NCT05564520招募中不适用
Etude Multicentrique Internationale Sur le déficit en Lipin-1
Imagine Institute1 个研究点 分布在 1 个国家目标入组 150 人开始时间: 2023年11月24日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 150
- 试验地点
- 1
- 主要终点
- Evaluation of survival in patients with Lipin-1 deficiency
研究概览
简要总结
LIPIN1 is an international registry that allows the collection of data on the follow-up, management and treatment of patients with Lipin-1 deficiency.
详细描述
This study evaluates survival, cardiac function, quality of life, fatigability, treatment tolerance and comparison of the efficacy of different current treatments in patients with Lipin-1 deficiency.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient at least 12 months with Lipin1 deficiency confirmed by molecular biology (two causal mutations in the LPIN1 gene)
排除标准
- •Opposition of the patient or his parents to participate to the study
结局指标
主要结局
Evaluation of survival in patients with Lipin-1 deficiency
时间窗: According to routine care
Patient survival as assessed by long-term follow-up
次要结局
- Evaluation the fatigability of patients with Lipin-1 deficiency(According to routine care)
- Evaluation of the quality of life of patients with Lipin-1 deficiency(According to routine care)
- Evaluation of cardiac function in patients with Lipin-1 deficiency(According to routine care)
- Comparison of the effectiveness of different current treatments in patients with Lipin-1 deficiency(According to routine care)
- Evaluation of the tolerance of treatments in patients with Lipin-1 deficiency.(According to routine care)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
不适用
Eso-Sponge RegistryLeakage After EsophagectomyLeakage After GastrectomyPerforation of the EsophagusNCT02662777Aesculap AG108
招募中
不适用
Lysosomal Acid Lipase (LAL) Deficiency RegistryLysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2Acid Lipase DeficiencyLIPA DeficiencyLAL-DeficiencyNCT01633489Alexion Pharmaceuticals, Inc.300
已完成
不适用
Observational Study to Assess Outcomes During Antipsychotic Treatment With Paliperidone ER or Other Oral AntipsychoticsSchizophreniaNCT00696813Janssen-Cilag International NV3,064
进行中（未招募）
不适用
Registry of Emergent Large veSsel oCclUsion duE to IntraCranial AtherosclerosiSIntracranial StenosisLarge Vessel OcclusionMechanical ThrombectomyNCT05403593Medical University of South Carolina600
进行中（未招募）
不适用
Post Market Registry of the CBSOAtrial Septal DefectPatent Foramen OvaleNCT04105595atHeart Medical6