A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: SGT-212-101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief 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. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

Conditions

Friedreich's Ataxia (FA)

Keywords

SGT-212; Gene Therapy; Frataxin; Repeat Expansion; Neuromuscular; Cardiac; Ataxia; Movement Disorder; Rare disease; AAV; AAVhu68

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

  Month 12

Secondary Outcomes (9)

• Incidence and Severity of TEAEs

  Months 18 and 60

• Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)

  Months 18 and 60

• Number of Treatment-emergent deaths

  Months 18 and 60

• Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)

  Baseline, Months 18 and 60

• Number of Participants with Change from Baseline in Significant Abnormalities in Laboratory Tests

  Baseline, Months 18 and 60

Study Arms (3)

Cohort 1 (Non-Ambulatory)

EXPERIMENTAL

Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.

Drug: SGT-212

Cohort 2 (Ambulatory)

EXPERIMENTAL

Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.

Drug: SGT-212

Cohort 3 (Ambulatory and Non-Ambulatory)

EXPERIMENTAL

Participants will receive bilateral IDN followed by systemic IV infusion.

Drug: SGT-212

Interventions

SGT-212 DRUG

Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)

Cohort 1 (Non-Ambulatory); Cohort 2 (Ambulatory); Cohort 3 (Ambulatory and Non-Ambulatory)

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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%
• 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)

Sponsors & Collaborators

• Solid Biosciences Inc.: lead

Study Sites (3)

The University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

NOT YET RECRUITING

The Ohio State University

Columbus, Ohio, 43210, United States

RECRUITING

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia; Ataxia; Movement Disorders; Rare Diseases

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Disease Attributes; Pathologic Processes

Central Study Contacts

Solid Biosciences Clinical Trials

CONTACT

+1617-337-4680; clinicaltrials@solidbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2025
• First Posted: September 18, 2025
• Study Start: October 22, 2025
• Primary Completion (Estimated): March 21, 2028
• Study Completion (Estimated): February 29, 2032
• Last Updated: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)