RecruitingPhase 1NCT07180355

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)

Sponsor

Solid Biosciences Inc.

Enrollment

10 participants

Start Date

Oct 22, 2025

Study Type

INTERVENTIONAL

Conditions

Friedreich's Ataxia (FA)

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.

Eligibility

Min Age: 18 YearsMax Age: 40 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called SGT-212 for people with friedreich's ataxia (fa). The study is currently recruiting participants at 3 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSGT-212

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

Locations(3)

The University of California, Los Angeles (UCLA)

Los Angeles, California, United States

The Ohio State University

Columbus, Ohio, United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

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NCT07180355