2025-523835-20-00招募中2 期
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
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 11
- 试验地点
- 10
- 主要终点
- Incidence of TEAEs and SAEs, and clinically significant changes from Baseline in safety laboratory values, vital signs, ECGs, physical/neurological exam measures, and C-SSRS summarized by study part (Part 1/SAD and Part 2/MAD).
研究概览
简要总结
To evaluate the safety and tolerability of ION337
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Participant has at least 1 authorized representative who is willing and able to give informed consent and attend all scheduled study visits.
- •Males and females age ≥ 2 to ≤ 12 years old at the time of informed consent.
- •Has a documented diagnosis of DS according to the ILAE criteria and as agreed by the 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 Period
排除标准
- •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.
- •Gain-of function variant in the SCN1A gene.
- •Current treatment with an 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.
研究组 & 干预措施
ION337
Test
干预措施: ION337 (Drug)
结局指标
主要结局
Incidence of TEAEs and SAEs, and clinically significant changes from Baseline in safety laboratory values, vital signs, ECGs, physical/neurological exam measures, and C-SSRS summarized by study part (Part 1/SAD and Part 2/MAD).
Incidence of TEAEs and SAEs, and clinically significant changes from Baseline in safety laboratory values, vital signs, ECGs, physical/neurological exam measures, and C-SSRS summarized by study part (Part 1/SAD and Part 2/MAD).
次要结局
- 1. - ION337 trough (pre-dose) in CSF and plasma for all doses in Part 1/SAD and Part 2/MAD. - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 1/SAD - Post-dose Cmax, AUC, and t1/2λz from plasma in Part 2/MAD – first dose only
- 2. Percent change from Baseline in 28-day normalized major motor seizure (MMS) frequency in Part 1/SAD and Part 2/MAD.
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.
研究点 (10)
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