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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Solid Biosciences Inc.
Study ID
NCT07180355
Phase
PHASE1
Status
Recruiting
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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

First listed
Sep 18, 2025
Start date
Oct 22, 2025
Status verified
Jun 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

Locations (3)

FacilityCityStateZIPSite coordinators
The University of California, Los Angeles (UCLA)Los AngelesCalifornia90095
Dillan Hayward
[email protected]
Susan Perlman, MA (PRINCIPAL_INVESTIGATOR)
The Ohio State UniversityColumbusOhio43210
OSU Gene Therapy Research Team
[email protected]
Yasushi Kisanuki, MD (PRINCIPAL_INVESTIGATOR)
The Children's Hospital of Philadelphia (CHOP)PhiladelphiaPennsylvania19104
McKenzie Wells
[email protected]
267-426-9608
David Lynch, MD (PRINCIPAL_INVESTIGATOR)

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By research site
The University of California, Los Angeles (UCLA)· Los Angeles, CAThe Ohio State University· Columbus, OHThe Children's Hospital of Philadelphia (CHOP)· Philadelphia, PA