RecruitingRare disease

Study on the Safety and Effects of Fosigotifator in Adults and 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 condition known as Vanishing White Matter disease, which affects the brain and nervous system. The study will explore the effects of a treatment called Fosigotifator, which is being tested to see if it is safe and well-tolerated by both adults and children with this disease. Fosigotifator is a type of medication known as a prodrug, which means it is converted into an active form in the body, specifically into a compound called A-1684909.

The purpose of the study is to evaluate the safety and tolerability of Fosigotifator in patients with Vanishing White Matter disease. Participants will take the medication in the form of film-coated granules, which are taken orally. The study will involve regular visits to monitor the participants' health, including checking vital signs, conducting physical exams, and performing brain scans using MRI technology. The study will also measure the levels of the active compound, A-1684909, in the blood and cerebrospinal fluid over time.

Throughout the study, participants will be closely monitored for any side effects or changes in their condition. The trial will last for several weeks, with follow-up visits to ensure the safety and effectiveness of the treatment. This research aims to provide valuable insights into the potential benefits of Fosigotifator for individuals with Vanishing White Matter disease, contributing to the development of new treatment options for this challenging condition.

The research process

The trial runs in 7 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 participant will be informed about the trial's objectives and procedures. The participant must provide informed consent, or a legally authorized representative must do so on their behalf if the participant is unable to provide consent.
Step 2
Initial assessment
An initial assessment will be conducted to confirm the diagnosis of Vanishing White Matter disease. This includes a review of medical history, a physical examination, and an MRI scan to ensure consistency with the disease.
Step 3
Medication administration
The participant will begin taking the medication ABBV-CLS-7262, which is administered orally in the form of film-coated granules. The dosage and frequency will be determined by the study protocol and adjusted as necessary based on the participant's response.
Step 4
Safety evaluation period
During this period, the safety and tolerability of the medication will be closely monitored. This includes regular assessments of vital signs, ECGs, laboratory tests, and physical examinations. Any adverse effects will be documented.
Step 5
Pharmacokinetic evaluation
The study will evaluate how the body processes the active compound A-1684909. Blood samples will be collected at specific times to measure the concentration of the compound and understand its absorption, distribution, metabolism, and excretion.
Step 6
Ongoing monitoring
Throughout the study, regular visits will be scheduled to monitor the participant's health and the medication's effects. This includes assessments of brain MRI, suicidality, and other health parameters up to Week 96.
Step 7
Completion of study
The study is expected to conclude by December 27, 2026. Upon completion, a final assessment will be conducted to evaluate the overall impact of the medication on the participant's condition.

Who can join the trial?

11 criteria

Participants can be males or females who are 18 years or older, 12 to 17 years old, or 6 to 11 years old, depending on the group they belong to.
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Participants must have **Vanishing White Matter (VWM) disease**, which means:
- A doctor who knows about VWM disease has diagnosed them.
- They have a confirmed genetic test showing VWM disease.
- An MRI scan shows signs of VWM disease, confirmed by a specialist.
- Participants need a caregiver who can help with assessments and attend visits, including online ones. The caregiver must agree to give consent.
- Participants must agree to give consent to join the study. If they can't, a legal representative must give consent, and the participant must agree as much as possible.
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  Participants must meet at least one of these criteria:
  - **Motor criteria**: They cannot walk 10 or more steps, even with light support.
  - **Cognitive criteria**:
    
    For those 16 years and older: A low score on a specific intelligence test.
    For those 6 to 15 years old: A low score on certain parts of a children's intelligence test.
    Male participants who are sexually active and not sterilized must use birth control and agree not to donate sperm during the study and for 30 days after the last dose.
    Female participants who are sexually active and can have children must use birth control and agree not to donate eggs during the study and for 30 days after the last dose.

Who cannot join the trial?

8 criteria

Patients who have any other serious medical condition that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who are pregnant or breastfeeding.
Patients who have a known allergy to the study medication or its ingredients.
Patients who have a history of drug or alcohol abuse.
Patients who are unable to comply with the study procedures or follow-up visits.
Patients who have a condition that affects their ability to understand the study and provide informed consent.

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Amsterdam UMC
Amsterdam, The Netherlands

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Investigated drugs

Fosigotifator is a medication being studied for its safety and how well it is tolerated in both adults and children with Vanishing White Matter Disease. It is a prodrug, which means it is converted into an active form in the body. The study aims to understand how the body processes this medication and its potential effects on the disease.

Sodium ({(2S)-1,4-Bis[2-(4-Chloro-3-Fluorophenoxy)Acetamido]Bicyclo[2.2.2]Octan-2-Yl}Oxy)Methyl Hydrogen Phosphate-2-Amino-2-(Hydroxymethyl)Propane-1,3-Diol (1/1/1)

What is already known about the treatment

Fosigotifator – This medication is administered orally and is currently being studied in clinical trials for its safety and effectiveness in treating Vanishing White Matter disease. It is in the early stages of research, specifically in Phase 1b/2 trials, and is not yet widely recognized in medical literature. The main therapeutic indication for Fosigotifator is to address Vanishing White Matter disease, a rare neurological condition. At the molecular level, Fosigotifator acts as a prodrug, which means it is converted in the body to its active form, A-1684909, to exert its effects. It is classified pharmacologically as a prodrug, designed to improve the pharmacokinetics and therapeutic potential of its active compound.

Investigated diseases

Vanishing White Matter Disease – 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 spinal cord. Symptoms often begin in childhood and can include motor difficulties, muscle stiffness, and problems with coordination. As the disease progresses, individuals may experience worsening neurological functions, including issues with movement and speech. The condition is caused by mutations in genes responsible for the production of proteins essential for maintaining the white matter. The progression of symptoms can vary widely among individuals, with some experiencing rapid decline while others have a slower progression.