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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.

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /natural sciences/biological sciences/genetics and heredity/mutation
  • /social sciences/sociology/governance

Call for proposal

H2020-SC1-2017-Two-Stage-RTD
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Address
Heidelberglaan 100
3584 CX Utrecht
Netherlands
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 434 417,50

Participants (9)

EUROPEAN CYSTIC FIBROSIS SOCIETY
Denmark
EU contribution
€ 334 740
Address
Kastanieparken 7
7470 Karup
Activity type
Other
PATERGRUS BVBA
Belgium
EU contribution
€ 302 075
Address
Sparhoekdreef 57
9880 Aalter
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
STICHTING HUBRECHT ORGANOID TECHNOLOGY
Netherlands
EU contribution
€ 1 088 050
Address
Yalelaan 62
3584 CM Utrecht
Activity type
Other
CYSTIC FIBROSIS EUROPE EV
Germany
EU contribution
€ 141 312,50
Address
In Den Dauen 6
53117 Bonn
Activity type
Other
FCIENCIAS.ID - ASSOCIACAO PARA A INVESTIGACAO E DESENVOLVIMENTO DE CIENCIAS
Portugal
EU contribution
€ 257 367,50
Address
Campo Grande, Edificio C1, Piso 3
1749 016 Lisbon
Activity type
Research Organisations
KATHOLIEKE UNIVERSITEIT LEUVEN
Belgium
EU contribution
€ 386 250
Address
Oude Markt 13
3000 Leuven
Activity type
Higher or Secondary Education Establishments
FLATLEY DISCOVERY LAB LLC
United States
EU contribution
€ 27 000
Address
45 Dan Road, Suite 320
02021 Canton, Massachusetts
Activity type
Other
JULIUS CLINICAL RESEARCH BV
Netherlands
EU contribution
€ 2 703 152,50
Address
Broederplein 41-43
3703 CD Zeist
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
GALAPAGOS

Participation ended

Belgium
EU contribution
€ 27 000
Address
Generaal De Wittelaan L11 A3
2800 Mechelen
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)