TIRCON

Treat Iron-Related Childhood-Onset Neurodegeneration

 Coordinatore LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN 

 Organization address address: GESCHWISTER SCHOLL PLATZ 1
city: MUENCHEN
postcode: 80539

contact info
Titolo: Prof.
Nome: Thomas
Cognome: Klopstock
Email: send email
Telefono: +49 89 4400 57474

 Nazionalità Coordinatore Germany [DE]
 Sito del progetto http://www.tircon.eu
 Totale costo 6˙977˙141 €
 EC contributo 5˙215˙219 €
 Programma FP7-HEALTH
Specific Programme "Cooperation": Health
 Code Call FP7-HEALTH-2011-two-stage
 Funding Scheme CP-FP
 Anno di inizio 2011
 Periodo (anno-mese-giorno) 2011-11-01   -   2015-10-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

 Organization address address: GESCHWISTER SCHOLL PLATZ 1
city: MUENCHEN
postcode: 80539

contact info
Titolo: Prof.
Nome: Thomas
Cognome: Klopstock
Email: send email
Telefono: +49 89 4400 57474

DE (MUENCHEN) coordinator 1˙346˙708.42
2    KLINIKUM RECHTS DER ISAR DER TECHNISCHEN UNIVERSITAT MUNCHEN

 Organization address address: ISMANINGER STRASSE 22
city: MUENCHEN
postcode: 81675

contact info
Titolo: Ms.
Nome: Renate
Cognome: Raedlein
Email: send email
Telefono: +49 89 4140 2842
Fax: +49 89 4140 4926

DE (MUENCHEN) participant 844˙340.00
3    APO-PHARMA INC CORP

 Organization address address: SIGNET DRIVE 150
city: Toronto
postcode: M9L 1T9

contact info
Titolo: Dr.
Nome: Michael
Cognome: Spino
Email: send email
Telefono: +001 416 401 7280
Fax: +001 416 401 3878

CA (Toronto) participant 473˙382.66
4    FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA

 Organization address address: VIA CELORIA 11
city: MILANO
postcode: 20133

contact info
Titolo: Dr.
Nome: Francesca
Cognome: Grechi
Email: send email
Telefono: +39 0223942320

IT (MILANO) participant 403˙432.95
5    ACIES BIO BIOTEHNOLOSKE RAZISKAVE IN RAZVOJ DOO

 Organization address address: TEHNOLOSKI PARK 21
city: LJUBLJANA
postcode: 1000

contact info
Titolo: Dr.
Nome: Enej
Cognome: Kuscer
Email: send email
Telefono: +38 659075990
Fax: +38 659075994

SI (LJUBLJANA) participant 394˙000.00
6    ACADEMISCH ZIEKENHUIS GRONINGEN

 Organization address address: Hanzeplein 1
city: GRONINGEN
postcode: 9713 GZ

contact info
Titolo: Ms.
Nome: Johanna M
Cognome: Van Apeldoorn
Email: send email
Telefono: +31 503 633 040
Fax: +31 503 632 883

NL (GRONINGEN) participant 387˙000.00
7    OREGON HEALTH AND SCIENCE UNIVERSITY

 Organization address address: 3181 SW SAM JACKSON PARK ROAD
city: PORTLAND, OREGON
postcode: 97239-3098

contact info
Titolo: Prof.
Nome: Susan
Cognome: Hayflick
Email: send email
Telefono: +1 503 4945866
Fax: +1 503 4946886

US (PORTLAND, OREGON) participant 281˙700.00
8    CHILDREN'S HOSPITAL & RESEARCH CENTER AT OAKLAND

 Organization address address: 52nd Street 747
city: OAKLAND CA
postcode: 94609

contact info
Titolo: Ms.
Nome: Rebecca
Cognome: Rosales
Email: send email
Telefono: +1 510 450 7698

US (OAKLAND CA) participant 248˙388.85
9    BAYERISCHE FORSCHUNGSALLIANZ GEMEINNUTZIGE GMBH

 Organization address address: PRINZREGENTENSTRASSE 52
city: MUNCHEN
postcode: 80538

contact info
Titolo: Dr.
Nome: Florence
Cognome: Gauzy
Email: send email
Telefono: +49 89 9901888180

DE (MUNCHEN) participant 246˙251.60
10    UNIVERSITY OF NEWCASTLE UPON TYNE

 Organization address address: Kensington Terrace 6
city: NEWCASTLE UPON TYNE
postcode: NE1 7RU

contact info
Titolo: Dr.
Nome: Amanda
Cognome: Gregory
Email: send email
Telefono: +44 191 282 4514
Fax: +44 191 282 4524

UK (NEWCASTLE UPON TYNE) participant 244˙094.92
11    HOFFNUNGSBAUM EV VEREIN ZUR FORDERUNG DER ERFORSCHUNG UND BEHANDLUNG VON NBIA (VORMALS: HALLERVORDEN-SPATZ-SYNDROM)*TREE OF HOPE EV

 Organization address address: HARDENBERGER STRASSE 73
city: VELBERT
postcode: 42549

contact info
Titolo: Mrs.
Nome: Angelika
Cognome: Klucken
Email: send email
Telefono: +49 205168075

DE (VELBERT) participant 153˙600.00
12    NBIA DISORDERS ASSOCIATION

 Organization address address: MONACO COURT 2082
city: EL CAJON CA
postcode: 92019-4235

contact info
Titolo: Ms.
Nome: Patricia
Cognome: Wood
Email: send email
Telefono: +1 619 588 2315
Fax: +1 619 588 4093

US (EL CAJON CA) participant 114˙720.00
13    INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA

 Organization address address: Aleja Dzieci Polskich 20
city: WARSZAWA
postcode: 4730

contact info
Titolo: Dr.
Nome: Tomasz
Cognome: Kmiec
Email: send email
Telefono: +48 22 8157405

PL (WARSZAWA) participant 77˙600.40

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

kinase    us    registry    iron    characterised    efficacy    pkan    childhood    treatment    trials    clinical    investigator    patient    chelating    neurodegenerative    lack    nbia    randomized    therapeutic    biomarkers    brain    deferiprone    core    therapy    levels    reverse    pantethine    collaborative    cohort    disorders    defect    onset    models    biomaterial    group    neurodegeneration    expertise    accumulation    rare    tircon    life    amenable    precluding    addition    form    rarity    patients    pantothenate    animal    derivatives    therapies    halt    options    worldwide    drug    trial    disease   

 Obiettivo del progetto (Objective)

Neurodegeneration with brain iron accumulation (NBIA) is a heterogeneous group of rare hereditary neurodegenerative disorders characterized by high levels of brain iron. The most common form is pantothenate kinase-associated neurodegeneration (PKAN). Classic PKAN and most other NBIA cases are characterised by early childhood onset and rapid progression. Currently, there is no proven therapy to halt or reverse PKAN or any other NBIA. This is especially unfortunate as both the iron accumulation in NBIA and the biochemical defect in PKAN are predicted to be amenable to drug-based treatment. Thus, the current absence of clinical trials is not due to lack of therapeutic options but to rarity of the disease, lack of patient registries and fragmentation of therapeutic research worldwide. For example, the iron-chelating drug deferiprone has been administered to PKAN patients on an individual basis or in pilot trials, both precluding firm conclusions about its efficacy. With TIRCON, we will address this urgent and unmet need for NBIA/PKAN therapy with an ambitious and highly collaborative plan that leverages worldwide expertise. We propose a large investigator-driven randomized clinical trial of deferiprone in PKAN, bringing together leading centres and patient advocacy groups from Europe and the US to reach the required patient cohort size. In addition, together with a European SME, we propose to pursue preclinical development of pantethine and its derivatives which have shown promising efficacy in a Drosophila PKAN model. To facilitate future research, we will develop a harmonized patient registry and biomaterial bank to allow for natural history studies and biomarker development, two critical needs in NBIA research. TIRCON partners, apart from their unique clinical and basic science expertise in NBIA, have longstanding experience in investigator-driven and industry-driven randomized clinical trials. Importantly, they have been closely collaborating in recent years.

Introduzione (Teaser)

A European consortium is working on new therapies for a group of rare neurodegenerative disorders called Neurodegeneration with Brain Iron Accumulation (NBIA).

Descrizione progetto (Article)

NBIA is a group of rare disorders characterised by high levels of iron accumulation in the brain. The most frequent form of NBIA is Pantothenate Kinase-Associated Neurodegeneration (PKAN). Symptoms in PKAN and most other NBIA forms present early in childhood, and the diseases progress to life-long disability and often to early death. As currently no cure is available to reverse or halt NBIA.

Iron accumulation and the enzyme defect in PKAN could in principle be amenable to drug-based treatment but scarcity and regional dispersal of patients have rendered research fragmented, precluding conduct of randomised clinical trials. At the core of the EU-funded http://www.tircon.eu/ (TIRCON) (Treat Iron-Related Childhood-Onset Neurodegeneration) project is a clinical phase 2/3 trial to test safety and efficacy of the iron-chelating drug deferiprone in a cohort of 90 PKAN patients from Europe and US, a drug that thus far is licensed only for other indications (thalassaemia). Despite of the rarity of PKAN with a prevalence of about 1 in 1 million, TIRCON has succeeded in fully recruiting this trial, which will be completed in autumn 2016.

In addition, TIRCON's now well established internationally harmonised patient registry comprises more than 230 patients, giving access to their pseudonymised data and biomaterial samples. This sample collection is used for genomic, proteomic, transcriptomic and metabolomic analyses to better understand causes and aetiology, and to identify biomarkers of NBIA.

The consortium is also active in evaluating the potency of other candidate therapies such as pantethine and its derivatives in pre-clinical animal models. Preliminary results show improvement of locomotor defects in NBIA animal models upon treatment with pantethine, which underscores its potential clinical use.

TIRCON infrastructure and tools as well as the highly collaborative new NBIA community will facilitate future research in NBIA, for example to identify biomarkers that can serve diagnosis, prognosis, and the monitoring of disease course and treatment effects. The interventional study at the core is anticipated to improve the therapeutic options for this progressive, highly disabling and life-threatening disease.

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