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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, 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 [email protected] or by ringing 01223 639717.