07180355 · Data updated 2026-11-12
This phase 1b test will try a gene therapy called SGT-212, given into the dentate nucleus and by IV, to see if it is safe and tolerated in people with Friedreich's ataxia.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 40 Years |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Solid Biosciences Inc. Industry-sponsored |
| Locations | 3 sites (Los Angeles, California and 2 other locations) |
| Status | Recruiting |
| Started | October 22, 2025 |
| Main results expected | March 21, 2028 |
| Study ends | February 29, 2032 |
| Registry record updated | November 6, 2026 |
| Trial ID | NCT07180355 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are people with a clinical and genetic diagnosis of FA who had symptom onset by age 25, have a FARS Functional Disability Staging Score of 1–5, and agree to the study's omaveloxolone-use rules and long-term follow-up.
✕Probably not a fit
Patients with very advanced disease, significant medical contraindications (for example severe liver disease), inability to comply with omaveloxolone restrictions, or who cannot commit to long-term follow-up are less likely to benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a phase 1b, first-in-human, open-label, dose-finding trial of SGT-212 in people with genetically confirmed Friedreich's ataxia. The investigational product is delivered using a dual approach—direct injection into the dentate nucleus combined with an intravenous infusion—and all participants will receive SGT-212. The primary focus is safety and tolerability with dose escalation to identify a recommended dose, and participants will be followed for approximately five years for adverse events and exploratory clinical and biomarker outcomes. Eligible participants must meet clinical and genetic criteria and follow specific rules regarding concurrent or recent use of omaveloxolone.
Why it matters
Potential benefit: If successful, SGT-212 could raise frataxin levels and potentially slow or stop neurological decline in people with Friedreich's ataxia.
How similar studies have gone: This is the first-in-human trial of SGT-212; although AAV-based gene therapies have produced successes in other neuromuscular disorders, gene replacement for Friedreich's ataxia has not yet been proven in humans.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Has history of FA symptom onset ≤25 years of age * Has a clinical and genetic diagnosis of FA * Has a staging score of ≥1 but \<6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score * Is willing to agree to the following rules for use of omaveloxolone (Skyclarys): 1. For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study 2. For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team. Exclusion Criteria: * Antibodies against adeno-associated virus serotype 9 (AAV9) * Has a modified FARS (mFARS) score \<20 * Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m\^2 * Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization * Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures) * Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c \>9% * Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening * Has received gene therapy at any time * Has contraindications to receiving corticosteroids * Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212 * Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD) * Other Inclusion/Exclusion criteria to be applied as per protocol.
Where it's running
-
The University of California, Los Angeles (UCLA)
Los Angeles, California, United States Recruiting -
The Ohio State University
Columbus, Ohio, United States Recruiting -
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States Recruiting
Study contacts
- Study coordinator: Solid Biosciences Clinical Trials
- Email: clinicaltrials@solidbio.com
- Phone: +1617-337-4680
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.