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Clinical Trials

N-Acetyl-L-Leucine (IB1001) Clinical Trial

January 2020. The clinical trial of the drug N-Acetyl-L-Leucine (IB1001) for the treatment of ataxia-telangiectasia is now recruiting adult participants in the UK, at the Royal Papworth Hospital site in Cambridge.

In a series of small-scale studies, the drug appeared to improve the neurological symptoms in a number of different hereditary ataxias, including A-T. This trial aims to confirm this positive effect on A-T in a larger study.

The trial is being organised by IntraBio Inc, a company specialising in discovering, developing, and commercialising novel therapies and treatments for rare and neurodegenerative diseases.

The study at Papworth is only open to adults (aged 18+) with A-T. However, IntraBio Inc. is keen to try and set up a centre for younger participants (aged 6 or over) and the A-T Society is working with them to try and make this happen.

The trial will consist of three study phases: a baseline period, a 6-week treatment period where the drug will be administered to patients orally, and a 6-week post-treatment washout period. For each individual participant, the study will last for approximately 3.5 – 4 months during which 6 visits to the Royal Papworth Hospital will be required. Participants (and a carer) will be reimbursed for reasonable out-of-pocket expenses incurred for participating in the trial, such as travel and parking.

For more information about the trial and the inclusion and exclusion criteria, please contact Hannah Munday, Senior Clinical Trial Coordinator by emailing or by ringing 01223 639716.