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临床试验/NCT06356233
NCT06356233尚未招募不适用

Phenotyping and Identification of Biological Markers in STXBP1 Encephalopathy

Fundación Iniciativa para las Neurociencias (FINCE)1 个研究点 分布在 1 个国家目标入组 10 人开始时间: 2024年5月1日最近更新:
适应症

试验速览

阶段
不适用
状态
尚未招募
发起方
入组人数
10
试验地点
1
主要终点
CSF biomarkers

研究概览

简要总结

This is a prospective observational study to evaluate the phenotype of 10 patients under 10 years of age with developmental epileptic encephalopathy due to mutation of the STXBP1 gene. The study will consist of a clinical and neurodevelopmental evaluation, magnetic resonance imaging, prolonged electroencephalogram, cardiological study, and analysis of biomarkers in cerebrospinal fluid. These patients will be followed up for 3 years. The aim of the study is, knowing the baseline phenotype, to analyse the response to commonly used drugs and to anticipate the response to different drugs available on the market in this group of patients based on clinical and biomarker assessment (EEG, MRI and study of specific proteins and neurotransmitters in plasma, urine and CSF).

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
1 Month 至 10 Years(Child)
性别
All
接受健康志愿者

入选标准

  • Patients under 10 years of age with confirmed mutation for STXBP
  • In cases where the diagnostic technique for the mutation is not optimal, a trio exome will be performed to confirm the mutation.

排除标准

  • Presence of functional disability that prevents the neuropsychological study from being carried out and absence of a reliable informant for the patient.

结局指标

主要结局

CSF biomarkers

时间窗: Baseline, 1 year and 2 years

EEG markers

时间窗: Baseline, 1 year and 2 years

MRI markers

时间窗: Baseline, 1 year and 2 years

次要结局

  • Clinical phenotype(Baseline, 1 year and 2 years)

研究者

发起方
Fundación Iniciativa para las Neurociencias (FINCE)
申办方类型
Other
责任方
Principal Investigator
主要研究者

Adrián Valls Carbó

MD

Fundación Iniciativa para las Neurociencias (FINCE)

研究点 (1)

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