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

NCT07180355 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-04-27

No results posted yet for this 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

DRUG

SGT-212

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

Sponsors & Collaborators

  • Solid Biosciences Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-10-22
Primary Completion
2028-03-21
Completion
2032-02-29
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07180355 on ClinicalTrials.gov