U.S., Sept. 19 -- ClinicalTrials.gov registry received information related to the study (NCT07180355) titled 'A Study of SGT-212 Gene Therapy in Friedreich's Ataxia' on Aug. 22.
Brief Summary: The purpose of this study is to evaluate the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212. 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.
Study Start Date: Oct. 01
Study Type: INTERVENTIONAL
Condition:
Friedreich's Ataxia (FA...