NCT07180355

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)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Solid Biosciences Inc. · Industry
• Sex: All
• Age: 18 Years – 40 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	SGT-212	Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)

Timeline

• Start date: 2025-10-22
• Primary completion: 2028-03-21
• Completion: 2032-02-29
• First posted: 2025-09-18
• Last updated: 2026-03-09

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study