Who may be eligible for the A Study of SGT-212 Gene Therapy in Friedreich's Ataxia trial?

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.

ClinicalTrials.gov record NCT07180355

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

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)

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.

Age18 years old to 40 years old

SexAll

LocationUnited States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a Phase 1b, first-in-human, open-label dose-finding study of SGT-212 in people with Friedreich's Ataxia. All participants receive SGT-212 delivered by dual intradentate nucleus (IDN) infusion and by intravenous (IV) infusion. Participants are followed in the study for about five years.

What it is studying. The study is testing the safety and tolerability of an SGT-212 gene therapy regimen delivered by IDN and IV infusion in people with FA. It will track treatment-emergent adverse events, serious adverse events and deaths, and will collect data on immune responses and blood biomarkers.

What the researchers hope to learn. To evaluate the safety and tolerability of SGT-212 at studied doses; to identify and describe treatment-emergent adverse events and serious adverse events; to measure immune responses and changes in blood biomarkers over time; and to inform dose selection for future studies.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: SGT-212

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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):
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
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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

All participants receive SGT-212 by bilateral intradentate nucleus (IDN) surgical infusion followed by a systemic intravenous (IV) infusion. Study procedures include invasive steps such as the IDN surgical infusion and planned cardiac procedures (endomyocardial biopsy or cardiac catheterization) and repeated brain and heart MRIs with contrast; there will also be regular safety visits with physical exams, lab and immune monitoring, and questionnaires (including C-SSRS). Participants are followed for safety and outcome assessments over about five years, with key safety checks reported at months 12, 18 and 60. Participants must follow the study's omaveloxolone rules as a condition of participation.

How the study is structured. An interventional, Phase 1b, open-label (non-masked), non-randomized, dose-finding trial with parallel cohorts. All participants receive the investigational drug SGT-212; cohorts include ambulatory and non-ambulatory participants and each participant undergoes an IDN infusion followed by an IV infusion.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Oct 22, 2025
Primary completion: Mar 21, 2028 (estimated)
Study completion: Feb 29, 2032 (estimated)
Target enrollment: 10 participants (estimated)
Last updated on registry: Sep 29, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Solid Biosciences Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Solid Biosciences Clinical Trials 📞 +1617-337-4680 ✉ clinicaltrials@solidbio.com

Participating sites (3)

The University of California, Los Angeles (UCLA)Recruiting
Los Angeles, California, 90095 · United States
- Dillan Hayward · dhayward@mednet.ucla.edu
- Susan Perlman, MA
The Ohio State UniversityRecruiting
Columbus, Ohio, 43210 · United States
- OSU Gene Therapy Research Team · OSUgenetherapyresearch@osumc.edu
- Yasushi Kisanuki, MD
The Children's Hospital of Philadelphia (CHOP)Recruiting
Philadelphia, Pennsylvania, 19104 · United States
- McKenzie Wells · 267-426-9608 · WELLSM@chop.edu
- David Lynch, MD

Terms used on this page · explanations by MedicalUpdates.ai

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose-finding: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
stable dose: A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
Sponsor: The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
investigational: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
contraindication: A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
biopsy: Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
MRI: A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
5 half-lives: The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
prior to Screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
corticosteroids: Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
Exclusion criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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