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Clinical Trials

ATTeST Clinical Trial

ATTeST, the first multinational trial aimed at treating A-T, was established by the Italian company EryDel. The A-T Society was part of the consortium awarded a European Commission Grant to support the trial in 2015. The randomised, double blind, placebo controlled trial will determine if the steroid, dexamethasone sodium phosphate, will delay the progression of ataxia in A-T patients, who are still able to walk. Red blood cells taken from an A-T individual are loaded with dexamethasone, then infused back into the bloodstream. The dexamethasone diffuses slowly over 3-4 weeks, avoiding harmful side effects caused by a burst of the steroid.

Currently, at Nottingham 11 UK A-T children aged 6-12 years have progressed through the trial with 9 having completed more than 12 months of treatment. Globally there have been 175 participants in 22 centres, with only 13 (7%) leaving the trial early. Recruitment to the trial is now closed. We will keep you informed of the results, but that may not be for 1 to 2 years yet.

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.