In short
Clinical trials are investigating Vgl101 in people with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a type of leukoencephalopathy. These studies are looking mainly at safety, tolerability, and early signs of benefit. The trial data here include adults with this rare condition.
Key points
- Clinical trial data for Vgl101 focus on adults with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), a rare form of leukoencephalopathy. The study is described as a safety, tolerability, and proof-of-concept trial. It enrolled 18 participants and is listed as completed. The main outcomes were safety checks such as adverse events, lab tests, heart testing, immune response testing, and a suicide severity scale. The trial was interventional, meaning participants received the study treatment as part of the research.
Trials overview
The available clinical trial data show one interventional study of Vgl101 in adults with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
This study was designed to look at safety, tolerability, and early signs of benefit, which is called proof-of-concept.
Study population and condition
The trial focused on people with ALSP, which is a rare form of leukoencephalopathy that affects the brain’s white matter.
The source data identifies the study population as patients with this condition, but it does not list more detailed entry rules such as age ranges, disease stage, or other eligibility limits.
Study design and phase
This was an interventional study, meaning the research team gave the study treatment to participants and then measured the results.
The trial is listed as Phase 4 in the source data, and it is also described as a phase 2 safety, tolerability, and proof-of-concept study.
The intervention was Vgl101 given as a 40 mg/kg intravenous infusion, according to the trial record.
What the trial measured
The main outcome was safety and tolerability, measured by the number and type of adverse events, serious adverse events, and treatment stops because of side effects.
The study also checked safety laboratory tests, immunogenicity tests, vital signs, electrocardiograms, and the Columbia-Suicide Severity Rating Scale (C-SSRS).
These tests help researchers see whether the treatment causes medical problems, affects the heart, triggers an immune response, or raises concerns about mood or safety.
Trial status and enrollment
The study status is listed as Completed.
The enrollment was 18 participants, which means 18 people joined the study.
Patient-friendly terms
Adverse event means any unwanted medical problem that happens during a study, whether or not it is caused by the study treatment.
Serious adverse event means a severe problem that can be life-threatening or cause major harm.
Immunogenicity means the body may make an immune response against the treatment.
Electrocardiogram is a test that records the heart’s electrical activity.
C-SSRS is a questionnaire used to check for suicidal thoughts or behavior.