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A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

This study is testing a gene therapy called SGT-212 for people with Friedreich's Ataxia (FA). SGT-212 uses a modified virus (AAVhu68) to deliver a specific gene. The main goal of this first-in-human study is to see how safe SGT-212 is and what side effects it might cause over 12 months. You might be able to join if you are between 18 and 40 years old, have a confirmed diagnosis of FA, and your symptoms started before age 25. You also need to meet specific criteria on a Friedreich's Ataxia Rating Scale and agree to rules about using another medication called omaveloxolone (Skyclarys). The study plans to enroll 10 participants, but its current status is unclear.

Study design
This is a Phase 1b, first-in-human study that is open-label, meaning both you and the study team will know you are receiving SGT-212. It will involve about 10 participants.
What's involved
You would receive SGT-212 through injections into your brain (intradentate nucleus) and into your vein (intravenous). You would be part of the study for approximately 5 years.
Compensation
Not stated in the trial record.
Follow-up
Your safety will be monitored for at least 12 months after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07180355

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

Recruiting
PHASE1Ages 18–40InterventionalTreatment
Solid Biosciences Inc.
~10 participants
Updated 2026-08-17 on ClinicalTrials.gov
What's tested:SGT-212

At a glance

Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Measured over Month 12

Conditions

Friedreich's Ataxia (FA)

Explore related trials

Friedreich Ataxia

NCT07180355

Where you'd take part

This study runs at 3 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • The Children's Hospital of Philadelphia (CHOP)

    Philadelphia, Pennsylvaniastudy coordinator listed

    Recruiting

  • The Ohio State University

    Columbus, Ohiostudy coordinator listed

    Recruiting

  • The University of California, Los Angeles (UCLA)

    Los Angeles, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Solid Biosciences Clinical Trials
[email protected]
+1617-337-4680
Email the study team

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Eligibility criteria

Inclusion

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):

Exclusion

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)

What this trial measures

  • Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)Month 12