Stopped: Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
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Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs
Timeframe: Up to 72 days (including screening)
Number of subjects with ISRs
Timeframe: Up to 37 days
Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)
Timeframe: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - PR interval
Timeframe: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QRS complex
Timeframe: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QT interval
Timeframe: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler
Timeframe: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - sodium
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - potassium
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - glucose
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood urea nitrogen
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - creatinine
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - calcium, chloride
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - phosphorus
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total protein
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total CO2
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - albumin
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - AST
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALT
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - GGT
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALP
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total bilirubin
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - uric acid
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - HbA1c
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - hemoglobin
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - hematocrit
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - RBC count
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - RDW
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCV
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCH
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - platelet count
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - WBC count
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ANC
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Eosinophils
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Monocytes
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Basophils
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Lymphocytes
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Bands
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Neutrophils
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Cholesterol
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - HDL cholesterol
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - LDL cholesterol
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Triglycerides
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - pH
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - protein
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ketones
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - bilirubin
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - urobilinogen
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - nitrites
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - leukocyte esterase
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - color
Timeframe: Baseline, Day 7
Change from baseline in clinical laboratory assessment - specific gravity
Timeframe: Baseline, Day 7
Change from baseline in pulse rate
Timeframe: Baseline, Day 7
Number of subjects with abnormal physical examinations
Timeframe: Up to 72 days (including screening)
Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)
Timeframe: Up to 72 days (including screening)
Number of subjects who discontinue treatment and/or study
Timeframe: Up to 37 days