Clinical Research Directory

Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)

This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.

Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia

Ataxia-telangiectasia (A-T) is a progressive disease which is, to date, incurable, affecting motor skills, and a high cancer and recurrent infections risk. Although there has been great progress in recent years in understanding its pathophysiology, we have little information that produces any benefit for its treatment. Thus, Sirolimus has a wide variety of potential mechanisms of action that could be beneficial in A-T, including neuroprotective effects, antiangiogenic, anti-infectious, antineoplastic, and aging slower. With that aim, we conducted a pilot study in our hospital evaluating the safety and potential benefit of sirolimus in the disease.

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

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.