A Phase Ib/II first-in-human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamic effect of intrathecal S230815 in paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy (A first-in-human study of S230815 in paediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy)

未提供0 个研究点目标入组 20 人开始时间: 待定

试验速览

阶段: 不适用
状态: 招募中
入组人数: 20

研究概览研究设计入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional
分配方式: Non Randomized

入排标准

年龄范围: 2age old over 至 12age old under（—）
性别: All

入选标准

•Male or female paediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of DEE (EIMFS or non-EIFMS EOEE phenotypes) due to a pathogenic or likely pathogenic variant in KCNT1 confirmed by central genetic testing.
•Stable dose of other regular medications and/or stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).

排除标准

•Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than EIMFS or EOEE (e.g., SHE).
•Documented pathogenic or likely pathogenic variants in any other gene known to cause epilepsy identified through prior genetic testing. Variants of uncertain significance in other genes known to cause epilepsy may be considered on discussion with the sponsor.
•Clinically significant medical history or clinical findings on physical examination, other than DEE, that in the judgment of the investigator, make the participant unsuitable for participation in the study and/or completion of the trial procedures, including, but not limited to:
•Clinically significant prior or ong/oing medical conditions within 30 days of the screening visit, as per investigator judgement.
•Clinically significant abnormality on ECG at the screening visit, as per investigator judgement.
•Clinically significant abnormality on laboratory testing at screening, including, but not limited to:
•Renal insufficiency, which is defined as creatinine clearance less than 40 mL/min assessed as estimated Glomerular Filtration Rate (eGFR) using Schwartz formula
•Hepatic derangement defined as transaminase values more than 3 times the Upper Limit of Normal (ULN) range or total bilirubin values more than 1.5 times the ULN
•Positive hepatitis B surface antigen test, positive hepatitis C antibody test, positive for human immunodeficiency virus (HIV), as reported by a laboratory test within 6 months prior to the screening visit, or on screening bloods.
•Bone, spine, bleeding disorders, or other disorder that exposes the participant to risk of injury or unsuccessful LP (e.g., haemophilia, Von Willebrand's disease, liver disease).
•Contraindications to MRI, LP procedure and IT administration.
•History of CNS tumors or malignancies, including CNS metastatic disease.
•Continuous respiratory support, defined as oxygen supplementation or non-invasive ventilation (e.g.: continuous positive airway pressure, bi-level intermittent positive airway pressure), required during waking hours. This does not include suctioning; cough assist devices or other devices that may be used regularly to clear airwways.
•Invasive ventilation including the presence of a tracheostomy.
•Use of quinidine within 30 days prior to the screening visit.
•Current use or anticipated use of antiplatelet or anticoagulant therapy during the study.
•Current or past enrolment in an interventional clinical study in which an investigational therapy is/was administered within 30 days (or 5 half-lives of study agent, whichever is longer) prior to the screening visit.
•Implantable CNS device that may interfere with the ability to administer the study drug via LP.
•Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction (e.g., changes in pulse, blood pressure, breathing function, etc...), or any other drug that in the opinion of the investigator may preclude study participation.

研究者

发起方

未提供

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