Actively Recruiting
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-05-04
32
Participants Needed
2
Research Sites
243 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
CONDITIONS
Official Title
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant is aged 2 to 12 years old at the time of informed consent.
- Has at least one parent or caregiver aged 2 who can provide informed consent and attend all study visits.
- Documented diagnosis of Dravet syndrome based on ILAE criteria and ESCI agreement.
- Confirmation of a pathogenic or likely pathogenic SCN1A variant.
- Currently receiving at least one anti-seizure medication at a stable dose for at least 4 weeks before consent.
- All other epilepsy treatments, including ketogenic diet or vagus nerve stimulator (VNS), and any other medications or supplements stable for at least 4 weeks before consent.
- Vagus nerve stimulator implantation must have been done at least 6 months before consent.
- Experiences the required number of major motor seizures during the screening period.
You will not qualify if you...
- Known brain or spinal disease that would interfere with lumbar puncture or cerebrospinal fluid circulation, or other safety concerns for lumbar puncture.
- Pathogenic or likely pathogenic variant in another gene causing epilepsy.
- Prior or current treatment in an interventional trial for gene therapy or another antisense oligonucleotide for Dravet syndrome.
- Prior or current treatment in an interventional trial of any investigational drug, biological agent, or device within 30 days before screening or for 5 half-lives of the agent, whichever is longer.
- Current treatment with an anti-seizure medication acting mainly as a sodium channel blocker.
- Previous brain surgeries including corpus callosotomy, deep brain stimulation device implantation, or other palliative brain surgeries aimed at reducing seizures.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
2
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here