Actively Recruiting
Phase 1-2 Open-Label Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Intrathecal ION337 in Children With Dravet Syndrome
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-05-20
32
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of ION337 in children with Dravet syndrome (DS) aged 2 to 12 years. Dravet syndrome is a severe form of epilepsy, and this open-label study aims to understand how well participants tolerate this investigational treatment and monitor its safety over time. The study is divided into two parts. In Part 1, participants receive a single intrathecal bolus injection of ION337 and are observed for 6 months in a single ascending dose phase. Participants who complete Part 1 may enter Part 2, where they receive multiple doses of ION337 over 24 months in a multiple ascending dose phase, followed by a 7-month safety follow-up period. All doses are administered intrathecally. Participants will be closely monitored throughout the study with assessments including physical and neurological exams, vital signs, ECGs, laboratory tests, and tracking of treatment-emergent adverse events. Researchers will also measure seizure frequency and analyze drug levels in blood and cerebrospinal fluid. The total study duration for participants can be up to 31 months, including treatment and follow-up.
CONDITIONS
Brief 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 at the time of informed consent
- Has at least one parent or caregiver aged 18 or older willing to provide consent and attend all study visits
- Diagnosed with Dravet syndrome according to agreed criteria
- Has a confirmed pathogenic or likely pathogenic SCN1A genetic variant
- Currently receiving at least one anti-seizure medication at a stable dose for at least 4 weeks before consent
- Other epilepsy treatments and medications, including ketogenic diet or vagus nerve stimulation, stable for at least 4 weeks before consent
- Experiences the required number of major motor seizures during screening period
You will not qualify if you...
- Known brain or spinal disease that would affect lumbar puncture safety or cerebrospinal fluid circulation
- Has a pathogenic variant in another gene causing epilepsy
- Previously treated with or currently enrolled in gene therapy or antisense oligonucleotide trials for Dravet syndrome
- Treated with or enrolled in other investigational drug or device trials within 30 days or 5 half-lives of that agent
- Currently receiving anti-seizure medications that act primarily as sodium channel blockers for maintenance
- Prior brain surgeries such as corpus callosotomy, deep brain stimulation device implantation, or other palliative brain surgeries intended to reduce seizures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 6 months
Participants receive a single intrathecal bolus injection of ION337 and are monitored for safety and tolerability.
Multiple visits over 6 months for safety monitoring
Duration - Up to 24 months
Participants who complete Part 1 receive multiple doses of ION337 at the same dose level as in Part 1, with ongoing safety and tolerability assessments.
Regular visits for dosing and assessments over 24 months
Duration - 7 months
Participants are monitored for safety for 7 months after completing multiple dose treatment.
Several follow-up visits during the 7-month safety monitoring period
Trial Site Locations
Total: 3 locations
1
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
3
Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
5
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