NCT07180355RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
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)
Source: ClinicalTrials.gov · Last updated 29 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 22 Aug 2025
- Start date
- 22 Oct 2025
- Primary completion date
- 21 Mar 2028
- Lead sponsor
- Solid Biosciences Inc.
- Diseases
- Friedreich ataxia
- NCT number
- NCT07180355
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 10
- From 22 Oct 2025 to the primary completion date, 21 Mar 2028
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- 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)
Lab values
- Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c \>9%
Other
- 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.
Who the study is looking for
- 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.
Age in the registry: 18 years to 40 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Ohio State University Coordinator of this site? Confirm its status | Columbus | United States | Recruiting |
| The University of California, Los Angeles (UCLA) Coordinator of this site? Confirm its status | Los Angeles | United States | Recruiting |
| The Children's Hospital of Philadelphia (CHOP) Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.