The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
This is an open-label study of ION337 in people with DS between the ages of 2 and 12 years old (inclusive). The study consists of 2 parts: Part 1) 6-month single ascending dose (SAD) and Part 2) 24-month multiple ascending dose (MAD), followed by a 7-month safety follow up.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
32
ION337 will be administered by ITB injection.
University of Michigan Health System
Ann Arbor, Michigan, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGDuke University Health Systems
Durham, North Carolina, United States
Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Number of Participants With Clinically Significant Change From Baseline in Safety Laboratory Values
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Parts 1 and 2: Number of Participants with Change in Columbia Suicidality Severity Rating Scale (C-SSRS)
Time frame: Part 1: up to 6 months; Part 2: up to 31 months
Pharmacokinetic (PK) Parameters Measure Description: Analysis of plasma concentrations of ION337
Time frame: Pre-Dose Day 1 (Dosing) until 6 months after dosing in Part 1 and up to 31 months in Part 2
Exposure of ION337 in Cerebrospinal Fluid (CSF) Measure Description: Measurement of ION337 concentrations
Time frame: Pre-dose Day 1 (Dosing) until 6 months after dosing in Part 1 and up to 31 months in Part 2
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Le Bonheur Children's Hospital
Memphis, Tennessee, United States
RECRUITINGSeattle Children's Hospital
Seattle, Washington, United States
RECRUITINGMcGill University Health Center - Research Institute
Montreal, Canada
RECRUITINGParts 1 and 2: Percent Change From Baseline in 28-day Normalized Major Motor Seizure (MMS) Frequency
Time frame: Part 1: up to 6 months; Part 2: up to 31 months