Recruiting
Phase 1

SGT-212

Sponsor:

Solid Biosciences Inc.

Code:

NCT07180355

Conditions

Friedreich's Ataxia (FA)

Eligibility Criteria

Sex: All

Age: 18 - 40

Healthy Volunteers: Not accepted

Interventions

SGT-212

Study Details

Brief summary:

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.

Conditions

Friedreich's Ataxia (FA)

Study ID

NCT07180355

Start date

Oct 22, 2025

Status verified date

Aug, 2026

Completion date

Feb 29, 2032

Anticipated

Primary completion date

Mar 21, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 40

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

10 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1 (Non-Ambulatory)

Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.

experimental: Cohort 2 (Ambulatory)

Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.

experimental: Cohort 3 (Ambulatory and Non-Ambulatory)

Participants will receive bilateral IDN followed by systemic IV infusion.

Interventions

SGT-212

Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)

Primary outcome measure

  • Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Month 12 ]

Central Contacts and Locations

Central contacts

Solid Biosciences Clinical Trials

+1617-337-4680clinicaltrials@solidbio.com

Locations

The University of California, Los Angeles (UCLA)

Recruiting

Los Angeles, California, United States, 90095

Contacts

Dillan Hayward

dhayward@mednet.ucla.edu

Principal Investigator:

Susan Perlman, MA

The Ohio State University

Recruiting

Columbus, Ohio, United States, 43210

Contacts

OSU Gene Therapy Research Team

OSUgenetherapyresearch@osumc.edu

Principal Investigator:

Yasushi Kisanuki, MD

The Children's Hospital of Philadelphia (CHOP)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

McKenzie Wells

267-426-9608WELLSM@chop.edu

Principal Investigator:

David Lynch, MD

More Information

Sponsor

Solid Biosciences Inc.

Last update posted

Aug 17, 2026

Last verified

Aug, 2026

Keywords

  • SGT-212
  • Gene Therapy
  • Frataxin
  • Repeat Expansion
  • Neuromuscular
  • Cardiac
  • Ataxia
  • Movement Disorder
  • Rare disease
  • AAV
  • AAVhu68

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Solid Biosciences Inc. on 2026-08-17.