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 University of California, Los Angeles (UCLA)
Los Angeles, California, United States
RECRUITINGThe Ohio State University
Columbus, Ohio, United States
RECRUITINGThe Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
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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