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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Adeno-associated virus serotype AAVhu68 containing a codon-optimized complementary DNA (cDNA)
The Ohio State University
Columbus, Ohio, United States
RECRUITINGThe Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGIncidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Time frame: Month 12
Incidence and Severity of TEAEs
Time frame: Months 18 and 60
Incidence and Severity of Treatment-emergent Serious adverse events (SAEs)
Time frame: Months 18 and 60
Number of Treatment-emergent deaths
Time frame: Months 18 and 60
Change from Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Laboratory Tests
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Vital Signs
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Significant Abnormalities in Physical Examination Findings
Time frame: Baseline, Months 18 and 60
Number of Participants with Change from Baseline in Innate and Adaptive Immune Responses
Time frame: Baseline, Months 18 and 60
Change from Baseline in Blood Biomarkers Including Inflammatory Markers
Time frame: Baseline, Month 12
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