ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
{ "ASCEND: ION337 for Dravet Syndrome", "This study, called ASCEND, is testing the safety and how well people tolerate a drug called ION337 in children with Dravet Syndrome (DS). Dravet Syndrome is a severe form of epilepsy. ION337 will be given by injection into the spinal fluid (ITB injection). We are looking for children aged 2 to 12 years old who have a confirmed diagnosis of DS. The main goal is to see how many participants experience side effects and how their lab tests and vital signs change over time. The study plans to enroll 32 participants. The current recruitment status is unclear.", "design": "This is an open-label study, meaning everyone knows what treatment is being given. It will enroll 32 participants and has two parts: a 6-month single dose part and a 24-month multiple dose part.", "commitments": "Participants will receive ION337 injections and attend scheduled study visits for up to 31 months, followed by a 7-month safety follow-up.", "compensation": "Not stated in the trial record.", "follow_up": "Participants will be followed for safety for up to 31 months during the study, and then for an additional 7 months after the treatment period ends.", }
- Study design
- Not specified.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Not specified.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
At a glance
Conditions
Where it's being run
4 sites across 4 statesWho to contact
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What this trial measures
- Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEsPart 1: up to 6 months; Part 2: up to 31 months
- Number of Participants With Clinically Significant Change From Baseline in Safety Laboratory ValuesPart 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 SignsPart 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)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 Physical and Neurological Examination FindingsPart 1: up to 6 months; Part 2: up to 31 months
- Parts 1 and 2: Number of Participants with Change in Columbia Suicidality Severity Rating Scale (C-SSRS)Part 1: up to 6 months; Part 2: up to 31 months