Levacetylleucine (Aqneursa) improved functioning in a phase III crossover trial of adults and children with ataxia-telangiectasia.Two patients had three treatment-emergent adverse events; none were serious and all were transient.Levacetylleucine is approved to treat Niemann-Pick disease type C, but is an investigational drug for ataxia-telangiectasia.
Levacetylleucine (Aqneursa) improved neurologic symptoms and functioning in adults and children with ataxia-telangiectasia in a phase III, randomized, crossover trial.
The primary endpoint was the mean 12-week change in Scale for the Assessment and Rating of Ataxia (SARA) scores from baseline among 73 adult and pediatric ataxia-telangiectasia patients. SARA scores range from 0 (no ataxia) to 40 (severe ataxia).
At 12 weeks, treatment with oral levacetylleucine led to an improvement in SARA scores of -1.88 points compared with placebo (-1.92 points vs -0.14 points, P<0.001), reported Kyriakos Martakis, MD, of the University of Cologne in Germany, and co-authors in Lancet Neurology. A 1-point change is considered clinically meaningful.
Levacetylleucine also showed improvements in quality of life compared with placebo. Overall, 54 adverse events occurred in 29 patients on levacetylleucine, and 75 adverse events occurred in 25 patients on placebo. Two patients had three treatment-emergent adverse events related to levacetylleucine (diarrhea, eczema, and insomnia); all were transient. There were no treatment-related discontinuations, serious adverse events, or deaths.
