IntraBio, a UK-based biopharmaceutical company, has received approval from the US Food and Drug Administration to run a clinical trial in patients who have Ataxia-Telangiectasia (A-T). The company will test their investigational new drug, IB1001, an orally-administered version of N-Acetyl-L-Leucine, to evaluate its safety and efficacy in improving symptoms, functioning, and quality of life.
N-Acetyl-L-Leucine is the active ingredient in N-Acetyl-DL-Leucine, a modified amino acid that has been approved in France since 1957 for the treatment of vertigo. More recently, papers have been published reporting efficacy of N-Acetyl-DL-Leucine in some other cerebellar ataxias.
IntraBio is now planning to run an open-label, blinded-rater trial using three clinical sites to test this drug in A-T patients, one site in the US and two in Europe, recruiting approximately 10 participants per site. The company expects to start later this year, testing the drug in US patients who are at least 18 years of age and UK/European patients at least six years of age.
More details on this trial can be found at clinicaltrials.gov