FREE WEBINAR

IEDAT Horizon2020 Project: Final Event

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The main objective of the IEDAT project is to provide a treatment of the neurological symptoms of patients with Ataxia Telangiectasia (AT), a rare progressively disabling and life-shortening genetic disease for which no therapy is currently available. To achieve this, the IEDAT Project has developped solutions, which will be presented during this Final Event, to conclude the project.

  • ► EryDex, an innovative product developed by EryDel, used to administer dexamethasone sodium phosphate by ex-vivo encapsulation in autologous erythrocytes, which are infused into the patient. EryDex provides long-term delivery of low doses of dexamethasone without the typical steroid side effects and has reached a successful Phase II trial conducted in AT patients. The phase III trial has been an international, multi-center, 1 year, randomized, prospective, double-blind, placebo-controlled, designed to assess the effect of 2 dose ranges of EryDex, administered monthly by IV infusion, on neurological symptoms of AT patients. The protocol of the trial and the regulatory path to registration has been agreed upon with EMA and FDA.

    ► ICARS, the International Cooperative Ataxia Rating Scale for pharmacological assessment of the cerebellar syndrome. The scale involves a compartimentalized quantification of postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders. A modified version of the ICARS has been identified as Primary Efficacy Endpoint for the ATTeST clinical study supported by the IEDAT project.
  • ► The AT NEST, the first scale to assess symptoms specific to AT patients, coordinated by the AT centre at the John’s Hopkins University, has been tested in parallel to the study and represents the 1st scale with the scope of assessing main areas of impairment specific to AT.

    ► MiniATM expression as biomarker. Ataxia telangiectasia (AT) results from biallelic mutations in the ataxia telangiectasia-mutated (ATM) gene. Recently, an effect of dexamethasone on the partial function recovery of the mutated gene, with the production of new ATM protein variant (miniATM) that retains partial enzymatic activity, has been demonstrated. This may explain in part the beneficial effect of EryDex on neurological symptoms of AT. In parallel to the clinical trial, investigations into the molecular mechanisms of action of EryDex has been performed with the objective to provide the validation of a new MiniATM biomarker predictive of treatment efficacy.

  • ► An international patient registry has also been set with the aim of establishing and maintaining a comprehensive clinical database of patients with AT and closely related conditions, enabling the monitoring of AT epidemiology, the development of an evidence-based natural history of the condition, identification of biomarkers as well as development of clinical guidelines.
  • Absiskey

  • ERYDEL

  • Johns Hopkins University

  • Chaim Sheba Medical Center

  • Goethe University

  • University of Urbino

  • A-T Society

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