RecruitingPhase 1

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

United States10 participantsStarted 2025-10-22

Plain-language 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.

Who can participate

Age range18 Years – 40 Years

SexALL

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Inclusion criteria

✓. 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.

What they're measuring

Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

Timeframe: Month 12

Trial details

NCT IDNCT07180355

SponsorSolid Biosciences Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-03-21

Contact for this trial

Solid Biosciences Clinical Trials

+1617-337-4680 clinicaltrials@solidbio.com

View on ClinicalTrials.gov More Friedreich's Ataxia (FA) trials