Trial Now Recruiting: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia (NCT07180355)

WHY IT MATTERS

This is the first human trial of SGT-212 for Friedreich's ataxia, offering eligible patients access to a potentially disease-modifying gene therapy that addresses the underlying genetic cause rather than just managing symptoms.

Researchers are testing a new gene therapy called SGT-212 for Friedreich's ataxia, a rare disease that affects how the body moves and coordinates. This is the first time this treatment is being tested in humans. The study will enroll 10 patients and follow them for about 5 years to see if the treatment is safe and works well.

NCT ID: NCT07180355 Status: RECRUITING Conditions: Friedreich's Ataxia (FA) Phase: PHASE1 Enrollment: 10 Sponsor: Solid Biosciences Inc. 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.

YOU CAN ACT ON THIS

If you have Friedreich's ataxia, contact Solid Biosciences or your neurologist immediately to ask if you meet eligibility criteria for this recruiting trial (NCT07180355).