Safety and efficacy of guanabenz in early-childhood onset vanishing white matter: primary analysis of a single-arm, phase 1/2 trial.
Lancet NeurolSep 1, 2026
Marjo S van der Knaap, Renate J Verbeek, Romy J van Voorst et al.
✦ AI-curated · Sources linked
In 30 seconds
This single-arm, phase 1/2 trial evaluated the safety and efficacy of guanabenz in 31 children with early-childhood onset vanishing white matter. The study found that treated patients had a significantly lower risk of losing the ability to walk with support compared to historical controls, with a hazard ratio of 0.33 (95% CI 0.16-0.69).
Key findings
- 63 serious adverse events were reported in 25 (76%) of 33 patients.
- 48% of serious adverse events were likely related to guanabenz, including 24 hallucinations in 18 (55%) patients.
- Treated patients had a significantly lower risk of losing the ability to walk with support than historical controls (HR 0.33).
- No life-threatening events or deaths occurred during the trial.
Why it matters
The findings suggest that guanabenz may provide a therapeutic option for children with vanishing white matter, a condition with no effective treatments. Understanding its safety and efficacy is crucial for managing this severe neurodegenerative disease.