A Phase 1 interventional study of SGT-212 in Friedreich's Ataxia (FA), sponsored by Solid Biosciences Inc.. Recruiting at 3 sites in United States. Open to participants aged 18 Years to 40 Years. Per ClinicalTrials.gov, last updated 2026-11-06.
Sponsored by Solid Biosciences Inc. · Phase 1, Interventional, and Treatment
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.
110 studies on the registry are indexed under Friedreich Ataxia; 27 are open to participants now.
This study's planned enrollment of 10 is below the median of 30 across 75 interventional studies indexed under Friedreich Ataxia.
Browse Friedreich Ataxia studies →Solid Biosciences Inc. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Is willing to agree to the following rules for use of omaveloxolone (Skyclarys):
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.
Drug: SGT-212
Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.
Drug: SGT-212
Participants will receive bilateral IDN followed by systemic IV infusion.
Drug: SGT-212
Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Month 12
Incidence and Severity of TEAEs
Time frame: Months 18 and 60
Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)
Time frame: Months 18 and 60
Number of Treatment-emergent deaths
Time frame: Months 18 and 60
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Laboratory Tests
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Vital Signs
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Physical Examination Findings
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Innate and Adaptive Immune Responses
Time frame: Baseline, Months 18 and 60
Change from Baseline in Blood Biomarkers Including Inflammatory Markers
Time frame: Baseline, Month 12
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Solid Biosciences Inc.