Levacetylleucine Shows Promise in Ataxia-Telangiectasia

Ataxia-telangiectasia is a progressive neurodegenerative disorder characterized by cerebellar ataxia, immunodeficiency, and increased risk for malignancy. There are no approved therapies. In a new phase 3 trial, researchers investigated the potential benefits of levacetylleucine in patients with genetically confirmed ataxia-telangiectasia.

Overall, 73 participants ≥4 years of age were randomly assigned to levacetylleucine or placebo for 12 weeks, after which they crossed over to the opposite treatment for another 12 weeks. The primary end point was the change in the Scale for the Assessment and Rating of Ataxia (SARA; a 40-point scale) from baseline to the end of each 12-week period.