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Guanabenz Acetate

Clinical trials investigating Guanabenz Acetate are studying its safety and possible benefit in Vanishing White Matter, a rare brain disorder. The trials focus on pediatric patients and children with early childhood onset disease, including a long-term extension study.

Table of Contents

Trial overview

Two interventional studies investigated Guanabenz Acetate in Vanishing White Matter, a rare condition listed in the trial records.[1][1] One study was titled “Guanabenz in VWM” and the other was an extension study titled “Guanabenz in VWM: extension study.”[1][1]

Who was studied

The first study aimed to evaluate the safety and tolerability profile of Guanabenz in pediatric patients with VWM.[1] The extension study focused on patients with early childhood onset disease, meaning the condition started at age 6 years or younger.[1]

The records do not give more detail about age, sex, or other eligibility rules beyond these target groups.[1][1]

What the trials measured

The main outcome in the first study was adverse events and serious adverse events, collected from the start of treatment until the end of the study.[1] The record says these events were assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0, which is a standard system for grading medical problems seen in trials.[1]

The extension study measured ambulation, which means the ability to walk, by looking at whether patients could walk at least 10 steps with no support or with light support from one hand.[1] This was assessed by clinical examination and by tools such as the Gross Motor Function Measure, version 88, item E, or the Health Utility Index item 9, depending on the available historical control data.[1]

Trial phases and status

Both studies were Phase 1 trials, which are early studies in clinical research.[1][1] The first study was marked Completed, and the extension study was marked Authorised.[1][1]

The planned enrollment was 40 participants for the first trial and 30 participants for the extension study.[1][1]

Trial design and treatment groups

Both studies were interventional, which means the researchers gave a treatment and then observed what happened.[1][1] The records list oral Guanabenz capsule formulations in several strengths and blend types, but they do not provide more detail about the dosing plan in the source data.[1][1]

What these studies mean

These trials show that Guanabenz Acetate was studied first in small groups of patients with Vanishing White Matter, with one study focused on safety and another on longer-term walking ability.[1][1] Because both studies were Phase 1, the main goal was early clinical research rather than a final proof of benefit.[1][1]

Trial IDPhaseCondition studiedStatusEnrollment
2023-503320-89-00Phase 1Vanishing White MatterCompleted40
2024-518834-95-02Phase 1Vanishing white matterAuthorised30

FAQ

  • What is being studied in the Guanabenz Acetate trials? These trials are studying Guanabenz Acetate in people with Vanishing White Matter. One study looks at safety and tolerability, and another looks at long-term efficacy, which means whether the treatment may help over time.
  • Who can take part in these studies? One trial enrolled pediatric patients with Vanishing White Matter. The extension study focused on patients with early childhood onset disease, meaning the condition started at age 6 years or younger.
  • What phases are the Guanabenz Acetate trials? Both trials are Phase 1 studies. Phase 1 trials are early studies that usually focus on safety and initial signs of benefit.
  • What outcomes are measured in these trials? One study measured adverse events and serious adverse events, which are unwanted medical problems during the study. The extension study measured ambulation, meaning the ability to walk, using clinical examination and motor function tools.
  • How many participants were planned for these trials? The safety study planned to enroll 40 participants, and the extension study planned to enroll 30 participants. These are small studies, which is common in early clinical research.

Ongoing Clinical Trials on Guanabenz Acetate

  • A study of guanabenz acetate for children with vanishing white matter disease who started treatment in early childhood

    Recruiting

    1 1
    Investigated drugs:
    The Netherlands

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

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

Glossary

  • Vanishing White Matter: A rare condition studied in these trials. It affects the brain's white matter, which helps signals move between different parts of the brain.
  • Pediatric patients: Children and young patients. One trial specifically studied Guanabenz Acetate in pediatric patients with Vanishing White Matter.
  • Early childhood onset: The disease begins very early in life, at age 6 years or younger. The extension study focused on this group.
  • Phase 1: An early stage of clinical research. These studies usually look first at safety and tolerability, and sometimes early signs of benefit.
  • Interventional study: A study where participants receive a treatment or intervention, such as Guanabenz Acetate, so researchers can measure its effects.
  • Safety: How well a treatment can be used without causing too many harmful problems.
  • Tolerability: How manageable the treatment is for participants, based on how well they can take it.
  • Adverse event: Any unwanted medical problem that happens during a study, whether or not it is caused by the treatment.
  • Serious adverse event: A more severe unwanted medical problem, such as one that is life-threatening or needs hospital care.
  • Ambulation: The ability to walk. In the extension study, researchers measured whether participants could walk at least 10 steps with or without light support.
  • Historical controls: People from earlier data who are used for comparison instead of a new control group in the same study.

References