Actively Recruiting
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Led by Solid Biosciences Inc. · Updated on 2026-04-27
10
Participants Needed
3
Research Sites
331 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- History of Friedreich's Ataxia symptom onset at 25 years of age or younger
- Clinical and genetic diagnosis of Friedreich's Ataxia
- Friedreich's Ataxia Rating Scale Functional Disability Staging Score of 1 or higher but less than 6
- Willing to follow rules for omaveloxolone (Skyclarys) use, including stable dosing or stopping as directed before and during the study
You will not qualify if you...
- Presence of antibodies against adeno-associated virus serotype 9 (AAV9)
- Modified Friedreich's Ataxia Rating Scale (mFARS) score less than 20
- Body weight 25 kilograms or less or body mass index (BMI) 33 kg/m² or higher
- Contraindications to endomyocardial biopsy or cardiac catheterization
- Unable to have cardiac and brain MRI with contrast due to hypersensitivity or incompatible devices
- Uncontrolled diabetes with HbA1c greater than 9%
- Participation in recent interventional studies or investigational therapy within 3 months or 5 half-lives before screening
- Previous gene therapy at any time
- Contraindications to corticosteroids
- Contraindications to the surgical procedures for IDN infusion of SGT-212
- Known cardiac disease unrelated to Friedreich's Ataxia including obstructive coronary artery disease
- Other inclusion or exclusion criteria as specified in the protocol
AI-Screening
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Trial Site Locations
Total: 3 locations
1
The University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Not Yet Recruiting
2
The Ohio State University
Columbus, Ohio, United States, 43210
Actively Recruiting
3
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
Research Team
S
Solid Biosciences Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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