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Recruitment commences for new clinical trial

We are delighted to announce that the clinical trial of the drug N-Acetyl-L-Leucine (IB1001) for the treatment of ataxia-telangiectasia (A-T) 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.

IntraBio Trial
Ecem Kaya, PhD student in the lab of an IntraBio founding scientist Professor Fran Platt

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. Please note there are inclusion and exclusion criteria for this trial.



Welcoming the news, William Davis, chief executive of the A-T Society said. “It is very exciting that there are now two separate clinical trials for potential treatments for A-T in the UK. While we must of course remain cautious about the potential outcomes, it is a really positive sign that things are moving forward in A-T research.”

For more information about the trial, please contact Jennifer Gray, Clinical Research Manager by emailing or by ringing 01223 639717.