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Study on the Safety and Tolerability of Guanabenz in Children with Vanishing White Matter Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare disease called Vanishing White Matter (VWM), which affects the brain and is known to start in early childhood. The study is investigating the use of a medication called Guanabenz, which is taken in capsule form. The purpose of the study is to explore how safe and tolerable Guanabenz is for children with VWM, as well as to understand how the body processes the medication.

Participants in the study will take Guanabenz capsules by mouth. The study will monitor participants over a period of time to observe any side effects and to gather information on how the medication is absorbed and broken down in the body. The study will also use MRI scans to look at changes in the brain and will track the overall health and survival of the participants.

The study aims to provide valuable information about the potential benefits and risks of using Guanabenz for treating VWM in children. This research could help in developing better treatment options for this rare condition in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient's parents or legal guardians must have signed an informed consent form. This indicates understanding of the study's purpose and procedures, and a commitment to attend all scheduled assessments, either on-site or via video consultation.
The patient must meet specific criteria, including a maximum disease duration of 8 years, genetically proven vanishing white matter (VWM) with relevant mutations, disease onset before age 6, and the ability to stand and walk at least 10 steps with minimal support.
Step 2
Medication administration
The patient will receive guanabenz in capsule form. The available dosages include 1 mg, 2 mg, 4 mg, 6 mg, 8 mg, 16 mg, and 24 mg. The specific dosage and frequency will be determined by the study protocol.
The medication is administered orally. The duration of administration will be specified in the study protocol.
Step 3
Monitoring and assessments
Throughout the study, the patient will undergo regular monitoring to evaluate the safety and tolerability of guanabenz. This includes tracking any adverse events or serious adverse events.
The study will also assess the pharmacokinetic profile of guanabenz, which involves measuring how the drug is absorbed, distributed, metabolized, and excreted in the body.
Step 4
Imaging and clinical evaluations
Quantitative brain MRI scans will be conducted to assess changes in brain structure and function.
Clinical evaluations will be performed to monitor the patient's overall health and progression of VWM.
Step 5
Study duration
The study is expected to continue until May 31, 2025. The duration of participation for each patient may vary depending on the time of trial entry, ranging from 1 to 4 years.
The primary endpoint of the study is to collect data on all adverse events from the start of treatment until the end of the study.

Who can join the trial?

6 criteria

The child's parents or legal guardians must sign a form to show they understand the study and agree to their child's participation. They must also agree to attend all scheduled assessments, either in person or by video, and follow all study rules for at least 1 to 4 years.
The child can be either a boy or a girl and must have had the disease for no more than 8 years.
The child must have a **genetically proven** condition called Vanishing White Matter (**VWM**), with two important changes in one of the **EIF2B1-5 genes** and a brain scan that matches the diagnosis.
The disease must have started before the child was 6 years old.
The child must be able to stand up and walk at least 10 steps, with or without light support from one hand. This means the child can hold onto something lightly but not rely on it completely to move forward.
The child must live within a reasonable travel distance from Amsterdam.

Who cannot join the trial?

3 criteria

Patients who are not diagnosed with Vanishing White Matter cannot participate. This is a specific condition affecting the brain.
Patients who are not within the age range specified for the study cannot participate. The study is for children.
Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.

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Amsterdam UMC
Amsterdam, The Netherlands

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Investigated drugs

Guanabenz is a medication being studied for its safety and how well it is tolerated in children with a condition called Vanishing White Matter (VWM). This study aims to understand how the body processes guanabenz and to see if it might help in treating VWM.

Guanabenz Acetate

What is already known about the treatment

Guanabenz – Guanabenz is administered orally in tablet form and is currently being studied for its safety and tolerability in treating Vanishing White Matter (VWM) disease, particularly in children. It is not yet widely recognized in medical literature for this specific use, as it is still under clinical investigation. The main therapeutic indication being explored is its potential efficacy in managing symptoms of VWM. Guanabenz works by reducing stress in cells, which may help protect brain cells from damage. It is classified pharmacologically as an alpha-2 adrenergic agonist, traditionally used to treat high blood pressure.

Investigated diseases

Vanishing White Matter – This is a rare genetic disorder that primarily affects the brain and spinal cord. It is characterized by the progressive loss of white matter, which is the part of the brain that helps transmit signals between different areas of the brain and between the brain and the rest of the body. Symptoms often begin in childhood and can include motor difficulties, muscle stiffness, and problems with coordination. As the disease progresses, individuals may experience a decline in cognitive functions and other neurological issues. The rate of progression can vary, and symptoms may worsen over time.