NL-OMON24121招募中不适用

A study to explore the safety, tolerability, pharmacokinetic profile, and potential efficacy of Guanabenz in patients with early childhood onset Vanishing White Matter (VWM)

Amsterdam University Medical Centers, location VUmc, De Boelelaan 1117, 1081HV Amsterdam0 个研究点目标入组 30 人开始时间: 待定最近更新: 2024年2月28日

研究概览

暂无简介。

•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 assessments (on site or by video consultation as indicated per protocol), 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 who has a maximum disease duration 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 light support of one hand.
•6.Lives within reasonable travel distance from Amsterdam.

•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 study 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.

发起方

Amsterdam University Medical Centers, location VUmc, De Boelelaan 1117, 1081HV Amsterdam