Dravet Syndrome study: ION337
Official title ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
ClinicalTrials.gov ID: NCT07531745
What this study is testing
What is ION337?
ION337 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for dravet syndrome.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The main goal is to see if the medicine is safe and how well people can tolerate it.
- Who it's for
- This study is looking for people who have Dravet syndrome.
- What's involved
- Participants will take the study medicine and have regular check-ups to monitor their health.
- Time commitment
- The source text does not specify the total duration or the number of visits required.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 12
You may be able to join if
- Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent.
- 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...
- Has a documented diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study...
- Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
- Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4 weeks prior to informed consent.
You likely can't join if
- Known brain or spinal disease that would interfere with the LP procedure or CSF circulation, or presence of other factors that would affect the...
- Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
- Has had prior treatment with or is currently enrolled in an treatment clinical trial for a gene therapy or for another antisense oligonucleotide...
- Has had treatment with or is currently enrolled in an treatment clinical trial of any other investigational drug, biological agent, or device within...
- Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment.
- Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended...
See the full eligibility criteria
- Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent.
- 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.
- 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).
- Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
- Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4 weeks prior to informed consent.
- 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...
- Experiences the required number of major motor seizures during the Screening Period. Key
- 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.
- Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
- Has had prior treatment with or is currently enrolled in an treatment clinical trial for a gene therapy or for another antisense oligonucleotide (ASO) for the treatment of DS.
- Has had treatment with or is currently enrolled in an treatment clinical trial of any other investigational drug, biological agent, or device within 30 days prior to Screening, or 5 half-lives of investigational agent...
- Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment.
- 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 is enrolling
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Durham, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Seattle, Washington, United States
- Montreal, Canada
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, but 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, 2 years to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Ann Arbor, Michigan, United States; Rochester, Minnesota, United States; Durham, North Carolina, United States; Philadelphia, Pennsylvania, United States; Memphis, Tennessee, United States; Seattle, Washington, United States and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.