Comparing 5 approaches for dravet syndrome
Official title: ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome
- Phase 1
- 5 groups
- One site, in Montreal
- Recruiting
Interventions
- Medication
ION337
ION337 will be administered by ITB injection.
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
McGill University Health Center - Research Institute
Montreal
Eligibility Criteria
See who this study is looking for14 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
- +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 support stable for ≥ 4 weeks prior to informed consent. Vagus nerve stimulator implantation must have occurred ≥ 6 months prior to informed consent.
- +Experiences the required number of major motor seizures during the Screening PeriodScreeningThe checks done before joining, to see whether a study fits.Read more →.
Exclusion
- −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 enrolledEnrolmentThe number of participants a study plans to include, or has included.Read more → in an interventionalInterventional studyA study where participants are given something to see what happens.Read more → 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 interventional clinical trial of any other investigational drug, biological agent, or device within 30 days prior to ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →, or 5 half-lives of investigational agent, whichever is longer.
- −Current treatment with an anti-seizure medication (ASM) acting primarily as a sodium channel blocker, as maintenance treatment.
- −Note: Other protocolProtocolThe detailed plan a study must follow.Read more → pre-specified inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → may apply.
- −Prior brain surgeries including: corpus callosotomy, implantation of device for deep brain stimulation or any other palliative brain surgery intended to reduce seizure burden.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 5 groups in this study.
Groups A, B, C, D and E receive ION337.
Registry label: A: Part 1: Single Ascending Dose (SAD): Dose Level 1 · B: Part 1: SAD: Dose Level 2 · C: Part 1: SAD: Dose Level 3 · D: Part 1: SAD: Dose Level 4 · E: Part 2: Multiple Ascending Dose (MAD): Dose Level 1-4
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 2 years to 12 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 32 people.
The study is currently expected to finish around December 2030.
The main measurement is taken over: Part 1: up to 6 months; Part 2: up to 31 months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Parts 1 and 2: Number of Participants with Treatment-emergent Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAEs) and Serious TEAEs — measured over Part 1: up to 6 months; Part 2: up to 31 months.
Number of Participants With Clinically Significant Change From BaselineBaselineYour starting measurements, taken before treatment begins.Read more → in Safety Laboratory Values — measured over 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 — measured over 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) — measured over Part 1: up to 6 months; Part 2: up to 31 months.
The study lists 2 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07531745 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About ION337Drug
ION337 will be administered by ITB injection.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 2 years to 12 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 2 years to 12 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Part 1: up to 6 months; Part 2: up to 31 months.
The study as a whole is currently expected to finish around 2030-12.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 32 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 4 locations, including: Montreal, Canada; Ann Arbor, Michigan, United States; Rochester, Minnesota, United States; Seattle, Washington, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
Other Sites (3)
University of Michigan Health System
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Seattle Children's Hospital
Seattle, Washington, United States
Think this trial might be right for you?
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.