Objective The objective of the current proposal is to advance the orphan drug OligoG CF-5/20 (OligoG) through a pivotal phase IIb clinical trial, to enable a new and improved therapeutic approach for the orphan disease cystic fibrosis by 2024.The study drug is an alginate oligosaccharide derived from seaweed. Several properties relevant to the treatment of CF have been demonstrated using in vitro and ex vivo model systems including release of stagnant mucus, disruption of bacterial biofilm and increased bacterial susceptibility to antibiotics. OligoG received an EU Orphan Drug Medicinal Product Designation in 2007 and an FDA Orphan Drug Designation February 2016, for the treatment of cystic fibrosis. The planned clinical trial will include approx 200 CF patients from approx. 35 European sites in coordination with the European Clinical Trial Network. The final study design, selection criteria, procedures and endpoints will be based on results from a recently finalized phase II study and will follow scientific advice and protocol assistance sought at the European Medicines Agency (EMA). A set of clinical and exploratory endpoints will be defined to assess the various effects of OligoG, comprising lung function assessed by spirometry and Lung Clearance Index (LCI) measurements, infection status assessed by frequency of pulmonary exacerbations and non-culture-dependent microbiology, and patient reported outcomes assessed by questionnaires, including standardised CF questionnaires (CFQ-R).A successful trial will enable preparation of international applications for conditional marketing authorisation (cMAA) throughout Europe and a New Drug Application (NDA) in the US, for the treatment of cystic fibrosis lung disease. In accordance with the work programme, a successful project will thus imply a new and improved therapeutic approach within cystic fibrosis available for CF patients by 2024. Fields of science natural sciencesbiological sciencesmicrobiologybacteriologysocial sciencessociologygovernancetaxationmedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsantibioticsmedical and health scienceshealth scienceshealth care servicessocial scienceseconomics and businesseconomicshealth economics Keywords cystic fibrosis pivotal clinical trial clinical development mutation independent therapy Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-PM-08-2017 - New therapies for rare diseases Call for proposal H2020-SC1-2016-2017 See other projects for this call Sub call H2020-SC1-2017-Two-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator ALGIPHARMA AS Net EU contribution € 3 224 712,50 Address INDUSTRIVEIEN 33 1337 Sandvika Norway 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 Norge Oslo og Viken Viken Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 9 374 712,50 Participants (7) Sort alphabetically Sort by Net EU contribution Expand all Collapse all SMERUD MEDICAL RESEARCH INTERNATIONAL AS Norway Net EU contribution € 566 250,00 Address KARENSLYST ALLE 6 0278 Oslo 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 Norge Oslo og Viken Oslo Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 2 501 250,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. SMERUD MEDICAL RESEARCH GERMANY GMBH Germany Net EU contribution € 1 282 500,00 Address O 7 1 68161 Mannheim 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 Baden-Württemberg Karlsruhe Mannheim, Stadtkreis Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 1 282 500,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. SMERUD MEDICAL RESEARCH UK LIMITED United Kingdom Net EU contribution € 652 500,00 Address SUITE 1S-B TRAFFORD HOUSE CHESTER ROAD TRETFORD M32 0RS Manchester 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 North West (England) Greater Manchester Greater Manchester South West Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 652 500,00 KLINIKUM DER UNIVERSITAET ZU KOELN Germany Net EU contribution € 123 037,50 Address Kerpener Strasse 62 50937 Koeln See on map Region Nordrhein-Westfalen Köln Köln, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 123 037,50 IMPERIAL COLLEGE OF SCIENCE TECHNOLOGY AND MEDICINE United Kingdom Net EU contribution € 79 990,00 Address SOUTH KENSINGTON CAMPUS EXHIBITION ROAD SW7 2AZ LONDON See on map Region London Inner London — West Westminster Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 79 990,00 EUROPEAN CYSTIC FIBROSIS SOCIETY Denmark Net EU contribution € 52 258,75 Address KASTANIEPARKEN 7 7470 Karup See on map Region Danmark Midtjylland Vestjylland Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 52 258,75 CYSTIC FIBROSIS EUROPE EV Germany Net EU contribution € 32 500,00 Address IN DEN DAUEN 6 53117 Bonn See on map Region Nordrhein-Westfalen Köln Bonn, Kreisfreie Stadt Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Total cost € 32 500,00
