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
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%
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- 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
Susan Perlman, MA
- 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
- Sep 2026
- First submitted
- 22 Aug 2025
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No