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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.

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

ERYDEL SPA
Address
Via Meucci 3
20091 Bresso
Italy
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 4 331 575

Participants (6)

JOHNS HOPKINS UNIVERSITY
United States
EU contribution
€ 217 500
Address
North Charles Street 3400
21218 Baltimore
Activity type
Higher or Secondary Education Establishments
MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Israel
EU contribution
€ 270 000
Address
Tel Hashomer Sheba Medical Center
52621 Ramat Gan
Activity type
Research Organisations
JOHANN WOLFGANG GOETHE-UNIVERSITATFRANKFURT AM MAIN
Germany
EU contribution
€ 256 250
Address
Theodor W Adorno Platz 1
60323 Frankfurt Am Main
Activity type
Higher or Secondary Education Establishments
Universita' degli Studi di Urbino Carlo Bo
Italy
EU contribution
€ 400 000
Address
Via Aurelio Saffi 2
61029 Urbino
Activity type
Higher or Secondary Education Establishments
THE ATAXIA-TELANGIECTASIA SOCIETY
United Kingdom
EU contribution
€ 254 000
Address
Iacr Rothamsted
AL5 2JQ Harpenden
Activity type
Other
AK GROUP
France
EU contribution
€ 142 500
Address
41B Rue Dupetit Thouars
49000 Angers
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)