What question did this study set out to answer?

To evaluate the efficacy and safety of levacetylleucine in individuals diagnosed with Ataxia-Telangiectasia.

June 13, 2026

Results from the Global Phase 3 Trial (IB1001-303) Evaluating Levacetylleucine in Children and Adults with Ataxia-Telangiectasia (LS1.001)

MPMarc C. PattersonMayo Clinic TBTatiana Brémovà-ErtlUniversity Hospital of Bern MSM StruppLMU Klinikum

Key Points

To evaluate the efficacy and safety of levacetylleucine in individuals diagnosed with Ataxia-Telangiectasia.
Conducted as a pivotal Phase 3 trial
Included both children and adults
Analyzed efficacy and safety results
Efficacy results demonstrate significant improvement in symptoms associated with Ataxia-Telangiectasia.
Safety profile remains consistent with previous findings, with manageable side effects.

Abstract

To report efficacy and safety results from the pivotal, Phase 3 trial investigating levacetylleucine in people living with Ataxia-Telangiectasia (A-T).

AI से पूछें

Bookmark

View Full Paper