PART 1 – organoid study

Between April 2018 and February 2020, biopsies were taken from 502 people with CF. 47 sites from 16 countries throughout Europe contributed to enroll people in HIT-CF, a huge effort.

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. 

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.

*18 years or older in some countries because of national laws

Inclusion and exclusion criteria

Inclusion criteria:

• Confirmed diagnosis of CF
• Sweat Chloride Concentration >60 mmol/L

Exclusion criteria:

• 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?

Recruitment of patients ended in February 2020.

The subsequent laboratory tests of drug candidates on organoids was finished by end 2021.

Based on the responses in organoids, 52 patients will be selected for CHOICES. This clinical trial will start in the summer of 2022.

The HIT-CF project had to deal with severe delays because of the Covid-19 pandemic and because of important rearrangements within the pharmaceutical industry partners. You can find more information about this in our newsletters.