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Clinical Trials

Information on this page currently being updated this week, please check back by 1st December.

AT Clinical Trials Summary

There are some encouraging clinical trials happening at the moment in the AT scientific world, aimed at slowing the progression of AT. In December we organised a clinical research conference with A-T scientists, clinicians and representatives from pharmaceutical companies to evaluate the treatments and assess their potential for individuals with AT.

The summary of this meeting can be found here: Clinical Trials Summary

For more information please contact Penny Jeggo @ [email protected]

ATTeST Clinical Trial

ATTeST, the first multinational trial aimed at treating AT, was established by the Italian company EryDel. The AT 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 AT patients, who are still able to walk. Red blood cells taken from an AT 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 AT 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.

Trial Update 11.04.22

EryDel to Present its Phase 3 Study Results for Ataxia Telangiectasia at the American Academy of Neurology 2022 Annual Meeting

Bresso (Milano); Italy – 5 April 2022EryDel SpA, a global late-stage biotech company aimed at developing and commercializing therapies for the treatment of rare diseases delivered by its proprietary red blood cell technology, announced today that it will present data from its phase 3 ATTeST trial at the American Academy of Neurology (AAN) 2022 Annual Meeting, taking place in Seattle, Washington (April 2-7) and virtually (April 24-26).

Data on the primary and key secondary endpoints from the Phase 3 ATTeST study in Ataxia Telangiectasia (AT) will be presented. The study assessed the efficacy and safety of monthly infusions of intra-erythrocyte dexamethasone sodium phosphate in patients with AT.

For conference information, visit

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 AT. This trial aims to confirm this positive effect on AT 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 AT. However, IntraBio Inc. is keen to try and set up a centre for younger participants (aged 6 or over) and the AT 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 [email protected] or by ringing 01223 639716.