A Study to Explore the Safety, Tolerability, Pharmacokinetic Profile, and Potential Efficacy of Guanabenz in Patients With Early Childhood Onset Vanishing White Matter (VWM)

VU University Medical Center0 个研究点目标入组 30 人开始时间: 2018年11月28日最近更新: 2021年6月28日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 30

研究概览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 clinical trial of medicinal product

入排标准

性别: All

入选标准

•1.Each patient’s parents/legal guardians must sign an informed consent form (ICF) indicating that they understand the purpose of and procedures required for this study, are willing for their child to participate in the study and attend all scheduled visits, and are willing and able to comply with all study-related procedures, including maintaining contact with the site for at least 1 year, and adhere to the prohibitions and restrictions as specified in the protocol.
•Note: For each patient, both parents/legal guardians must give written consent.
•2.Male or female with a maximum disease duration at screening of 8 years.
•3.Genetically proven VWM with 2 clinically relevant mutations in one of the EIF2B1-5 genes and a brain MRI compatible with the diagnosis.
•4.Disease onset before the age of 6 years.
•5.Able to stand up and walk at least 10 steps with or without the support of one hand.
•Note: The support of one hand” should be light. It does not refer to full support or to physically propelling the child forward.
•6.Lives within reasonable travel distance from the VUmc site.
•For historical controls:
•1.Be in the same stratum as at least one patient included in the present study with respect to age at disease onset, disease duration at study entry, and disease severity category on the basis of the patient’s genotype.
•2.VWM diagnosis after 2001, the year the gene defect for VWM was identified, after which it was realized that the defect affects the integrated stress response (ISR) and circumstances activating the ISR should be avoided, such as febrile infections.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 30
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

排除标准

•1.Clinically asymptomatic.
•2.Comorbidity with another genetic defect.
•3.Presence of an unrelated serious condition (eg, developmental anomaly, cardiac, liver or kidney disease).
•4.Participation in another clinical study with therapeutic intervention.
•5.Unable or unwilling to come to the VUmc site as required by the protocol.
•6.Unable to undergo MRI due to metal-containing implants, such as cochlea implant, neurostimulator or pacemaker.
•7.Family situation in which adherence to the study medication or follow-up procedures cannot be guaranteed.
•8.Known allergy or hypersensitivity to guanabenz or to any of the other components of the formulation used in this study.

研究者

发起方

VU University Medical Center

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