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

Study 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)

Interventions / Drugs

SGT-212

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment10 participants

Primary Completion—

Lead SponsorSolid Biosciences Inc.

Data Retrieved2026-04-11 04:27:33.472554+00:00

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