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

Conditions

• Friedreich's Ataxia (FA)

Eligibility Criteria

Sex

ALL

Age

18 Years - 40 Years

Healthy Volunteers

Not accepted

Interventions

• SGT-212 — DRUG
  Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)

Study Details

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.

Key Dates

Start date

Oct 22, 2025

Status verified

Apr 2026

Primary completion

Mar 21, 2028

Completion

Feb 29, 2032

Study Design

Enrollment

10 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1 (Non-Ambulatory)
  Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.
• Experimental: Cohort 2 (Ambulatory)
  Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.
• Experimental: Cohort 3 (Ambulatory and Non-Ambulatory)
  Participants will receive bilateral IDN followed by systemic IV infusion.

Primary Outcome Measure

Incidence and Severity of Treatment Emergent Adverse Events (TEAEs) [ Time Frame: Month 12 ]

Central Contacts

• Solid Biosciences Clinical Trials
  +1617-337-4680
  [email protected]

Locations (3)

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