In short
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.
Key points
- Clinical trials of Guanabenz Acetate are being done in Vanishing White Matter, a rare condition that affects the brain. The available studies are both Phase 1 trials, so they are early research studies. One trial focused on safety and tolerability in pediatric patients, while the other looked at long-term efficacy in patients with early childhood onset disease. The main outcomes included adverse events, serious adverse events, and walking ability. Together, these trials show that Guanabenz Acetate is being studied first in small patient groups before larger research can be considered.
Trial overview
Two interventional studies investigated Guanabenz Acetate in Vanishing White Matter, a rare condition listed in the trial records. One study was titled “Guanabenz in VWM” and the other was an extension study titled “Guanabenz in VWM: extension study.”
Who was studied
The first study aimed to evaluate the safety and tolerability profile of Guanabenz in pediatric patients with VWM. The extension study focused on patients with early childhood onset disease, meaning the condition started at age 6 years or younger.
The records do not give more detail about age, sex, or other eligibility rules beyond these target groups.
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. 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.
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. 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.
Trial phases and status
Both studies were Phase 1 trials, which are early studies in clinical research. The first study was marked Completed, and the extension study was marked Authorised.
The planned enrollment was 40 participants for the first trial and 30 participants for the extension study.
Trial design and treatment groups
Both studies were interventional, which means the researchers gave a treatment and then observed what happened. 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.
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. Because both studies were Phase 1, the main goal was early clinical research rather than a final proof of benefit.