ION337: ION337 will be administered by ITB injection.
Study summary
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
Eligibility
Sex
ALL
Min age
2 Years
Max age
12 Years
Healthy volunteers
No
Key Inclusion Criteria:
1. Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent.
2. Participant has at least 1 parent or caregiver ≥ 18 years old who is willing and able to provide informed consent (signed and dated) and attend all scheduled study visits.
3. Has a documented diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium, Inc (ESCI).
4. Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
5. Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4 weeks prior to informed consent.
6. Must have all other interventions for epilepsy (including ketogenic diet or VNS) as well as any other concomitant medications including medications for behavioral management, sleep, and supplements or nutritional support stable for ≥ 4 weeks prior to informed consent. Vagus nerve stimulator implantation must have occurred ≥ 6 months prior to informed consent.
7. Experiences the required number of major motor seizures during the Screening Period.
Key Exclusion Criteria:
1. Known brain or spinal disease that would interfere with the LP procedure or CSF circulation, or presence of other factors that would affect the safety of the LP procedure.
2. Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
3. Has had prior treatment with or is currently enrolled in an interventional clinical trial for a gene therapy or for another antisense oligonucleotide (ASO) for the treatment of DS.
4. Has had treatment with or is currently enrolled in an interventional clinical trial of any other investigational drug, biological agent, or device within 30 days prior to Screening, or 5 half-lives of investigational agent, whichever is longer.
5. Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment.
6. Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden.
Note: Other protocol pre-specified inclusion/exclusion criteria may apply.
Primary outcome measure(s)
Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs — 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 — 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 — 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) — 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 — 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) — Part 1: up to 6 months; Part 2: up to 31 months
Trial sites (6)
Facility
City
Region
Status
University of Michigan Health System
Ann Arbor
Michigan
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Duke University Health Systems
Durham
North Carolina
Recruiting
Le Bonheur Children's Hospital
Memphis
Tennessee
Recruiting
Seattle Children's Hospital
Seattle
Washington
Recruiting
McGill University Health Center - Research Institute
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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