Which patients were enrolled in HIT-CF Europe?
Patients could participate in this European project when they were 16 years of age* or older at the day of signing the informed consent for biopsy taking. They had to have a confirmed diagnosis of CF and two rare CFTR-mutations (see below: inclusion and exclusion criteria). They also needed to be able to make one visit to one of the participating hospitals to collect rectal biopsies for generating intestinal organoids. Also, if they were selected for a clinical trial they will need to visit the hospital for probably 6-8 times.
*18 years or older in some countries because of national laws
Inclusion and exclusion criteria
- Confirmed diagnosis of CF
- Sweat Chloride Concentration >60 mmol/L
- Only patients with rare genotypes could participate in the HIT-CF Europe project. Patients could not participate if they had:
- One of the following mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T, or
- A combination of any two of the following mutations: G542X, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3 and 2183AA->G
- Patients who had had a lung transplantation
- Patients with any comorbidity that might pose an additional risk in potentially administering study drugs
What is the timeline for this project?
From April 2018 on biopsies will be taken from 500 people with CF.
Recruitment of patients ended in February 2020.
The subsequent laboratory tests of drug candidates on organoids will be finished by June 2020.
Based on the responses in organoids, 78 patients will be selected for clinical trials. The clinical trials with the drug candidates are excepted to start in Autumn 2020.
Which hospitals are involved?
Hospitals that are part of the European Cystic Fibrosis Society – Clinical Trial Network (ECFS-CTN) could participate in the HIT-CF Europe project after approval of the study by individual national Ethical Committee. Hospitals that are not part of the ECFS-CTN were able to refer their patients to other participating HIT-CF Europe study sites.
All hospitals that are part of the ECFS-CTN can be found on the following website:
The recruitment phase ended in February 2020; 47 sites from 16 countries throughout Europe contributed to the enrollment of 502 patients.
For more information on this, please contact our Study team via HITCF@umcutrecht.nl
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