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IEDAT Report Summary

Project ID: 667946
Funded under: H2020-EU.3.1.

Periodic Reporting for period 1 - IEDAT (Intra Erythrocyte Dexamethasone in the treatment of Ataxia Telangiectasia)

Reporting period: 2016-01-01 to 2016-12-31

Summary of the context and overall objectives of the project

Aim of this project is to provide a safe and efficacious treatment (EryDex) of the neurological symptoms to patients affected by Ataxia Telangiectasia (AT). AT is a rare progressively disabling and life-shortening genetic disease; currently no therapy is available on the market. To achieve this objective EryDel s.p.a developed EryDex which is an investigational product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes, creating the EDS end product (EDS-EP), which is infused into the patient. EryDel has just designed a worldwide pivotal Phase III study to obtain marketing authorization in EU and USA by 2019.
EryDex provides long-term delivery of low doses of dexamethasone without the typical steroid side effects. It reached a successful Phase II trial conducted in AT patients. The phase III will be an international, multi-center, 1 year, randomized, prospective, double-blind, placebo-controlled trial, to assess the effect of 2 dose ranges of EryDex, administered monthly by IV infusion, on neurological symptoms of AT patients. The protocol and the regulatory path to registration has already been discussed and agreed with FDA, EMA and the other National Regulatory Authorities involved. An international patient registry will be established with the aim of creating and maintaining a comprehensive clinical database of patients with AT. Moreover the registry is extremely useful in monitoring AT epidemiology, in developing an evidence-based natural history of the disease, in identifying biomarkers as well as developing clinical guidelines. The AT NEST, the first scale to assess symptoms specific to AT patients, coordinated by the AT center at the John’s Hopkins University, will be tested in the study and, if validated, will represent the 1st scale assessing specific AT’s areas of impairment. In parallel to the clinical trial, investigations into the molecular mechanisms of action of EryDex will be performed.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

During the first year of the project the preliminary work for clinical study was performed, and the study regulatory documentation has been prepared and submitted to local/competent authorities (WP1, WP2 and WP3). Parallel activities (Development of AT database, WP4, Definition and validation of rating scale, WP5; Mini ATM expression as biomarker, WP6) were performed in line with Annex 1 of the project.
To properly conduct the project, a Kick-off meeting and an Annual Project Assembly were held respectively in February 2016 and in November 2016. Four Technical Meetings were also performed.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

This section is not applicable to the project at the moment.
The project has the ambition to bring a satisfactory treatment for a rare disabling disease as no treatment is currently available. This ambition, to provide treatment for unmet medical need, is also supported by the complementary project objectives that will be pursued during the study, i.e. the creation of an AT EU patient registry (Obj2), the potential validation of a molecular biomarker of treatment efficacy (Obj3), and the validation of the new AT NEST scale specific for the symptoms of AT (Obj4).
These three additional activities will enhance the further understanding of the disease potentially opening to new treatment opportunities.
The objective of creating an AT EU registry (Obj2) is achievable and realistic, thanks to the participation of the UK A-T Society that will coordinate the initiative.
Objectives 3 and 4 are and reachable, as the study protocol clearly stipulates sample collection for miniATM measurement and time points of AT-NEST assessments to coincide with other efficacy endpoints in the phase III study., and the clinical study database will allow consolidating information for further statistical relationship analysis of parameters of interest.
The project therefore, advances science, creates the opportunity for the development of new therapies by the introduction of meaningful tools to monitor and understand the pathophysiology and clinical presentation of AT, and delivers a treatment for children with AT for whom no treatment is available today by the end of 2019 on market approval by the regulatory agencies.

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