Friedreich's Ataxia (FA)Phase 1FALCONNCT07180355
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
A Phase 1 study testing SGT-212 in people with Friedreich's Ataxia (FA), run by Solid Biosciences Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 40 years
- Conditions the study is for
- Friedreich's Ataxia (FA)
- What is being tested?
- SGT-212
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 3 sitesUnited States
- Study dates
- 22 Oct 2025to 29 Feb 2032
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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):
- 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
- For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team.
Who cannot join
- 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/m^2
- Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization
- Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)
- Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c >9%
Show all 12Show fewer
- Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening
- Has received gene therapy at any time
- Has contraindications to receiving corticosteroids
- Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212
- Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD)
- Other Inclusion/Exclusion criteria to be applied as per protocol.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
The Ohio State University
Columbus, Ohio, 43210
OSU Gene Therapy Research Team, OSUgenetherapyresearch@osumc.edu
Yasushi Kisanuki, MD
- Recruiting
The University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
Dillan Hayward, dhayward@mednet.ucla.edu
Perry Shieh, MD
- Recruiting
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
McKenzie Wells, 267-426-9608, WELLSM@chop.edu
David Lynch, MD
About this study
Official title: 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)
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
- Friedreich's Ataxia (FA)
- Being tested
- SGT-212
- Keywords
- SGT-212, Gene Therapy, Frataxin, Repeat Expansion, Neuromuscular, Cardiac, Ataxia, Movement Disorder, Rare disease, AAV, AAVhu68
- Medical subject headings
- Friedreich Ataxia, Ataxia, Movement Disorders, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SGT-212Drug
Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Non-Ambulatory) | Experimental | Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion. Receives: Drug: SGT-212 |
| Cohort 2 (Ambulatory) | Experimental | Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion. Receives: Drug: SGT-212 |
| Cohort 3 (Ambulatory and Non-Ambulatory) | Experimental | Participants will receive bilateral IDN followed by systemic IV infusion. Receives: Drug: SGT-212 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Measured: Month 12
Secondary outcomes (9)
Incidence and Severity of TEAEs
Measured: Months 18 and 60
Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)
Measured: Months 18 and 60
Number of Treatment-emergent deaths
Measured: Months 18 and 60
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
Measured: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Laboratory Tests
Measured: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Vital Signs
Measured: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Physical Examination Findings
Measured: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Innate and Adaptive Immune Responses
Measured: Baseline, Months 18 and 60
Change from Baseline in Blood Biomarkers Including Inflammatory Markers
Measured: Baseline, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2025
- Study start22 Oct 2025
- Primary completion21 Mar 2028 (estimated)
- Study completion29 Feb 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's Ataxia (FA)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Solid Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07180355
- Sponsor's protocol ID
- SGT-212-101
- Acronym
- FALCON
- Status last verified
- Nov 2026
- First submitted
- 22 Aug 2025
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07180355
- Acronym / sponsor ID
- FALCON / SGT-212-101
- Conditions
- Friedreich's Ataxia (FA)
- Keywords
- SGT-212, Gene Therapy, Frataxin, Repeat Expansion, Neuromuscular, Cardiac, Ataxia, Movement Disorder
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Oct 2025 (actual)
- Primary completion
- 21 Mar 2028 (estimated)
- Study completion
- 29 Feb 2032 (estimated)
- First posted
- 18 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Non-Ambulatory) | Experimental | Drug: SGT-212Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion. |
| Cohort 2 (Ambulatory) | Experimental | Drug: SGT-212Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion. |
| Cohort 3 (Ambulatory and Non-Ambulatory) | Experimental | Drug: SGT-212Participants will receive bilateral IDN followed by systemic IV infusion. |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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):
Key exclusion criteria (12 listed)
- 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/m^2
- Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization
- Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SGT-212Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA) | Drug | Cohort 1 (Non-Ambulatory), Cohort 2 (Ambulatory), Cohort 3 (Ambulatory and Non-Ambulatory) |
Primary outcome
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Month 12
9 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SGT-212 in people with Friedreich's Ataxia (FA), run by Solid Biosciences Inc. at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Solid Biosciences Clinical Trials (+1617-337-4680, clinicaltrials@solidbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) (time frame: Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.