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Intra Erythrocyte  Dexamethasone  in  the  treatment  of  Ataxia Telangiectasia

Intra Erythrocyte Dexamethasone in the treatment of Ataxia Telangiectasia

Objective

The main objective of the 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, a pivotal Phase III study will be conducted, to allow regulatory filing to obtain market authorization in EU and USA by 2019. EryDex is 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 will be 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 already been agreed upon with EMA and FDA.
An international patient registry will also be 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.
The AT NEST, the first scale to assess symptoms specific to AT patients, coordinated by the AT centre at the John’s Hopkins University, will be tested in the study and if validated will represent the 1st scale assessing chief areas of impairment specific to AT.
In parallel to the clinical trial, investigations into the molecular mechanisms of action of EryDex will be performed with the objective to provide the validation of a new biomarker predictive of treatment efficacy.
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Coordinator

EryDel S.p.A.

Address

Via Sasso 36
61029 Urbino

Italy

Activity type

Private for-profit entities (excluding Higher or Secondary Education Establishments)

EU Contribution

€ 4 331 575

Participants (7)

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JOHNS HOPKINS UNIVERSITY

United States

EU Contribution

€ 217 500

MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER

Israel

EU Contribution

€ 270 000

JOHANN WOLFGANG GOETHE-UNIVERSITATFRANKFURT AM MAIN

Germany

EU Contribution

€ 256 250

Universita' degli Studi di Urbino Carlo Bo

Italy

EU Contribution

€ 400 000

THE ATAXIA-TELANGIECTASIA SOCIETY

United Kingdom

EU Contribution

€ 254 000

ABSISKEY

France

EU Contribution

€ 142 500

ABSISKEY CP

France

Project information

Grant agreement ID: 667946

Status

Ongoing project

  • Start date

    1 January 2016

  • End date

    28 February 2021

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 871 825

  • EU contribution

    € 5 871 825

Coordinated by:

EryDel S.p.A.

Italy

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