A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)

Summary

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Timeline

Start

2025-03-18

Primary completion

2027-12-31

Completion

2028-06-01

Outcome

Met primary endpoint

paper linear mixed model treatment effect -1·88 [SD 0·41], 95% CI -2·70 to -1·06; p<0·0001 PMID 42309084 ↗

release “Phase III trial successfully achieved its primary endpoint and key secondary endpoints with high statistical significance” intrabio.com ↗

release “Treatment with levacetylleucine demonstrated a statistically significant and clinically meaningful -1.88 point improvement of the SARA score compared to placebo” intrabio.com ↗

Publications

• Martakis K, Bremova-Ertl T, Bolton C, Foltan T, Del Mar Garcia Romero M, Gautschi M, Han V, Hahn A, Kolnikova M, Panagioti O, Perlman S, Prasad M, Schmahmann J, Skorvanek M, Thakur N, Thiel M, Hoche F. Safety and efficacy of levacetylleucine in ataxia-telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial. Lancet Neurol. 2026 Jul;25(7):633-644. doi: 10.1016/S1474-4422(26)00158-4.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Levacetylleucine	Small molecule	0.1 g/kg	Oral
Subject	Levacetylleucine	Small molecule	4 g	Oral

Indications