2023-503320-89-00招募中2 期
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 UMC1 个研究点 分布在 1 个国家目标入组 40 人开始时间: 2024年9月26日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- All adverse events and serious adverse events collected from the start of study treatment until the end of the study, applying the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, version 5.0, 27-Nov-2017), as well as applying
研究概览
简要总结
To evaluate the safety and tolerability profile of guanabenz in pediatric patients with VWM
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •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 up to 4 years depending on time of trial entry, and adhere to the prohibitions and restrictions as specified in the protocol.
- •Male or female who has a maximum disease duration of 8 years.
- •Genetically provenVWM with 2 clinically relevant mutations in one of the EIF2B1-5 genes and a brain MRI compatible with the diagnosis.
- •Disease onset before the age of 6 years.
- •Able to stand up and walk at least 10 steps with or without the support of one hand. The “support of one hand” should be light. It does not refer to full support or to physically propelling the child forward
- •Lives within reasonable travel distance from Amsterdam.
排除标准
- •Clinically asymptomatic.
- •Comorbidity with another genetic defect.
- •Presence of an unrelated serious condition (eg, developmental anomaly, cardiac, liver or kidney disease).
- •Participation in another clinical study with therapeutic intervention.
- •Unable or unwilling to come to the study site as required by the protocol.
- •Unable to undergo MRI due to metal-containing implants, such as cochlea implant, neurostimulator or pacemaker.
- •Family situation in which adherence to the study medication or follow-up procedures cannot be guaranteed.
- •Known allergy or hypersensitivity to guanabenz or to any of the other components of the formulation used in this study.
结局指标
主要结局
All adverse events and serious adverse events collected from the start of study treatment until the end of the study, applying the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, version 5.0, 27-Nov-2017), as well as applying
All adverse events and serious adverse events collected from the start of study treatment until the end of the study, applying the most recent version of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, version 5.0, 27-Nov-2017), as well as applying
次要结局
- Guanabenz PK parameters in plasma, such as Cmax, AUC, half-life, and predicted trough concentration (Ctrough) at steady state
- Quantitative brain MRI parameters:
- Clinical parameters:
- Overall survival
研究者
Prof Dr MS van der Knaap
Scientific
Amsterdam UMC
研究点 (1)
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