BridgeMD
Tests treatment safety and results for Dravet Syndrome
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 primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
- 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. Note: Other protocol pre-specified...
The study team makes the final eligibility decision.
Where it's taking place
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Memphis, Tennessee, United States
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, 2 years to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Ann Arbor, Michigan, United States; Rochester, Minnesota, United States; Memphis, Tennessee, United States. 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 ClinicalTrials.gov; the study team decides eligibility.