Health Trials

Friedreich's Ataxia (FA)

1

Review clinical trials related to Friedreich's Ataxia (FA). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

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

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.

Participants needed: 10
Trial details
Phase: Phase 1Age: 18-40Biological sex: AllType: InterventionalSponsor: Solid Biosciences Inc.Updated: Jun 22, 2026Locations: 3
Eligibility criteria

Has history of FA symptom onset ≤25 years of age [+4]

Antibodies against adeno-associated virus serotype 9 (AAV9) [+11]

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