Evolving Treatment of Hemophilia's Impact on Neurodevelopment, Intelligence and Other Cognitive Functions (eTHINK)

Novo Nordisk A/S1 个研究点分布在 1 个国家目标入组 563 人开始时间: 2018年9月27日最近更新: 2019年11月22日

适应症

试验速览

阶段: 不适用
状态: 已完成
发起方: Novo Nordisk A/S
入组人数: 563
试验地点: 1
主要终点: Cognition composite score according to the BAYLEY-III instrument

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents. The eTHINK study is designed to understand whether advances in hemophilia treatment have removed any impact of hemophilia. If there still is an impact of hemophilia, the eTHINK study will help to identify which children or adolescents are still at risk, and how to screen other children. Participants and their child will participate in a comprehensive assessment of child's brain development, thinking and behavior through completing tests and questionnaires. There is only one study visit of 1 to 1.5 hours with no follow-up required

研究设计

研究类型: Observational
观察模型: Ecologic Or Community
时间视角: Cross Sectional

入排标准

年龄范围: 1 Year 至 21 Years（Child, Adult）
性别: Male
接受健康志愿者: 否

入选标准

•Male child or young adult (ages 1:0-3:6 and 4:0-21:11 years with diagnosis of congenital hemophilia A or B, any severity, with or without inhibitors - Parent able and willing to provide consent, and young adult with hemophilia (age 18-21) able to provide consent; children (ages 7-14) and adolescents/young adults (ages 14-17) able and willing to provide assent based upon local institutional policies - Parent able and willing to complete neurodevelopment and cognitive parent reported scales during comprehensive or other Hemophilia Treatment Center visit for about 45-60 minutes in English or Spanish depending on age group) - Children, adolescents and young adults with hemophilia (ages 1-21) able to participate in assessments of development and intelligence for about 40-45 minutes and adolescents and young adults (age 11-21) with hemophilia able and willing to complete additional self-assessment scales for about 30-45 minutes during comprehensive or other elective visit for in English or Spanish (depending on age group) - Provision of informed consent before the start of any study-related activities

排除标准

•Patient aged 3:7-3:11 years (43-47 months) - Prior participation in the study - Prior neurocognitive screening with the same or related instruments in the prior 6 months - Patients with hemophilia or their caregivers for whom test measures may be culturally inappropriate - Patients with hemophilia with known history of alcohol or substance abuse - Patients with hemophilia who have used opiates in the past 24 hours or other illicit drugs within the past 48 hours - Patients with hemophilia currently experiencing an acute bleed that has not resolved - Patients with hemophilia with recent head injury or concussion within the past 4 weeks - Concurrent diagnosis of another bleeding or thrombotic disorder - Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation