Tests SGT-212 gene therapy in people with Friedreich's ataxia
Tests SGT-212 gene therapy for safety in adults with Friedreich's ataxia.
Plain-language title generated from the registry record
Registry title: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Key facts
- Where
- Columbus, Los Angeles, and Philadelphia, United StatesSee the 3 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 40 years old?
Source: NCT07180355, who can take part
Where in United States
- The Ohio State UniversityColumbus, United StatesTaking people
- The University of California, Los Angeles (UCLA)Los Angeles, United StatesTaking people
- The Children's Hospital of Philadelphia (CHOP)Philadelphia, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Solid Biosciences Inc.
- Trial code
- NCT07180355
- Record updated
- 6 November 2026
- Original description
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.Read full original descriptionShow less
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)
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.
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):; 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: Antibodies against adeno-associated virus serotype 9 (AAV9); 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 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 any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD); Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c >9%; Has a modified FARS (mFARS) score <20; Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m\^2; Other Inclusion/Exclusion criteria to be applied as per protocol.