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

Recruiting Phase 1 Interventional Study
Friedreich's Ataxia (FA)
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 – 40
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
10 (estimated)
Sponsor
Solid Biosciences Inc. · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * 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 omavelox…
Contacts

Solid Biosciences Clinical Trials

+1617-337-4680

clinicaltrials@solidbio.com

CONTACT