Clinical Trials for White Matter Lesion
There are currently 2 ongoing clinical trials investigating treatments for white matter lesion, specifically focusing on Vanishing White Matter disease. Both trials are being conducted in the Netherlands and are exploring different medications to improve safety and tolerability in patients with this rare neurological condition.
Clinical trial locations
- Netherlands
Study on the Safety and Effects of Fosigotifator in Adults and Children with Vanishing White Matter Disease
This trial is investigating Fosigotifator, a medication given as oral film-coated granules, in both adults and children diagnosed with Vanishing White Matter disease. The study aims to evaluate how safe and well-tolerated this treatment is in patients with this rare brain and nervous system condition.
Who can participate:
The trial is open to males and females in three age groups: adults 18 years and older, adolescents 12 to 17 years old, and children 6 to 11 years old. Participants must have a confirmed diagnosis of Vanishing White Matter disease through genetic testing and brain MRI scans that show signs consistent with the condition. They need a caregiver who can attend visits and help with assessments. Importantly, participants must meet specific motor or cognitive criteria, such as being unable to walk 10 or more steps even with light support, or having low scores on intelligence tests appropriate for their age. Those who are sexually active must use birth control during the study.
Who cannot participate:
The trial excludes individuals with other serious medical conditions that could interfere with the study, those currently in another clinical trial, or anyone who has had recent major surgery or is planning surgery during the study. Pregnant or breastfeeding women cannot join, nor can those with known allergies to the study medication. People with a history of drug or alcohol abuse, those unable to follow study procedures, or individuals who cannot understand the study and provide informed consent are also excluded.
What the trial involves:
The main focus is to study the safety and tolerability of Fosigotifator, which is a prodrug that converts into an active compound called A-1684909 in the body. Participants will take the medication orally and attend regular monitoring visits that include vital sign checks, physical examinations, and brain MRI scans. The study will measure how the body processes the medication by analyzing blood and cerebrospinal fluid samples. Close monitoring for any side effects or changes in condition will continue throughout the trial, which is expected to last until December 2026, with assessments continuing up to 96 weeks.
Investigational drug: Fosigotifator is the medication being tested in this trial. It is designed to convert into its active form in the body to potentially address the underlying issues in Vanishing White Matter disease.
Study on the Safety and Tolerability of Guanabenz in Children with Vanishing White Matter Disease
This clinical trial is studying Guanabenz, a medication given in capsule form, specifically in children with Vanishing White Matter disease. The study’s primary goal is to understand how safe and tolerable Guanabenz is in this young patient population, as well as to learn how the body processes the medication.
Who can participate:
The trial is for children whose parents or legal guardians provide informed consent and agree to attend all scheduled assessments for 1 to 4 years. Children of either gender can participate if they have had the disease for no more than 8 years. They must have genetically proven Vanishing White Matter disease with specific mutations in the EIF2B1-5 genes and brain scans confirming the diagnosis. The disease must have started before age 6, and the child must be able to stand and walk at least 10 steps with minimal or no support. Families must live within reasonable travel distance from Amsterdam.
Who cannot participate:
Children without a confirmed diagnosis of Vanishing White Matter disease cannot join the trial. Those outside the specified age range for the study or who belong to vulnerable populations requiring special protection are also excluded.
What the trial involves:
The study examines the safety and tolerability of Guanabenz, which is available in various dosages ranging from 1 mg to 24 mg capsules. Participants will take the medication orally according to the study protocol. Throughout the trial, researchers will monitor for any adverse events and study how the drug is absorbed, distributed, metabolized, and eliminated from the body. Regular assessments include brain MRI scans to track changes in brain structure and clinical evaluations to monitor overall health and disease progression. Guanabenz works by reducing stress in cells, which may help protect brain cells from damage. The study is expected to continue until May 2025.
Investigational drug: Guanabenz, traditionally used to treat high blood pressure, is being studied for its potential to help children with Vanishing White Matter disease by protecting brain cells from damage.
Summary
Both ongoing clinical trials for white matter lesion are concentrated in the Netherlands, reflecting the country’s expertise in researching this rare neurological condition. The trials target different patient populations: one includes both adults and children, while the other focuses exclusively on children. Each study is investigating a different medication approach—Fosigotifator and Guanabenz—both aiming to improve safety and tolerability in patients with Vanishing White Matter disease. These trials represent important steps in developing potential treatment options for this challenging condition, which currently has limited therapeutic alternatives. The research focuses on understanding not only the safety profiles of these medications but also how they might help protect or preserve brain function in affected individuals.
