Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients with Dravet Syndrome

Condition studied

Dravet Syndrome

Investigational medicinal product(s)

Eligibility

Primary endpoint

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).

Endpoint detail

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.