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Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
EU CTIS ID: 2025-523835-20-00
What this study is testing
To evaluate the safety and tolerability of ION337
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 01. Participant has at least 1 authorized representative who is willing and able to give informed consent and attend all scheduled study visits.
- 02. Males and females age ≥ 2 to ≤ 12 years old at the time of informed consent.
- 03. Has a documented diagnosis of DS according to the ILAE criteria and as agreed by the ESCI
- 04. Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
- 05. Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4 weeks prior to informed consent.
- 06. 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.
You likely can't join if
- 01. 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.
- 02. Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
- 03. Gain-of function variant in the SCN1A gene.
- 04. Current treatment with an ASM acting primarily as a sodium channel blocker, as maintenance treatment.
- 05. Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden.
See the full eligibility criteria
- 01. Participant has at least 1 authorized representative who is willing and able to give informed consent and attend all scheduled study visits.
- 02. Males and females age ≥ 2 to ≤ 12 years old at the time of informed consent.
- 03. Has a documented diagnosis of DS according to the ILAE criteria and as agreed by the ESCI
- 04. Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
- 05. Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4 weeks prior to informed consent.
- 06. 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.
- 07. Experiences the required number of major motor seizures during the Screening Period
- 01. 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.
- 02. Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
- 03. Gain-of function variant in the SCN1A gene.
- 04. Current treatment with an ASM acting primarily as a sodium channel blocker, as maintenance treatment.
- 05. Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Australia
- Japan
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Australia; Japan; United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.