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A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Sponsor: Solid Biosciences Inc.
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
Conditions
Interventions
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