Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Number of participants with treatment-emergent adverse events |
To evaluate the safety and tolerability of AT-007 after a single dose administered to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. |
1 Day after Dosing |
|
Primary |
Number of participants with treatment-emergent adverse events |
To evaluate the safety and tolerability of AT-007 after multiple doses administered to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. |
7 Days after Dosing |
|
Primary |
Number of participants with treatment-emergent adverse events |
To evaluate the safety and tolerability of AT-007 after multiple doses administered to Classic Galactosemia Patients, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. |
28 Days after Dosing |
|
Secondary |
Cmax of AT-007 |
Maximum (peak) plasma drug concentration |
Sequential sampling up to 48 hours following drug administration |
|
Secondary |
Tmax of AT-007 |
Time to reach maximum (peak) plasma drug concentration |
Sequential sampling up to 48 hours following drug administration |
|
Secondary |
t1/2 of AT-007 |
Terminal Elimination Half-life |
Sequential sampling up to 48 hours following drug administration |
|
Secondary |
AUClast of AT-007 |
Area under the plasma concentration-time curve from time zero to time of last quantifiable concentration |
Sequential sampling up to 48 hours following drug administration |
|
Secondary |
AUCinf of AT-007 |
Area under the plasma concentration-time curve from time zero extrapolated to infinity |
Sequential sampling up to 48 hours following drug administration |
|
Secondary |
Galactose Concentration in Plasma |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Sequential sampling for 28 days following drug administration |
|
Secondary |
Galactose Concentration in Urine |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Sequential sampling for 28 days following drug administration |
|
Secondary |
Galactitol Concentration in Plasma |
Disease Specific Biomarker in Classic Galactosemia Patients |
Sequential sampling for 28 days following drug administration |
|
Secondary |
Galactitol Concentration in Urine |
Disease Specific Biomarker in Classic Galactosemia Patients |
Sequential sampling for 28 days following drug administration |
|
Secondary |
Galactose-1-P Concentration in Erythrocytes |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Sequential sampling for 28 days following drug administration |
|