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Recruiting NCT07180355

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

Conditions: Friedreich's Ataxia (FA)

Sex: All

Ages: 18 Years – 40 Years

Healthy volunteers: No

Phase: PHASE1

Enrollment: 10

Sponsor: Solid Biosciences Inc.

Location: The University of California, Los Angeles (UCLA) Los Angeles California

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 Criteria

Inclusion Criteria: * Has history of FA symptom onset ≤25 years of age * Has a clinical and genetic diagnosis of FA * Has a staging score of ≥1 but \

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View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07180355). StuddyBuddy aggregates publicly available trial information.