Skip to main content

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-2014-2015

See other projects for this call

Sub call

H2020-PHC-2015-two-stage

Coordinator

ERYDEL SPA
Net EU contribution
€ 4 331 575,00
Address
Via Meucci 3
20091 Bresso
Italy

See on map

Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00

Participants (6)

JOHNS HOPKINS UNIVERSITY
United States
Net EU contribution
€ 217 500,00
Address
North Charles Street 3400
21218 Baltimore

See on map

Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Israel
Net EU contribution
€ 270 000,00
Non-EU contribution
€ 0,00
JOHANN WOLFGANG GOETHE-UNIVERSITAET FRANKFURT AM MAIN
Germany
Net EU contribution
€ 256 250,00
Address
Theodor W Adorno Platz 1
60323 Frankfurt Am Main

See on map

Region
Baden-Württemberg Stuttgart Stuttgart, Stadtkreis
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
Universita' degli Studi di Urbino Carlo Bo
Italy
Net EU contribution
€ 400 000,00
Address
Via Aurelio Saffi 2
61029 Urbino

See on map

Region
Centro (IT) Marche Pesaro e Urbino
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
THE ATAXIA-TELANGIECTASIA SOCIETY
United Kingdom
Net EU contribution
€ 254 000,00
Address
Iacr Rothamsted
AL5 2JQ Harpenden

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
East of England Bedfordshire and Hertfordshire Hertfordshire
Activity type
Other
Non-EU contribution
€ 0,00
ABSISKEY
France
Net EU contribution
€ 142 500,00
Address
41b Rue Dupetit Thouars
49000 Angers

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Pays de la Loire Pays de la Loire Maine-et-Loire
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00