A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Sponsor: Solid Biosciences Inc. • ClinicalTrials.gov ID: NCT07180355
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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.
Interventions
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SGT-212
(Drug)
Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)
Eligibility criteria
Conditions
Locations
- 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
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Learn MoreOfficial Source
Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
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