A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)
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.
Eligibility
- Age range
- 18–40 years
- Sex
- All
- Healthy volunteers
- No
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 \<6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score * Is willing to agree to the following rules for use of omaveloxolone (Skyclarys): 1. For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Mo…
Interventions
- DrugSGT-212
Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)
Locations (3)
- The University of California, Los Angeles (UCLA)Los Angeles, California
- The Ohio State UniversityColumbus, Ohio
- The Children's Hospital of Philadelphia (CHOP)Philadelphia, Pennsylvania