The main objective of the project is to improve tools and scientific knowledge for the diagnosis and clinical prognosis alongside with the treatment of the neurological symptoms in patients affected by Ataxia Telangiectasia, a rare progressively disabling and life-shortening genetic disease for which no therapy is currently available.
The project will be conducted along with ATTEST, a pivotal Phase III study, conducted by EryDel with the aim of developing an effective treatment for the neurological symptoms of this devastating disorder.
The ATTEST trial will involve more than 20 centers worldwide and should allow to collect relevant safety and efficacy data for regulatory filing to obtain market authorization in the EU and the USA by 2019.
As part of the IEDAT project, additional scientific activities along with ATTEST will be conducted.
As a part of the IEDAT project, the following activities will be conducted:
a) An international patient registry will be set up 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 Ataxia Telangiectasia Society will be in charge of the register design and development.
b) A neurological scale specific for AT (AT-NEST) will be developed and tested under the responsibility of Johns Hopkins University.
c) Under the responsibility of the University of Urbino, investigations on the mechanisms of action of the EDS will be conducted with the objective to provide supportive data to the validation of a possible biomarker predictive of treatment efficacy.
d) The ATTEST phase III trial, conducted under the responsibility of EryDel, is an international, multi-center, 1 year, randomized, prospective, double-blind, placebo-controlled, designed to assess the effect of 2 dose ranges of EDS, administered monthly by IV infusion, on neurological symptoms of AT patients.
This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667946 . This publication [communication] reflects the views only of the author, and the Commission cannot be held responsible for any use which may be made of the information contained therein.
The consortium met remotely at the IEDAT General Assembly #5, on May 14th 2020. During the meeting, the latest achievements of...View more
The consortium met remotely at the IEDAT General Assembly #4, on June 6th 2020. During the meeting, the latest achievements of...View more
The consortium will meet in Naples to discuss and share the latest achievements of the project, and the ongoing clinical...View more
The IEDAT consortium will meet in Stansted (London) next October, for the second General Assembly, an important step for the...View more
IEDAT Consortium will attend and will be involved in different sessions as speakers during the meeting of the Ataxia...View more
January, 2016 - Investigator meeting of the IEDAT H2020 project organised by Erydel.View more
EU Funding for development of EDS for the treatment of Ataxia Telangiectasia (AT).
On behalf of the IEDAT Consortium, EryDel...View more
"Update on the clinical trial AtteST", by Mauro Magnani, PhD.
With a presentation "Red Blood Cells-based Drug Delivery: New applications and Current Clinical Developments" by Mauro Magnani
The final webinar has taken place on August 4th, at 2:30pm.
More details on the public deliverable D7.6 Final evaluation workshop.
Center for Targeted Therapeutics and Translational Nanomedicine (CT³N), a seminar entitled “Red Blood Cell-based Drug Delivery: our contribution to new applications and current clinical developments” with Mauro Magnani, PhD.
2020 Meeting of the European Red Cell Society, PAvia online meeting, 11 September 2020.
SIB 2019 60th CONGRESS - Italian Society of Biochemistry and Molecular Biology Lecce, September 18 – 20, 2019
The interregional mapping of SIB (Italian Society of Biochemistry and Molecular Biology) issues between Tuscany, Umbria and Marche took place at Ancona, Italy. The university of Urbino representatives were present at the congress.
The University of Urbino participated in the International Ataxia-Telangiectasia Workshop, which took place on May 1-4,2019 at the Houston Methodist Research Institute (Texas Medical Center).
Representatives of the IEDAT project participated in the Ataxia Telangiectasia Clinical Research Conference which took place from November 28 to December 1, 2018 in Naples.
Before atteding the A-T Clinical Research Conferences in Naples, the consortium meet to discuss the last achievements of the on-going Clincial Study.
DDIT4 gene expression is switched on by a new HDAC4 function in ataxia telangiectasia - Research Article, https://faseb.onlinelibrary.wiley.com/doi/full/10.1096/fj.201902039R
Proteomics and transcriptomics analyses of ataxia telangiectasia cells treated with Dexamethasone - https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0195388
The nucleoplasmic interactions among Lamin A/C‑pRB‑LAP2α‑E2F1 are modulated by dexamethasone - https://www.nature.com/articles/s41598-021-89608-3
Transcriptomic profile of ataxia telangiectasia cells treated for 30 days with a low dose of dexamethasone - Scientific article - https://www.tandfonline.com/doi/full/10.1080/26895293.2021.1911863