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 administration to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. |
Events after 1 day of administration. |
|
Primary |
Number of Participants With Treatment-emergent Adverse Events |
To evaluate the safety and tolerability of AT-007 after multiple ascending doses are administered to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. Part B and Part C are combined. |
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 of Dosing |
|
Primary |
Number of Participants With Treatment-emergent Adverse Events |
To evaluate the safety and tolerability of AT-007 administered to Classic Galactosemia Patients, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results. |
90 Days after Dosing |
|
Secondary |
Cmax of AT-007 |
Maximum (peak) plasma drug concentration |
Part A: Sampling- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs Parts B, C, D: Day of Last Dose for each Part- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs D Extension: Day 30, 60, 90- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs |
|
Secondary |
Tmax of AT-007 |
Time to reach maximum (peak) plasma drug concentration |
Part A: Sampling- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs Parts B, C, D: Day of Last Dose for each Part- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs D Extension: Day 30, 60, 90- predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, 48 hrs |
|
Secondary |
t1/2 of AT-007 |
Terminal elimination half life |
Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Parts B, C, D: Determined using Day of Last Dose for the respective Part; predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. |
|
Secondary |
AUClast of AT-007 |
Area under the plasma concentration-time curve from time zero to time of last quantifiable concentration |
Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Parts B, C, D: Determined using Day of Last Dose for the respective Part at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. |
|
Secondary |
AUCinf of AT-007 |
Area under the plasma concentration-time curve from time zero extrapolated to infinity |
Part A: Sequential sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. Part D: Determined using Day 1 with sampling at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours. |
|
Secondary |
Maximal Galactitol Reduction in Plasma |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Part D: Samples at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours on days 1, 12, 32. Part D Extension: Samples at predose, 0.5, 1, 1.5, 2, 4, 6, 8, 12, 24, 36, and 48 hours on days 1, 30, 60, & 90. |
|
Secondary |
Galactose Concentration in Plasma |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Part D: Day 32. Part D Extension: Days 30, 60, & 90. |
|
Secondary |
Galactose-1-Phosphate (Gal-1p) Concentration in Plasma |
Disease-Specific Biomarker in Classic Galactosemia Patients |
Part D: Day 32. Part D Extension: Days 30, 60, & 90. |
|