Dravet Syndrome
Conditions
Brief summary
Incidence of TEAEs and SAEs, and clinically significant changes from Baseline in safety laboratory values, vital signs, ECGs, physical/neurological exam measures, and C-SSRS summarized by study part (Part 1/SAD and Part 2/MAD).
Detailed description
1. - ION337 trough (pre-dose) in CSF and plasma for all doses in Part 1/SAD and Part 2/MAD. - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 1/SAD - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 2/MAD – first dose only, 2. Percent change from Baseline in 28-day normalized major motor seizure (MMS) frequency in Part 1/SAD and Part 2/MAD.
Interventions
DRUGION337
Sponsors
Ionis Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of TEAEs and SAEs, and clinically significant changes from Baseline in safety laboratory values, vital signs, ECGs, physical/neurological exam measures, and C-SSRS summarized by study part (Part 1/SAD and Part 2/MAD). | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. - ION337 trough (pre-dose) in CSF and plasma for all doses in Part 1/SAD and Part 2/MAD. - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 1/SAD - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 2/MAD – first dose only, 2. Percent change from Baseline in 28-day normalized major motor seizure (MMS) frequency in Part 1/SAD and Part 2/MAD. | — |
Outcome results
None listed