A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
ConditionFriedreich's Ataxia (FA)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-40
SponsorSolid Biosciences Inc.
About this trial
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 criteria
Qualifiers
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
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 Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study
Disqualifiers
Antibodies against adeno-associated virus serotype 9 (AAV9)
Has a modified FARS (mFARS) score <20
Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg/m²
Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization
Trial design
Treatments tested in this trial
- SGT-212
Treatment groups
10 Participants
are divided into 3 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov