Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial - PubMed
5 hours ago
- #Ataxia-Telangiectasia
- #Levacetylleucine
- #Phase 3 Trial
- Levacetylleucine demonstrated a statistically significant improvement in ataxia symptoms compared to placebo, with a mean reduction in SARA score of -1.92 vs. -0.14.
- The phase 3 trial design was a double-blind, placebo-controlled crossover study conducted across multiple countries in participants aged 4 and older with ataxia-telangiectasia.
- Safety data indicated fewer adverse events with levacetylleucine (54 events in 29 patients) than placebo (75 events in 25 patients) and no treatment-related serious adverse events or deaths.
- Most participants were White (75%) and under 18 years old (64%); the study reported a favorable benefit-risk profile for treating ataxia-telangiectasia.
- An open-label extension phase is ongoing to evaluate long-term, neuroprotective, and potential disease-modifying effects of levacetylleucine.