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Gene Therapy Trial Report

Summary

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


NCTID NCT07180355 (View at clinicaltrials.gov)
Description
Development Status πŸ”„ Active
Indication πŸ”„ Friedreich's Ataxia
Disease Ontology Term πŸ”„ DOID:0111218
Compound Name πŸ”„ SGT-212
Sponsor Solid Biosciences Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 10 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant πŸ”„ FXN
Therapy Type πŸ”„ Gene transfer
Therapy Route πŸ”„ In-vivo
Mechanism of Action πŸ”„ Functional gene replacement
Route of Administration πŸ”„ Intradentate nucleus, intravenous
Drug Product Type πŸ”„ Viral vector
Delivery System πŸ”„ Viral transduction
Vector Type πŸ”„ AAVhu68
Dose 1 πŸ”„ Undisclosed

Study Record Dates


Current Stage Phase1
Submit Date 2025-08-22
Completion Date 2032-02-29
Last Update 2026-03-09

Participation Criteria


Eligible Age 18 Years - 40 Years
Standard Ages Adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations πŸ”„ Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed January 2026, initial data expected H2 2026

Resources/Links