Skip to main content

Personalised Treatment For Cystic Fibrosis Patients With Ultra-rare CFTR Mutations (and beyond)

Objective

In our HIT-CF project, we aim to bring personalised disease modifying therapies to cystic fibrosis (CF) patients with ultra-rare CFTR mutations, who could otherwise never get access to such treatment. Once we have proven our unique concept, the CF community can easily extend our state-of-the-art methodology to all CF patients such that HIT-CF will impact the entire CF field.

We will achieve our goals by means of a randomised, double-blind, placebo-controlled, repeated-crossover, three-armed platform trial with prospectively defined meta-analysis to evaluate efficacy at group and individual level. HIT-CF is designed to enable access to the most relevant global drug products, and each trial arm will test a drug product candidate (a single compound or a compound combination) from one of our pharmaceutical consortium partners. The patients will be assigned to the specific trial based on the effect of the drug product candidates on cultured intestinal miniature organs (termed organoids) grown from rectal biopsies, instead of based on typical genotyping only.

In parallel with this H2020 project, our pharmaceutical partners will obtain market approval of their drug product candidates for common (F508del or gating) mutations in the CFTR gene. Ultimately, our project will enable ‘managed’ off-label access to these therapies towards patient groups or individuals who show response to the therapy in a prospective intestinal organoid test.

One of the major impacts of this project will be the innovative methodologies to acquire reimbursement for current and future off-label treatments of people with CFTR mutations. This will represent a real paradigm shift in CF treatment as it implements a new type of personalized medicine paradigm based on organoids, by shifting therapeutic trials from patients to the laboratory.

Call for proposal

H2020-SC1-2016-2017

See other projects for this call

Sub call

H2020-SC1-2017-Two-Stage-RTD

Coordinator

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Net EU contribution
€ 1 434 417,50
Address
Heidelberglaan 100
3584 CX Utrecht
Netherlands

See on map

Region
West-Nederland Utrecht Utrecht
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00

Participants (10)

EUROPEAN CYSTIC FIBROSIS SOCIETY
Denmark
Net EU contribution
€ 334 740,00
Address
Kastanieparken 7
7470 Karup

See on map

Region
Danmark Midtjylland Vestjylland
Activity type
Other
Other funding
€ 0,00
PATERGRUS BVBA
Belgium
Net EU contribution
€ 302 075,00
Address
Sparhoekdreef 57
9880 Aalter

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
Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
STICHTING HUBRECHT ORGANOID TECHNOLOGY
Netherlands
Net EU contribution
€ 1 088 050,00
Address
Yalelaan 62
3584 CM Utrecht

See on map

Region
West-Nederland Utrecht Utrecht
Activity type
Other
Other funding
€ 0,00
CYSTIC FIBROSIS EUROPE EV
Germany
Net EU contribution
€ 141 312,50
Address
In Den Dauen 6
53117 Bonn

See on map

Region
Nordrhein-Westfalen Köln Bonn, Kreisfreie Stadt
Activity type
Other
Other funding
€ 0,00
FCIENCIAS.ID - ASSOCIACAO PARA A INVESTIGACAO E DESENVOLVIMENTO DE CIENCIAS
Portugal
Net EU contribution
€ 218 712,50
Address
Campo Grande, Edificio C1, Piso 3
1749 016 Lisbon

See on map

Region
Continente Área Metropolitana de Lisboa Área Metropolitana de Lisboa
Activity type
Research Organisations
Other funding
€ 0,00
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.

FACULDADE DE CIENCIAS DA UNIVERSIDADE DE LISBOA
Portugal
Net EU contribution
€ 38 655,00
Address
Campo Grande C5 Piso 3
1749-016 Lisboa

See on map

Region
Continente Área Metropolitana de Lisboa Área Metropolitana de Lisboa
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
KATHOLIEKE UNIVERSITEIT LEUVEN
Belgium
Net EU contribution
€ 386 250,00
Address
Oude Markt 13
3000 Leuven

See on map

Region
Vlaams Gewest Prov. Vlaams-Brabant Arr. Leuven
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
FLATLEY DISCOVERY LAB LLC
United States
Net EU contribution
€ 27 000,00
Other funding
€ 1 040 500,00
JULIUS CLINICAL RESEARCH BV
Netherlands
Net EU contribution
€ 2 703 152,50
Address
Broederplein 41-43
3703 CD Zeist

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
West-Nederland Utrecht Utrecht
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
GALAPAGOS

Participation ended

Belgium
Net EU contribution
€ 27 000,00
Address
Generaal De Wittelaan L11 A3
2800 Mechelen
Region
Vlaams Gewest Prov. Antwerpen Arr. Mechelen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 1 011 750,00