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Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia

Objective

Primary Ciliary Dyskinesia (PCD) is a rare genetically heterogeneous disorder which results from dysfunction of motile hair-like organelles (cilia) that results in severe, chronic airways disease. Due to other cilia-related disease mechanisms several other organ systems like the heart can be affected. The complexity of the disease phenotype, late diagnosis, as well as lack of evidence based management guidelines contribute to a high burden of disease and cause high health care costs.
Therefore, there is a great need for observational trials as well as well-designed randomised controlled trials to put evidence-based diagnostic and treatment approaches into effect.
The main objective of our project is to improve diagnosis and treatment of PCD patients. To accomplish this, we propose to:
1) Establish widespread, early diagnosis by introduction of nasal Nitric Oxide measurement as screening tool, and by introduction of high-speed videomicroscopy as diagnostic tool;
2) Develop new outcome criteria, especially a PCD-specific quality of life questionnaire, as a prerequisite for controlled PCD trials;
3) Establish a PCD registry for both cross-sectional analysis of current disease status and longitudinal observational analysis of disease progression under different regimens;
4) Generate evidence-based treatment guidelines by conducting two prospective randomized trials on the inhalation of hypertonic saline and long term azithromycin therapy.
To achieve these goals members of the European Respiratory Society’s PCD task force will join forces with members of the NIH-funded US-PCD-network.
In our multi-national project, we will for the first time establish evidence-based guidelines for diagnosis, clinical management and therapy. We expect that in a high proportion of children the diagnosis will be established before irreversible lung damage has occurred. In later diagnosed individuals the disease burden will be reduced and chronic respiratory failure retarded.

Call for proposal

FP7-HEALTH-2012-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

WESTFAELISCHE WILHELMS-UNIVERSITAET MUENSTER
Address
Schlossplatz 2
48149 Munster
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 563 718
Administrative Contact
Ingo Niendorf (Mr.)

Participants (11)

UNIVERSITAET BERN
Switzerland
EU contribution
€ 393 598
Address
Hochschulstrasse 6
3012 Bern
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Claudia Kuehni (Prof.)
TECHNOLOGIKO PANEPISTIMIO KYPROU
Cyprus
EU contribution
€ 259 720
Address
Archbishop Kyprianos 31 Savings Cooperative Bank Building 3Rd Floor
3036 Lemesos
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Charalambos Yiallouros (Dr.)
THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL
United States
EU contribution
€ 213 793
Address
"Airport Drive 104 Suite 2200 Cb #1350"
27599 Chapel Hill
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Vanessa Peoples (Mrs.)
REGION HOVEDSTADEN
Denmark
EU contribution
€ 463 950,80
Address
Kongens Vaenge 2
3400 Hillerod
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Klaus Tofting (Mr.)
STICHTING VU
Netherlands
EU contribution
€ 113 053
Address
De Boelelaan 1105
1081 HV Amsterdam
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Eric Haarman (Dr.)
UNIVERSITY OF SOUTHAMPTON
United Kingdom
EU contribution
€ 193 683
Address
Highfield
SO17 1BJ Southampton
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Yan Qiao (Ms.)
UNIVERSITY OF MIAMI
United States
EU contribution
€ 108 040
Address
1320 South Dixie Highway, Suite 1200
33146 Coral Gables Miami
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Maria Ortiz (Mrs.)
INTERNATIONAL INSTITUTE OF MOLECULAR AND CELL BIOLOGY
Poland
EU contribution
€ 261 080
Address
Ks. Trojdena 4
02-109 Warsaw
Activity type
Research Organisations
Administrative Contact
Michal Witt (Prof.)
ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON
Greece
EU contribution
€ 222 934,80
Address
6 Christou Lada Str
10561 Athina
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Efi Kafentzi (Ms.)
EUROPEAN RESEARCH SERVICES GMBH
Germany
EU contribution
€ 100 284
Address
Roentgenstrasse 19
48149 Muenster
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Oliver Panzer (Mr.)
ROYAL BROMPTON AND HAREFIELD NATIONAL HEALTH SERVICE TRUST
United Kingdom
EU contribution
€ 99 820
Address
Sydney Street
SW3 6NP London
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Damian O Brien (Mr.)