NCT02090179已完成不适用
Evaluation of Blood Brain Barrier Integrity and Relationship to Structural Brain Abnormalities in MPS IIIB Patients Using Cerebrospinal Fluid/Serum Albumin Index (CSF-AI) and Multimodal Magnetic Resonance Imaging
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 5
- 主要终点
- Blood Brain Barrier integrity in MPS IIIB subjects
研究概览
简要总结
The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject and/or subject's parent or legal guardian provides informed consent
- •Subject is ≥5 years of age.
- •Subject has a definitive diagnosis of MPS IIIB, as determined by either a documented deficiency in alpha-N-acetylglucosaminidase (NAGLU) enzyme activity or documented functionally-relevant mutations in both alleles of the NAGLU gene.
排除标准
- •The subject has any internal or non-removable external metal items that may present a safety risk (for MRI), or any other medical condition or circumstance in which an MRI is contraindicated.
- •The subject has a known or suspected hypersensitivity to anaesthesia, a bleeding disorder, or any other medical condition or circumstance in which a lumbar puncture (for collection of CSF) is contraindicated.
- •Previous allergic reaction to gadolinium-based MRI contrast media.
结局指标
主要结局
Blood Brain Barrier integrity in MPS IIIB subjects
时间窗: Day 0
Blood Brain Barrier integrity in MPS IIIB subjects by estimating the CSF-AI.
Blood Brain Barrier transfer coefficient
时间窗: Day 0
The Blood Brain Barrier transfer coefficient will be measured by DCE-MRI in MPS IIIB subjects.
次要结局
- Structural brain abnormalities in MPS IIIB(Day 0)
研究者
相似试验
撤回
不适用
Evaluation of Blood Brain Barrier Integrity and Relationship to Structural Brain Abnormalities in MPS IIIB Patients Using Cerebrospinal Fluid/Serum Albumin Index (CSF-AI) and Multimodal Magnetic Resonance ImagingSanfilippo BSanfilippo disease10021605NL-OMON38912Synegeva6
招募中
不适用
Measuring blood-brain barrier integrity non-invasively with MRIdementiaNL-OMON53975Amsterdam UMC
已完成
不适用
Evaluation of Blood Brain Barrier Permeability in Chronic StrokeA Positive or a Negative Brain SPECTNCT01203800Wolfson Medical Center30
Unknown
不适用
Characteristics of Blood- Brain Barrier Permeability in Neurological PatientsTraumatic Brain InjuryCerebral InfarctionCerebral HemorrhageNCT00419874Soroka University Medical Center100
进行中（未招募）
1 期
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment of Pediatric DIPGBrain TumorNCT05615623InSightec10