Tundra Space

Tundra Space

Clinical Research Directory

Browse clinical research sites, groups, and studies.

Back to Studies
RECRUITING
NCT07180355
PHASE1

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

Sponsor: Solid Biosciences Inc.

View on ClinicalTrials.gov

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.

Official title: 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)

Key Details

Gender

All

Age Range

18 Years - 40 Years

Study Type

INTERVENTIONAL

Enrollment

10

Start Date

2025-10-22

Completion Date

2032-02-29

Last Updated

2026-03-09

Healthy Volunteers

No

Interventions

DRUG

SGT-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